Sodium 4-Phenylbutyrate | 4-PBA sodium;4-Phenylbutyric acid sodium;TriButyrate;NSC 657802;Benzenebutanoic Acid;NaPB;4-Phenylbutyric acid;Sodium Phenylbutyrate;4-PBA;Benzenebutyric acid sodium | 4-苯基丁酸钠盐 | Apoptosis Inducer | HDAC

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Sodium 4-Phenylbutyrate/苯丁酸钠 {[allProObj[0].p_purity_real_show]}

货号：A455726 同义名： 4-苯基丁酸钠盐 / 4-PBA sodium; 4-Phenylbutyric acid sodium

Sodium 4-Phenylbutyrate (4-PBA sodium) 是一种HDAC和内质网（ER）应激的抑制剂，用于癌症和感染研究。

Sodium 4-Phenylbutyrate/苯丁酸钠化学结构
CAS号：1716-12-7

Sodium 4-Phenylbutyrate/苯丁酸钠 3D分子结构
CAS号：1716-12-7

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Sodium 4-Phenylbutyrate/苯丁酸钠纯度/质量文件产品仅供科研

货号：A455726 标准纯度： {[allProObj[0].p_purity_real_show]} 产品说明书

批次查询：批次纯度: COA SDS NMR HPLC CNMR LC-MS

全球学术期刊中引用的产品: • Nature, 2025, 645, 793-800. Ambeed. [ A201204 , A444152 , A344107 , A952055 ]; • Cell, 2025. Ambeed. [ A122167 ]; • Science, 2025, 387(6729): eadp5637. Ambeed. [ A875019 ]; • Sig. Transduct. Target. Ther., 2025, 10, 257. Ambeed. [ A104916 ]; • Nat. Nanotechnol., 2025. Ambeed. [ A243018 , A1216705 , A522597 , A125401 , A1355641 ]; 更多 >

组蛋白去乙酰化酶亚型比较

产品名称	HD1 ↓ ↑	HD2 ↓ ↑	HDAC ↓ ↑	HDAC1 ↓ ↑	HDAC10 ↓ ↑	HDAC11 ↓ ↑	HDAC2 ↓ ↑	HDAC3 ↓ ↑	HDAC4 ↓ ↑	HDAC5 ↓ ↑	HDAC6 ↓ ↑	HDAC7 ↓ ↑	HDAC8 ↓ ↑	HDAC9 ↓ ↑	其他靶点	纯度
Givinostat HCl monohydrate	++++ HD1-B, IC50: 7.5 nM HD1-A, IC50: 16 nM	+++ HD2, IC50: 10 nM														99%+
MC1568	++ HD1-B (Maize), IC50: 3.4 μM HD1-A (Maize), IC50: 100 nM															96%
Trichostatin A			++++ HDAC, IC50: ~1.8 nM													99%+
Scriptaid			✔													99%+
Valproic acid sodium			✔												Autophagy	97%
AR-42			+++ HDAC, IC50: 30 nM													99%+
Dacinostat			+++ HDAC, IC50: 32 nM													98+%
CUDC-101			++++ HDAC, IC50: 4.4 nM	++++ HDAC1, IC50: 4.5 nM	+++ HDAC10, IC50: 26.1 nM		+++ HDAC2, IC50: 12.6 nM	++++ HDAC3, IC50: 9.1 nM	+++ HDAC4, IC50: 13.2 nM	+++ HDAC5, IC50: 11.4 nM	++++ HDAC6, IC50: 5.1 nM	+ HDAC7, IC50: 373 nM	++ HDAC8, IC50: 79.8 nM	++ HDAC9, IC50: 67.2 nM	EGFR,HER2	99%+
M344			++ HDAC, IC50: 100 nM													99%+
Splitomicin			+ Sir2p, IC50: 60 μM													99%
Panobinostat			++++ HDAC (Reh cells), IC50: 20 nM HDAC (MOLT-4 cells), IC50: 5 nM													98%
Sodium 4-Phenylbutyrate			✔													98%
Vorinostat			+++ HDAC, IC50: ~10 nM													98%
Curcumin			✔												Nrf2,NF-κB	98%
Belinostat			+++ HDAC, IC50: 27 nM													98%
RG-2833				++ HDAC1, Ki: 32 nM				++ HDAC3, Ki: 5 nM								98%
Valproic acid				+ HDAC1, IC50: 0.4 mM												98%
BG45				+ HDAC1, IC50: 2 μM			+ HDAC2, IC50: 2.2 μM	+ HDAC3, IC50: 289 nM								99%+
Entinostat				+ HDAC1, IC50: 0.51 μM				+ HDAC3, IC50: 1.7 μM								98%
Resminostat				+++ HDAC1, IC50: 42.5 nM				++ HDAC3, IC50: 50.1 nM			++ HDAC6, IC50: 71.8 nM					98+%
Romidepsin				+++ HDAC1, IC50: 36 nM			+++ HDAC2, IC50: 47 nM									99%+
Parthenolide				✔											NF-κB,p53	97% HPLC
Tacedinaline				+ HDAC1, IC50: 0.9 μM			+ HDAC2, IC50: 0.9 μM	+ HDAC3, IC50: 1.2 μM								98%
Mocetinostat				++ HDAC1, IC50: 0.15 μM		+ HDAC11, IC50: 0.59 μM	+ HDAC2, IC50: 0.29 μM	+ HDAC3, IC50: 1.66 μM								98%
WT-161				++++ HDAC1, IC50: 8.35 nM			+++ HDAC2, IC50: 15.4 nM				++++ HDAC6, IC50: 0.4 nM					99%+
Fimepinostat				++++ HDAC1, IC50: 1.7 nM	++++ HDAC10, IC50: 2.8 nM	++++ HDAC11, IC50: 5.4 nM	++++ HDAC2, IC50: 5.0 nM	++++ HDAC3, IC50: 1.8 nM			+++ HDAC6, IC50: 27 nM					99%+
Tucidinostat				++ HDAC1, IC50: 95 nM	++ HDAC10, IC50: 78 nM		++ HDAC2, IC50: 160 nM	++ HDAC3, IC50: 67 nM								99%+
Santacruzamate A							++++ HDAC2, IC50: 119 pM									99%+
(E,E)-RGFP966								++ HDAC3, IC50: 80 nM								99%+
LMK-235									+++ HDAC4, IC50: 11.9 nM	++++ HDAC5, IC50: 4.2 nM						99%+
Tasquinimod									✔							99%+
CAY10603											++++ HDAC6, IC50: 2 pM					98%
Tubastatin A											+++ HDAC6, IC50: 15 nM					98%
Tubacin											++++ HDAC6, IC50: 4 nM					99%+
ACY-738											++++ HDAC6, IC50: 1.7 nM					99%+
Nexturastat A											++++ HDAC6, IC50: 5 nM					99%+
BRD73954											+++ HDAC6, IC50: 36 nM		++ HDAC8, IC50: 120 nM			99%
Tubastatin A HCl											+++ HDAC6, IC50: 15 nM		+ HDAC8, IC50: 854 nM			98%
PCI-34051													+++ HDAC8, IC50: 10 nM			99%+
Ricolinostat				++ HDAC1, IC50: 58 nM			++ HDAC2, IC50: 48 nM	++ HDAC3, IC50: 51 nM			++++ HDAC6, IC50: 4.7 nM		++ HDAC8, IC50: 100 nM			99%+
Droxinostat								+ HDAC3, IC50: 16.9 μM			+ HDAC6, IC50: 2.47 μM		+ HDAC8, IC50: 1.46 μM			99%+
Abexinostat				++++ HDAC1, Ki: 7 nM	+++ HDAC10, IC50: 24 nM		+++ HDAC2, Ki: 19 nM	++++ HDAC3/SMRT, Ki: 8.2 nM			+++ HDAC6, Ki: 17 nM		+ HDAC8, IC50: 280 nM			98%+
Citarinostat				+++ HDAC1, IC50: 35 nM			+++ HDAC2, IC50: 45 nM	+++ HDAC3, IC50: 46 nM			++++ HDAC6, IC50: 2.6 nM		++ HDAC8, IC50: 137 nM			99%+
HPOB				+ HDAC1, IC50: 2.9 μM	+ HDAC10, IC50: 3.0 μM		+ HDAC2, IC50: 4.4 μM	+ HDAC3, IC50: 1.7 μM			++ HDAC6, IC50: 56 nM		+ HDAC8, IC50: 2.8 μM			97%
Quisinostat 2HCl				++++ HDAC1, IC50: 0.11 nM	++++ HDAC10, IC50: 0.46 nM	++++ HDAC11, IC50: 0.37 nM	++++ HDAC2, IC50: 0.33 nM	++++ HDAC3, IC50: 4.86 nM	++++ HDAC4, IC50: 0.64 nM	++++ HDAC5, IC50: 3.69 nM			++++ HDAC8, IC50: 4.26 nM			97%
Domatinostat				+ HDAC1, IC50: 1.20 μM	+ HDAC10, IC50: 21 μM	+ HDAC11, IC50: 9.7 μM	+ HDAC2, IC50: 1.12 μM	+ HDAC3, IC50: 0.57 μM		+ HDAC5, IC50: 11.3 μM				+ HDAC9, IC50: 50 μM		99%+
TMP269									++ HDAC4, IC50: 157 nM	++ HDAC5, IC50: 97 nM		+++ HDAC7, IC50: 43 nM		+++ HDAC9, IC50: 23 nM		99%+
Pracinostat				++ HDAC1, IC50: 49 nM	+++ HDAC10, IC50: 40 nM	++ HDAC11, IC50: 93 nM	++ HDAC2, IC50: 96 nM	+++ HDAC3, IC50: 43 nM	++ HDAC4, IC50: 56 nM	+++ HDAC5, IC50: 47 nM	+ HDAC6, IC50: 1.008 μM	++ HDAC7, IC50: 137 nM	++ HDAC8, IC50: 140 nM	++ HDAC9, IC50: 70 nM		99%+
TMP195									++ HDAC4, Ki: 59 nM	++ HDAC5, Ki: 60 nM		+++ HDAC7, Ki: 26 nM		+++ HDAC9, Ki: 15 nM		99%+
1. 鼠标悬停在“+”上可以显示相关IC50的具体数值。"+"越多，抑制作用越强。2. "✔"表示该化合物对相应的亚型有抑制作用，但抑制强度暂时没有相关数据。

Sodium 4-Phenylbutyrate/苯丁酸钠生物活性

靶点

HDAC

描述

Sodium 4-Phenylbutanoate (Sodium Phenylbutyrate) is the sodium salt of phenylbutyrate, a derivative of the short-chain fatty acid butyrate, with potential antineoplastic activity. Phenylbutyrate reversibly inhibits class I and II histone deacetylases (HDACs), which may result in a global increase in gene expression, decreased cellular proliferation, increased cell differentiation, and the induction of apoptosis in susceptible tumor cell populations. Sodium 4-phenylbutanoate (NaPB) can induce cellular differentiation and cell cycle arrest. NaPB treatment led to time dependent growth inhibition in hepatocellular carcinoma cells Bel-7402. NaPB treatment caused a significant decline in the fraction of S phase cells and a significant increase in G0/G1 cells. NaPB increased the expression of P21(WAF1/CIP1) and E-cadherin in Bel-7402 and significantly decreased the level of HDAC4 in Bel-7402. NaPB significantly improved the level of acetylating histone H4^[3]. Pre- or post-treatment with 4-PBA at therapeutic doses attenuated infarction volume, hemispheric swelling, and apoptosis and improved neurological status in a mouse model of hypoxia-ischemia. Moreover, 4-PBA suppressed ER-mediated apoptosis by inhibiting eukaryotic initiation factor 2alpha phosphorylation, CCAAT/enhancer-binding protein homologous protein induction, and caspase-12 activation. 4-PBA could protect against cerebral ischemia through inhibition of ER stress-mediated apoptosis and inflammation^[4]. 4-phenylbutyrate protects cells from the deleterious effects of ER(endoplasmic reticulum)-retained aggregated mutant myocilin^[5].

Sodium 4-Phenylbutyrate/苯丁酸钠细胞实验

Cell Line rat heart cells MDA-MB-231 cells HeLa cells bone marrow-derived early outgrowth cells (EOCs) Tomato plant leaves Human Brain Microvascular Endothelial Cells (HBMECs) ND1-mutant cybrids NIH-3T3 cells human hepatocytes (from OTCD patients)	Concentration	Treated Time	Description	References
rat heart cells	10 mM	30 min	relieves ER stress and improves contractile function	J Am Heart Assoc. 2013 May 17;2(3):e004796.
MDA-MB-231 cells	20 μM	24 h	Reduce UPR activation and protect cells from apoptosis	Cell Death Dis. 2013 Jul 18;4(7):e732.
HeLa cells	5 mM	4 h	To evaluate the effect of 4-PBA on GRP78 protein levels and caspase-12 activity. 4-PBA significantly reduced the increase of GRP78 induced by δ-T3 and TRF treatment and significantly inhibited the γ-T3- and δ-T3-induced increase of caspase-12 activity.	Genes Nutr. 2016 Dec 23;11:32.
bone marrow-derived early outgrowth cells (EOCs)	50 μM	7 days	To investigate the effect of ER stress on EOC function under high glucose conditions, 4-phenylbutyrate significantly alleviated HG-induced ER stress, apoptosis, and functional impairment.	Diabetologia. 2015 Sep;58(9):2181-90.
Tomato plant leaves	1 mM	6 h	To evaluate how a chemical chaperone modulates Tm-induced ER stress, it was found that it decreased the Tm-induced increase in SlBiP, SlIRE1a, and SlBI1 transcripts	Biomolecules. 2020 Jul 10;10(7):1031.
Human Brain Microvascular Endothelial Cells (HBMECs)	10 μM	1 h	Inhibition of Tat-induced ER stress and apoptosis	Mol Neurobiol. 2016 Jan;53(1):132-142.
ND1-mutant cybrids	1 mM	48 h	To investigate the protective effects of 4-Phenylbutyrate on ND1-mutant cells under energetic stress conditions. Results showed that 4-Phenylbutyrate partially attenuated the expression of ER stress markers such as Bip/GRP78, CHOP, and sXBP1 mRNA levels.	Cell Death Dis. 2018 May 31;9(6):658.
NIH-3T3 cells	20 μM	24/48 h	Increase cell folding capacity, reduce UPR activation, and protect transformed cells from death	Cell Death Dis. 2013 Jul 18;4(7):e732.
human hepatocytes (from OTCD patients)	0.01, 0.1, and 1 mM	2 weeks	To evaluate the effect of 4-PB and SB on reducing ammonia levels in hepatocytes from OTCD patients. Results showed that 4-PB treatment was more effective than SB, and hetero-OTCD hepatocytes were more susceptible than hemi-OTCD hepatocytes.	J Inherit Metab Dis. 2021 May;44(3):618-628.

Sodium 4-Phenylbutyrate/苯丁酸钠动物实验

Species ApoE-/- mice Rats	Animal Model 5/6 nephrectomy-induced chronic kidney disease model Neonatal ventral hippocampal lesion model	Administration	Dosage	Frequency	Description	References
ApoE-/- mice	5/6 nephrectomy-induced chronic kidney disease model	Intraperitoneal injection	250 mg/kg	Once daily for 8 weeks	To investigate the effect of 4-PBA on CKD-dependent vascular calcification. Results showed that 4-PBA treatment significantly reduced aortic calcified lesions.	J Am Heart Assoc. 2013 Sep 5;2(5):e000238
Rats	Neonatal ventral hippocampal lesion model	Subcutaneous injection	20 mg/kg	Daily from the day after surgery until adulthood (week 9)	To investigate whether the HDAC inhibitor phenylbutyrate could counteract long-term consequences of neonatal ventral hippocampal lesions in rats. Results showed that phenylbutyrate treatment reduced hypersensitivity of the dopaminergic system and deterioration of associative learning, but did not affect anxiety or anatomical brain changes.	Front Psychiatry. 2011 Jan 7;1:153

Sodium 4-Phenylbutyrate/苯丁酸钠临床研究

NCT号	适应症或疾病	临床期	招募状态	预计完成时间	地点
NCT00999167	Cirrhosis Hep... 展开 >>atic Encephalopathy 收起 <<	Phase 2	Completed	-	-
NCT01135680	Drug Toxicity	Phase 1	Completed	-	United States, Wisconsin ... 展开 >> Covance Clinical Pharmacology, Inc. Madison, Wisconsin, United States, 53704 收起 <<
NCT00999167	-		Completed	-	-

Sodium 4-Phenylbutyrate/苯丁酸钠参考文献

[1]Gardian G, Yang L, Cleren C, Calingasan NY, Klivenyi P, Beal MF. Neuroprotective effects of phenylbutyrate against MPTP neurotoxicity. Neuromolecular Med. 2004;5(3):235-41.

[2]Goh M, Chen F, Paulsen MT, Yeager AM, Dyer ES, Ljungman M. Phenylbutyrate attenuates the expression of Bcl-X(L), DNA-PK, caveolin-1, and VEGF in prostate cancer cells. Neoplasia. 2001 Jul-Aug;3(4):331-8.

[3]Wang CT, Meng M, Zhang JC, Jin CJ, Jiang JJ, Ren HS, Jiang JM, Qin CY, Yu DQ. Growth inhibition and gene induction in human hepatocellular carcinoma cell exposed to sodium 4-phenylbutanoate. Chin Med J (Engl). 2008 Sep 5;121(17):1707-11

[4]Qi X, Hosoi T, Okuma Y, Kaneko M, Nomura Y. Sodium 4-phenylbutyrate protects against cerebral ischemic injury. Mol Pharmacol. 2004 Oct;66(4):899-908

[5]Yam GH, Gaplovska-Kysela K, Zuber C, Roth J. Sodium 4-phenylbutyrate acts as a chemical chaperone on misfolded myocilin to rescue cells from endoplasmic reticulum stress and apoptosis. Invest Ophthalmol Vis Sci. 2007 Apr;48(4):1683-90

Sodium 4-Phenylbutyrate/苯丁酸钠实验方案

计算器

存储液制备

1mg

5mg

10mg

1 mM

5 mM

10 mM

5.37mL

1.07mL

0.54mL

26.86mL

5.37mL

2.69mL

53.71mL

10.74mL

5.37mL

Sodium 4-Phenylbutyrate/苯丁酸钠技术信息

CAS号	1716-12-7
分子式	C₁₀H₁₁NaO₂
分子量	186.18
SMILES Code	O=C([O-])CCCC1=CC=CC=C1.[Na+]
MDL No.	MFCD00800247

别名	4-苯基丁酸钠盐 ;4-PBA sodium; 4-Phenylbutyric acid sodium; TriButyrate; NSC 657802; Benzenebutanoic Acid; NaPB; 4-Phenylbutyric acid; Sodium Phenylbutyrate; 4-PBA; Benzenebutyric acid sodium
运输	蓝冰
InChI Key	VPZRWNZGLKXFOE-UHFFFAOYSA-M
Pubchem ID	5258

存储条件

In solvent -20°C: 3-6个月 -80°C: 12个月

Pure form Sealed in dry,2-8°C

溶解方案

细胞实验：

H₂O: 100 mg/mL(537.11 mM)，配合低频超声助溶

动物实验：配制的工作液建议现用现配，短期内尽快用完。以下溶剂前显示的百分比是指该溶剂在终溶液中的体积占比；如在配制过程中出现沉淀、析出现象，可以通过加热和/或超声的方式助溶。

方案一

临床研究

NCT号	适应症或疾病	临床期	招募状态	预计完成时间	地点

NCT00999167	Cirrhosis Hep... 展开 >>atic Encephalopathy 收起 <<	Phase 2	Completed	-	-
NCT01135680	Drug Toxicity	Phase 1	Completed	-	United States, Wisconsin ... 展开 >> Covance Clinical Pharmacology, Inc. Madison, Wisconsin, United States, 53704 收起 <<
NCT00999167	-		Completed	-	-
NCT01347073	Urea Cycle Disorders	Phase 3	Completed	-	United States, California ... 展开 >> UCLA Pediatrics/Genetics Los Angeles, California, United States, 90404 United States, District of Columbia Children's National Medical Center Washington, District of Columbia, United States, 20010 United States, Maine Maine Medical Center Portland, Maine, United States, 04101 United States, Minnesota University of Minnesota Minneapolis, Minnesota, United States, 55455 United States, New York Mount Sinai School of Medicine New York, New York, United States, 10029 United States, Ohio University Hospitals Case Medical Center Cleveland, Ohio, United States, 44106 United States, Oregon Oregon Health & Science University Portland, Oregon, United States, 97239 United States, Texas Baylor College of Medicine Houston, Texas, United States, 77030 收起 <<
NCT01948427	-		Recruiting	September 2026	United States, Pennsylvania ... 展开 >> United BioSource Corporation Recruiting Blue Bell, Pennsylvania, United States, 19422 Contact: THRIVE Registry Team 855-823-2595 thriveregistry@unitedbiosource.com 收起 <<
NCT03488524	Amyotrophic Lateral Sclerosis ... 展开 >> ALS 收起 <<	Phase 2	Recruiting	January 1, 2020	United States, Massachusetts ... 展开 >> Massachusetts General Hospital Recruiting Boston, Massachusetts, United States, 02114 Contact: Aileen Shaughnessy, MA 617-724-9196 ANSHAUGHNESSY@mgh.harvard.edu Principal Investigator: James Berry, MD University of Massachusetts Memorial Medical Center Recruiting Worcester, Massachusetts, United States, 01655 Contact: Diane McKenna-Yasek, RN, BSN 508-856-4697 diane.mckenna-yasek@umassmed.edu Principal Investigator: Margaret Owegi, DO 收起 <<
NCT00067756	Alpha 1-Antitrypsin Deficiency	Phase 2	Completed	-	United States, Florida ... 展开 >> University of Florida Gainesville, Florida, United States, 32610 收起 <<
NCT02094222	-		-	-	-
NCT00947297	-		Completed	-	-
NCT00771901	-		Completed	-	-
NCT01347073	-		Completed	-	-
NCT01257737	Urea Cycle Disorders	Phase 4	Completed	-	United States, California ... 展开 >> UCLA Pediatrics/Genetics Los Angeles, California, United States, 90095 Stanford University School of Medicine Palo Alto, California, United States, 94305 United States, Colorado Denver Children's Hospital Aurora, Colorado, United States, 80045 United States, District of Columbia Children's National Medical Center Washington, District of Columbia, United States, 20010 United States, Maine Maine Medical Center Portland, Maine, United States, 04102 United States, Minnesota University of Minnesota Medical Center Minneapolis, Minnesota, United States, 55454 United States, New York Mount Sinai School of Medicine New York, New York, United States, 10029 United States, Ohio University Hospitals Case Medical Center Cleveland, Ohio, United States, 44106 Nationwide Children's Hospital Columbus, Ohio, United States, 43205 United States, Oregon Oregon Health & Science University Portland, Oregon, United States, 97239 United States, Pennsylvania Children's Hospital of Pittsburg of UPMC Pittsburgh, Pennsylvania, United States, 15224 United States, Texas Baylor College of Medicine Houston, Texas, United States, 77030 United States, Utah University of Utah Salt Lake City, Utah, United States, 84112 United States, Washington Seattle Children's Hospital Seattle, Washington, United States, 98105 United States, Wisconsin Children's Hospital of Wisconsin Milwaukee, Wisconsin, United States, 53226 Canada, Ontario The Hospital for Sick Children Toronto, Ontario, Canada, M5G 1X8 收起 <<
NCT00771901	Insulin Resistance ... 展开 >> Diabetes Obesity 收起 <<	Not Applicable	Completed	-	United States, Missouri ... 展开 >> Washington University School of Medicine Saint Louis, Missouri, United States, 63110 收起 <<
NCT00004767	Amino Acid Metabolism, Inborn ... 展开 >>Errors 收起 <<	Phase 2	Completed	-	-
NCT00947297	Urea Cycle Disorders	Phase 3	Completed	-	-
NCT03533257	Alzheimer Disease	Phase 2	Recruiting	September 1, 2020	United States, Illinois ... 展开 >> Rush University Medical Center Recruiting Chicago, Illinois, United States, 60612 Contact: Judy Phillips 312-942-0050 Judy_Phillips@rush.edu United States, Kansas University of Kansas Clinical Research Center Recruiting Fairway, Kansas, United States, 66205 Contact: KUMC Recruitment Division 913-588-0555 KUAMP@kumc.edu United States, Massachusetts Massachusetts General Hospital Recruiting Boston, Massachusetts, United States, 02114 Contact: Alison J McManus, DNP 617-643-4848 ajmcmanus@mgh.harvard.edu Contact: John Ernandez 617-643-4047 jernandez@partners.org United States, New Jersey Rowan University Not yet recruiting Stratford, New Jersey, United States, 08084 Contact: Lauren Fedor 856-566-6003 fedor@rowan.edu United States, New York Mount Sinai Alzheimer's Disease Research Center Not yet recruiting New York, New York, United States, 10029 Contact: Caroline Meuser 212-659-8885 caroline.meuser@mssm.edu Columbia University Recruiting New York, New York, United States, 10032 Contact: Ruth Tejeda 212-305-7661 rbt41@cumc.columbia.edu United States, Pennsylvania Penn Memory Center Recruiting Philadelphia, Pennsylvania, United States, 19104 Contact: Laura Schankel 215-349-8727 laura.schankel@pennmedicine.upenn.edu 收起 <<
NCT00006239	Leukemia Myel... 展开 >>odysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases 收起 <<	Phase 1	Completed	-	United States, Maryland ... 展开 >> Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Baltimore, Maryland, United States, 21231-2410 收起 <<
NCT01257737	-		Completed	-	-
NCT00986895	Hepatic Encephalopathy ... 展开 >> Urea Cycle Disorders 收起 <<	Phase 1	Completed	-	Ukraine ... 展开 >> National University of Pharmacy Kharkiv, Ukraine, 61057 Department of General Surgery #2; Kharkiv State Medical University Kharkiv, Ukraine, 61128 收起 <<
NCT00387530	Gastric Cancer ... 展开 >> Head and Neck Cancer Lymphoma Lymphoproliferative Disorder 收起 <<	Phase 2	Unknown	-	United States, California ... 展开 >> Rebecca and John Moores UCSD Cancer Center La Jolla, California, United States, 92093-0658 收起 <<
NCT00005934	Beta Thalassemia	Phase 2	Completed	-	United States, Maryland ... 展开 >> National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Bethesda, Maryland, United States, 20892 收起 <<
NCT03127514	Amyotrophic Lateral Sclerosis ... 展开 >> Motor Neuron Disease Neuromuscular Diseases Neurodegenerative Diseases Spinal Cord Diseases TDP-43 Proteinopathies Nervous System Diseases Central Nervous System Diseases 收起 <<	Phase 2	Recruiting	May 2019	-
NCT02343094	Proteinuric Diseases	Phase 2 Phase 3	Completed	-	France ... 展开 >> Hôpital Necker Enfants Malades Paris, France, 75015 收起 <<
NCT02246218	Urea Cycle Disorder	Phase 4	Completed	-	United States, California ... 展开 >> Kaiser Permanente Regional Metabolic Center Los Angeles, California, United States, 90027 Stanford Center for Clinical & Translational Research & Education Palo Alto, California, United States, 94034 United States, District of Columbia Children's National Medical Center Washington, D.C., District of Columbia, United States, 20010 United States, Florida Shands at University of Florida Gainesville, Florida, United States, 32610 United States, Georgia Emory University Decatur, Georgia, United States, 30033 United States, Indiana Indiana University Indianapolis, Indiana, United States, 46202 United States, Maine Maine Medical Center Portland, Maine, United States, 04102 United States, Michigan Children's Hospital of Michigan Detroit, Michigan, United States, 48201 United States, Minnesota University of Minnesota Minneapolis, Minnesota, United States, 55455 United States, New York Mount Sinai School of Medicine New York, New York, United States, 10029 United States, Ohio University Hospitals Case Medical Center Cleveland, Ohio, United States, 44106 United States, Oregon Oregon Health & Science University Portland, Oregon, United States, 97239 United States, Pennsylvania Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States, 15224 United States, Utah University of Utah Salt Lake City, Utah, United States, 84132 United States, Washington Seattle Children's Hospital Seattle, Washington, United States, 98105 Canada The Hospital for Sick Children Toronto, Canada, M5G 1Hs 收起 <<
NCT00006240	Leukemia	Phase 2	Completed	-	United States, Maryland ... 展开 >> Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support Bethesda, Maryland, United States, 20892-1182 National Heart, Lung, and Blood Institute Bethesda, Maryland, United States, 20892 United States, New York Mount Sinai Medical Center, NY New York, New York, United States, 10029 United States, Pennsylvania University of Pittsburgh Cancer Institute Pittsburgh, Pennsylvania, United States, 15213-3489 收起 <<
NCT00005639	Lymphoma Smal... 展开 >>l Intestine Cancer Unspecified Adult Solid Tumor, Protocol Specific 收起 <<	Phase 1	Completed	-	United States, Maryland ... 展开 >> Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Baltimore, Maryland, United States, 21231 收起 <<
NCT01881984	Medium-chain Acyl-CoA Dehydrog... 展开 >>enase (MCAD) Deficiency 收起 <<	Phase 1	Completed	-	United States, Pennsylvania ... 展开 >> Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States, 15224 收起 <<
NCT00006019	Leukemia Lung... 展开 >> Cancer Lymphoma Multiple Myeloma and Plasma Cell Neoplasm Myelodysplastic Syndromes Prostate Cancer 收起 <<	Phase 2	Completed	-	United States, New York ... 展开 >> Memorial Sloan-Kettering Cancer Center New York, New York, United States, 10021 收起 <<
NCT02323100	Cystic Fibrosis	Phase 1 Phase 2	Recruiting	April 2020	United States, Alabama ... 展开 >> University of Alabama at Birmingham Not yet recruiting Birmingham, Alabama, United States, 35294-0006 Contact: Steven M Rowe, MD, MSPH 205-934-9640 smrowe@uab.edu Principal Investigator: Steven M Rowe, MD, MSPH United States, Colorado National Jewish Health Recruiting Denver, Colorado, United States, 80206 Contact: Connie StClair 303-270-2321 shiven@njhealth.org Principal Investigator: Noah Lechtzin, MD Sub-Investigator: Peter Mogazel, MD, PhD United States, Pennsylvania Children's Hospital of Philadelphia Not yet recruiting Philadelphia, Pennsylvania, United States, 19104 Contact: Ron Rubenstein, MD, PhD 215-590-1281 rrubenst@mail.med.upenn.edu Principal Investigator: Ron Rubenstein, MD, PhD 收起 <<
NCT00016744	Cystic Fibrosis	Phase 1 Phase 2	Completed	-	-
NCT01881984	-		Completed	-	-
NCT00006450	Brain Tumor	Phase 2	Completed	-	United States, Maryland ... 展开 >> National Cancer Institute (NCI) Bethesda, Maryland, United States, 20892 收起 <<
NCT00590538	-		Terminated(12/15/2008 Voluntar... 展开 >>ily placed on inactive status-requested by the PI) 收起 <<	-	-
NCT00590538	Cystic Fibrosis	Phase 1 Phase 2	Terminated(12/15/2008 Voluntar... 展开 >>ily placed on inactive status-requested by the PI) 收起 <<	-	United States, Pennsylvania ... 展开 >> The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 收起 <<
NCT00992459	Urea Cycle Disorders	Phase 3	Completed	-	-
NCT00977600	Healthy	Phase 1	Completed	-	Ukraine ... 展开 >> Medical Sanitary Division #2 Kharkiv, Ukraine, 61011 收起 <<
NCT00439569	Spinal Muscular Atrophy Type I... 展开 >>I Spinal Muscular Atrophy Type III 收起 <<	Phase 1 Phase 2	Terminated(Due to poor complia... 展开 >>nce with study drug administration.) 收起 <<	-	United States, California ... 展开 >> Stanford University Medical Center, 300 Pasteur Drive, Room A343 Stanford, California, United States, 94305-5235 United States, Missouri Washington University Medical School, Washington University, 660 S. Euclid Avenue, Box 8111 St. Louis, Missouri, United States, 63110 United States, New York Columbia University, 180 Fort Washington Avenue, 5th Floor New York, New York, United States, 10032 United States, Pennsylvania The Children's Hospital of Philadelphia, Clinical Trials Office, A-230, 34th St. and Civic Center Boulevard Philadelphia, Pennsylvania, United States, 19104-4399 United States, Texas University of Texas Southwestern Medical Center at Dallas, Division of Pediatric Neurology, Children's Medical Center of Dallas, Ambulatory Care Pavilion, 2350 Stemmons Freeway, Suite #5074 Dallas, Texas, United States, 75207 收起 <<
NCT00001565	Brain Neoplasms ... 展开 >> Neuroblastoma 收起 <<	Phase 1	Completed	-	United States, Maryland ... 展开 >> National Cancer Institute (NCI) Bethesda, Maryland, United States, 20892 收起 <<
NCT00004871	Leukemia Myel... 展开 >>odysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases 收起 <<	Phase 1	Completed	-	United States, Maryland ... 展开 >> Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Baltimore, Maryland, United States, 21231-2410 收起 <<
NCT00439569	-		Terminated(Due to poor complia... 展开 >>nce with study drug administration.) 收起 <<	-	-
NCT00992459	-		Completed	-	-
NCT00002909	Lymphoma Unsp... 展开 >>ecified Adult Solid Tumor, Protocol Specific 收起 <<	Phase 1	Completed	-	United States, Maryland ... 展开 >> Johns Hopkins Oncology Center Baltimore, Maryland, United States, 21287 收起 <<
NCT01949766	-		-	-	-
NCT00002796	Mucinous Adenocarcinoma of the... 展开 >> Colon Mucinous Adenocarcinoma of the Rectum Recurrent Colon Cancer Recurrent Rectal Cancer Signet Ring Adenocarcinoma of the Colon Signet Ring Adenocarcinoma of the Rectum Stage IVA Colon Cancer Stage IVA Rectal Cancer Stage IVB Colon Cancer Stage IVB Rectal Cancer 收起 <<	Phase 1 Phase 2	Terminated(Administratively co... 展开 >>mplete.) 收起 <<	-	United States, New York ... 展开 >> Mount Sinai Medical Center New York, New York, United States, 10029 收起 <<
NCT00439218	Spinal Muscular Atrophy Type I	Phase 1 Phase 2	Terminated(Extremely slow enro... 展开 >>llment) 收起 <<	-	United States, California ... 展开 >> Stanford University Medical Center, 300 Pasteur Drive, Room A343 Stanford, California, United States, 94305-5235 United States, Massachusetts Children's Hospital, Boston, 300 Longwood Avenue, Fegan 11 Boston, Massachusetts, United States, 02115 United States, New York Columbia University, 180 Fort Washington Avenue, 5th Floor New York, New York, United States, 10032 United States, Pennsylvania The Children's Hospital of Philadelphia, Clinical Trials Office, A-230, 34th St. and Civic Center Boulevard Philadelphia, Pennsylvania, United States, 19104-4399 United States, Texas University of Texas Southwestern Medical Center at Dallas, Division of Pediatric Neurology, Children's Medical Center of Dallas, Ambulatory Care Pavilion, 2350 Stemmons Freeway, Suite #5074 Dallas, Texas, United States, 75207 收起 <<
NCT03335488	Urea Cycle Disorder	Phase 4	Recruiting	March 31, 2020	United States, New York ... 展开 >> Mount Sinai School of Medicine Recruiting New York, New York, United States, 10029 Contact: Dr. George Diaz, M.D., PhD. 212-241-0858 George.Diaz@mssm.edu Contact: Luca Fierro 212 659 1477 luca.fierro@mssm.edu Principal Investigator: Dr. George Diaz, M.D., PhD. United States, Ohio University Hospitals Case Medical Center Recruiting Cleveland, Ohio, United States, 44106-6005 Contact: Dr. Jirair Bedoyan, MD 216-844-7124 Jirair.Bedoyan@UHhospitals.org Contact: Audrey Lynn 216 844 7124 audrey.lynn@UHhospitals.org United States, Pennsylvania Children's Hospital of Pittsburgh of UPMC Recruiting Pittsburgh, Pennsylvania, United States, 15224 Contact: Dr. Gerard Vockley, MD, PhD 412-692-7530 Jennifer.baker@chp.edu Contact: Jennifer Baker 412-692-7530 Jennifer.baker@chp.edu Principal Investigator: Dr. Gerard Vockley, MD, PhD United States, Utah University of Utah Recruiting Salt Lake City, Utah, United States, 84132 Contact: Dr. Nicola Longo, M.D., PhD. 801-587-3605 Nicola.Longo@hsc.utah.edu Contact: Carrie Bailey 801 587 3605 Carrie.Bailey@hsc.utah.edu Principal Investigator: Dr. Nicola Longo, M.D., PhD. Belgium Cliniques Universitaires Saint-Luc Recruiting Brussels, Belgium, 1200 Contact: Prof. Etienne Sokal, MD 32 2 764 13 87 etienne.sokal@uclouvain.be Contact: Julia Versavau 32 2 764 19 33 julia.versavau@uclouvain.be Principal Investigator: Prof. Etienne Sokal, MD Spain Hospital Materno-Infantil (HRU Carlos Haya) Recruiting Málaga, Andalucia, Spain, 29006 Contact: Dr. Javier Blasco-Alonso, MD 34 951 29 21 91 javierblascoalonso@yahoo.es Contact: Almudena Frias 34 951 29 21 91 Principal Investigator: Dr. Javier Blasco-Alonso Hospital Clinico Universitario de Santiago Recruiting Santiago de Compostela, Galicia, Spain, 15706 Contact: Dr. Maria Luz Couce-Pico, MD 34 981 950 162 maria.luz.couce.pico@sergas.es Contact: Maria Teresa Oreiro-Garcia 34 981 955 627 maria.teresa.oreiro.garcia@sergas.es Principal Investigator: Dr. Maria Luz Couce-Pico, MD Hospital Universitario de Cruces Recruiting Barakaldo, Vizcaya, Spain, 48903 Contact: Dr. Luis Jose Aldamiz-Echevar Azuara, MD 34 94 600 6327 LUISJOSE.ALDAMIZ-ECHEVARAZUARA@osakidetza.net Contact: Fernando Olatz 34 94 600 6327 fernando.andradelodeiro@osakidetza.eus Principal Investigator: Dr. Luis Jose Aldamiz-Echevar Azuara, MD Switzerland Universitätsspital, Inselspital Bern Recruiting Bern, Switzerland, 3010 Contact: Matthias Gautchshi,, MD 41 (0)31 632 01 47 Matthias.Gautschi@insel.ch Contact: Alexandra Nuoffer +41 (0)31 632 51 37 alexandra.nuoffer@insel.ch Principal Investigator: Dr. Matthias Gautchshi, MD University Children's Hospital Recruiting Zurich, Switzerland, 75, 8032 Contact: Prof Johannes Häberle, MD 41- 44-266-7111 johannes.haeberle@kispi.uzh.ch Contact: Ms. Doris Anzengruber 41- 44-266-7722 Doris.Anzengruber@kispi.uzh.ch Principal Investigator: Prof Johannes Häberle, MD 收起 <<
NCT00528268	Spinal Muscular Atrophy	Phase 1 Phase 2	Completed	-	United States, Utah ... 展开 >> University of Utah Salt Lake City, Utah, United States, 84132 收起 <<
NCT00439218	-		Terminated(Extremely slow enro... 展开 >>llment) 收起 <<	-	-
NCT01784718	-		-	-	-
NCT00528268	-		Completed	-	-
NCT01702974	HIV Infection	Phase 2	Completed	-	Ethiopia ... 展开 >> Black Lion Hospital (BLH), Addis Ababa University, Faculty of Medicine Addis Ababa, Lideta sub city, Ethiopia 收起 <<
NCT00107770	Amyotrophic Lateral Sclerosis	Phase 1 Phase 2	Completed	-	United States, Iowa ... 展开 >> VA Medical Center, Iowa City Iowa City, Iowa, United States, 52246-2208 United States, Kentucky VA Medical Center, Lexington Lexington, Kentucky, United States, 40502 United States, Maryland VA Maryland Health Care System, Baltimore Baltimore, Maryland, United States, 21201 United States, Massachusetts Edith Nourse Rogers Memorial Veterans Hospital, Bedford Bedford, Massachusetts, United States, 01730 VA Medical Center, Jamaica Plain Campus Boston, Massachusetts, United States, 02130 United States, New York VA Medical Center, Syracuse Syracuse, New York, United States, 13210 United States, North Carolina VA Medical Center, Durham Durham, North Carolina, United States, 27705 United States, Ohio VA Medical Center, Cincinnati Cincinnati, Ohio, United States, 45220 United States, Pennsylvania VA Pittsburgh Health Care System Pittsburgh, Pennsylvania, United States, 15240 United States, Texas Michael E. DeBakey VA Medical Center (152) Houston, Texas, United States, 77030 收起 <<
NCT00533559	Diabetes Insu... 展开 >>lin Resistance 收起 <<	Phase 4	Completed	-	Canada, Ontario ... 展开 >> Toronto General Hospital Toronto, Ontario, Canada, M5G 2C4 收起 <<
NCT01698476	Pulmonary Tuberculosis (TB)	Phase 2	Completed	-	Ethiopia ... 展开 >> Black Lion Hospital (BLH), Addis Ababa University, Faculty of Medicine Addis Ababa, Lideta sub city, Ethiopia 收起 <<
NCT00947544	Urea Cycle Disorders	Phase 2	Completed	-	United States, California ... 展开 >> UCLA Los Angeles, California, United States, 90095 United States, District of Columbia The George Washington DC Children's National Medical Center Washington, District of Columbia, United States, 20010 United States, New York Mount Sinai School of Medicine New York, New York, United States, 10029 United States, Pennsylvania University of Pittsburgh Pittsburgh, Pennsylvania, United States, 15201 United States, Texas Baylor College of Medicine Houston, Texas, United States, 77030 United States, Washington Seattle Children's Hospital Seattle, Washington, United States, 98105 United States, Wisconsin Medical College of Wisconsin Milwaukee, Wisconsin, United States, 53226 Canada, Ontario The Hospital for Sick Children Toronto, Ontario, Canada, M5G1X8 收起 <<
NCT01580007	Pulmonary Tuberculosis	Phase 2	Completed	-	Bangladesh ... 展开 >> National Institute of Diseases of Chest and Hospital (NIDCH) Dhaka, Bangladesh, 1212 收起 <<
NCT00947544	-		Completed	-	-
NCT00345605	Argininosuccinic Aciduria ... 展开 >> Amino Acid Metabolism, Inborn Errors Urea Cycle Disorders 收起 <<	Phase 2	Completed	-	United States, Texas ... 展开 >> Baylor College of Medicine Houston, Texas, United States, 77030 收起 <<
NCT00345605	-		Completed	-	-
NCT00551200	Urea Cycle Disorders	Phase 2	Completed	-	United States, Minnesota ... 展开 >> University of Minnesota Minneapolis, Minnesota, United States, 55455 United States, Texas Baylor College of Medicine Houston, Texas, United States, 77030 收起 <<
NCT03734263	Pyruvate Dehydrogenase Complex... 展开 >> Deficiency 收起 <<	Phase 1 Phase 2	Recruiting	October 1, 2019	Italy ... 展开 >> Federico II University Recruiting Napoli, Italy, 80131 Contact: Nicola Brunetti-Pierri +390817463288 brunetti@tigem.it 收起 <<
NCT01096095	Spinocerebellar Ataxia Type 3	Phase 2	Withdrawn(Regulatory authoriti... 展开 >>es did not allow the entrance of the study drug in the country) 收起 <<	July 2011	Brazil ... 展开 >> Hospital de Clínicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil, 90.035-903 收起 <<
NCT02111200	Urea Cycle Disorders, Inborn	Not Applicable	Completed	-	United States, Texas ... 展开 >> Children's Nutrition Research Center/Baylor College of Medicine Houston, Texas, United States, 77030 收起 <<
NCT01529060	Maple Syrup Urine Disease	Phase 2 Phase 3	Active, not recruiting	December 2017	United States, Texas ... 展开 >> Baylor College of Medicine Houston, Texas, United States, 77030 收起 <<
NCT02111200	-		Completed	-	-
NCT00551200	-		Completed	-	-
NCT00212316	Huntington's Disease	Phase 2	Completed	-	United States, Alabama ... 展开 >> University of Alabama Birmingham, Alabama, United States United States, California University of California—San Diego San Diego, California, United States United States, Iowa University of Iowa Hospital and Clinics Iowa City, Iowa, United States United States, Kansas University of Kansas Medical Center Kansas City, Kansas, United States United States, Maryland Johns Hopkins University Baltimore, Maryland, United States United States, Massachusetts Massachusetts General Hospital Boston, Massachusetts, United States United States, New York Columbia University New York, New York, United States University of Rochester Rochester, New York, United States 收起 <<
NCT02046434	Parkinson's Disease	Phase 1	Active, not recruiting	September 2018	United States, Colorado ... 展开 >> University of Colorado Denver Anschutz Medical Center Aurora, Colorado, United States, 80045 收起 <<

A498 cells	4 mM	Function assay	8-48 h	Induction of CAMP mRNA expression in human A498 cells at 4 mM after 8 to 48 hrs by RT-PCR analysis	19770273
Hela cells		Function assay		Inhibition of HDAC in human Hela cells nuclear extracts by fluorimetric assay	19520580
HT-29 cells	4 mM	Function assay	8-48 h	Induction of CAMP mRNA expression in human HT-29 cells at 4 mM after 8 to 48 hrs by RT-PCR analysis	19770273
U937 cells	4 mM	Function assay	8-48 h	Induction of CAMP mRNA expression in human U937 cells at 4 mM after 8 to 48 hrs by RT-PCR analysis	19770273
VA10 cells		Function assay		Induction of LL-37 protein expression in human VA10 cells at 20 nM after 24 hrs by Western blotting	19770273

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