4-PBA (Sodium Phenylbutyrate) Clinical Trials

Size	Price	Qty
50mg	USD 147
1g	USD 470

Clinical Trials

Numerous clinical trials evaluate 4-PBA (sodium phenylbutyrate) across Early Phase 1 through Phase 4 for conditions such as urea cycle disorders, alpha 1-antitrypsin deficiency, cystic fibrosis, corticobasal syndrome, and inclusion body myositis. Sponsored by academic and industry entities including the Technical University of Munich, Children's Hospital of Philadelphia, and Horizon Therapeutics LLC, recruitment statuses range from actively recruiting to completed and approved for marketing. Notable investigations include a Phase 1 safety trial in sporadic inclusion body myositis, an ongoing Phase 2 trial for corticobasal syndrome, and a Phase 4 study in pediatric urea cycle disorder.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT06069375	SUSPENDED	Medium-chain Acyl-CoA Dehydrogenase Deficiency	Jerry Vockley, MD, PhD	2024-04-01	PHASE2
NCT06645769	NOT_YET_RECRUITING	Diabetes	University of Pennsylvania	2027-06-01	EARLY_PHASE1
NCT07125066	ENROLLING_BY_INVITATION	Combined D,L-2-hydroxyglutaric Aciduria	Jerry Vockley, MD, PhD	2025-07-30	PHASE1
NCT06773026	RECRUITING	Medium-chain Acyl-CoA Dehydrogenase Deficiency	Jerry Vockley, MD, PhD	2025-06-30	PHASE2
NCT05983588	Recruiting	Corticobasal Syndrome (CBS)	Technical University of Munich	2023-12-12	Phase 2
NCT05028803	COMPLETED	Obese Non Diabetic	University of Nottingham	2021-09-01
NCT03335488	COMPLETED	Urea Cycle Disorder	Amgen	2018-02-20	PHASE4
NCT05349435	TERMINATED	Hyperammonemia	Liminal BioSciences Ltd.	2022-05-13	PHASE1
NCT05019417	Unknown status	Monocarboxylate Transporter 8 Deficiency	Kaplan Medical Center\|Weizmann Institute of Science	2021-06-30	Phase 2\|Phase 3
NCT04937062	Enrolling by invitation	STXBP1 Encephalopathy With Epilepsy SLC6A1 Neurodevelopmental Disorder\|Developmental and Epileptic Encephalopathy	Weill Medical College of Cornell University\|Children''s Hospital Colorado\|SLC6A1 Connect\|STXBP1 Foundation\|Clara Inspired\|University of Pennsylvania Orphan Disease Center\|Horizon Therapeutics	2021-03-01	Early Phase 1
NCT03533257	COMPLETED	Alzheimer Disease	Amylyx Pharmaceuticals Inc.	2018-09-14	PHASE2
NCT04421677	Completed	Inclusion Body Myositis\|Sporadic Inclusion Body Myositis	University of Kansas Medical Center	2020-08-20	Phase 1
NCT03734263	COMPLETED	Pyruvate Dehydrogenase Complex Deficiency	Fondazione Telethon	2018-10-01	PHASE2
NCT02111200	COMPLETED	Urea Cycle Disorders, Inborn	Baylor College of Medicine	2014-09
NCT01702974	COMPLETED	HIV Infection	Karolinska Institutet	2012-09	PHASE2
NCT01698476	COMPLETED	Pulmonary Tuberculosis (TB)	Karolinska Institutet	2012-09	PHASE2
NCT00771901	COMPLETED	Insulin Resistance; Diabetes; Obesity	Washington University School of Medicine	2008-02
NCT02246218	Completed	Urea Cycle Disorder	Horizon Therapeutics LLC\|Horizon Pharma Ireland Ltd. Dublin Ireland	2014-12-31	Phase 4
NCT01580007	COMPLETED	Pulmonary Tuberculosis	International Centre for Diarrhoeal Disease Research, Bangladesh	2010-12	PHASE2
NCT00528268	COMPLETED	Spinal Muscular Atrophy	University of Utah	2007-07	PHASE1; PHASE2
NCT00947544	COMPLETED	Urea Cycle Disorders	Amgen	2010-03	PHASE2
NCT00345605	COMPLETED	Argininosuccinic Aciduria; Amino Acid Metabolism, Inborn Errors; Urea Cycle Disorders	Brendan Lee	2008-02	PHASE2
NCT01096095	WITHDRAWN	Spinocerebellar Ataxia Type 3	Hospital de Clinicas de Porto Alegre	2010-06	PHASE2
NCT01096095	Withdrawn	Spinocerebellar Ataxia Type 3	Hospital de Clinicas de Porto Alegre\|Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul Brazil	2010-06	Phase 2
NCT00533559	COMPLETED	Diabetes; Insulin Resistance	University Health Network, Toronto	2007-09	PHASE4
NCT00439218	TERMINATED	Spinal Muscular Atrophy Type I	Westat	2008-01	PHASE1; PHASE2
NCT00439569	TERMINATED	Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type III	Westat	2008-01	PHASE1; PHASE2
NCT00551200	COMPLETED	Urea Cycle Disorders	Amgen	2007-10	PHASE2
NCT00387530	WITHDRAWN	Gastric Cancer; Head and Neck Cancer; Lymphoma; Lymphoproliferative Disorder	University of California, San Diego	2006-05	PHASE2
NCT00107770	COMPLETED	Amyotrophic Lateral Sclerosis	US Department of Veterans Affairs	2005-04	PHASE1; PHASE2
NCT00016744	COMPLETED	Cystic Fibrosis	Children's Hospital of Philadelphia	2001-09	PHASE1; PHASE2
NCT00005639	COMPLETED	Lymphoma; Small Intestine Cancer; Unspecified Adult Solid Tumor, Protocol Specific	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	2000-03	PHASE1
NCT00006239	COMPLETED	Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	2000-12	PHASE1
NCT00002796	TERMINATED	Mucinous Adenocarcinoma of the 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	National Cancer Institute (NCI)	1997-05	PHASE1; PHASE2
NCT00067756	COMPLETED	Alpha 1-Antitrypsin Deficiency	University of Florida	2001-11	PHASE2
NCT00002909	COMPLETED	Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific	National Cancer Institute (NCI)	1997-02	PHASE1
NCT00006240	COMPLETED	Leukemia	National Heart, Lung, and Blood Institute (NHLBI)	2000-10	PHASE2
NCT00004767	COMPLETED	Amino Acid Metabolism, Inborn Errors	National Center for Research Resources (NCRR)	1985-01	PHASE2
NCT00005934	COMPLETED	Beta Thalassemia	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	2000-06	PHASE2
NCT00004871	COMPLETED	Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	2000-05	PHASE1
NCT00212316	COMPLETED	Huntington's Disease	University of Rochester	2005-08	PHASE2
NCT00006019	COMPLETED	Leukemia; Lung Cancer; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes; Prostate Cancer	Memorial Sloan Kettering Cancer Center	2000-05	PHASE2

(data from https://clinicaltrials.gov, updated on 2026-07-06)

Check the 4-PBA (Sodium Phenylbutyrate) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

4-PBA acts as a histone deacetylase inhibitor and molecular chaperone, inhibiting HDAC enzymes to promote histone hyperacetylation and prevent endoplasmic reticulum stress driven by protein misfolding. By restoring intracellular protein homeostasis and serving as an alternative pathway for nitrogen excretion, 4-PBA mitigates systemic toxicity and cellular damage associated with clinical conditions such as urea cycle disorders and protein-misfolding encephalopathies.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).