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Showing 25–40 of 40 matching trials from the live ClinicalTrials.gov search.
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
4 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Leukemia
Interventions
sargramostim, dexamethasone, oral sodium phenylbutyrate
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
4
States / cities
Bethesda, Maryland • New York, New York • Pittsburgh, Pennsylvania
Conditions
Gastric Cancer, Head and Neck Cancer, Lymphoma, Lymphoproliferative Disorder
Interventions
oral sodium phenylbutyrate, valganciclovir, polymerase chain reaction, protein expression analysis, biopsy
Drug · Genetic · Procedure
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
La Jolla, California
Conditions
Huntington's Disease
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
8
States / cities
Birmingham, Alabama • San Diego, California • Iowa City, Iowa + 5 more
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
sodium phenylbutyrate, tretinoin
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Cystic Fibrosis
Interventions
Sodium 4-Phenylbutyrate, Genistein (Unconjugated Isoflavones 100), Placebo
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Conditions
ALS
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Type 1 Diabetes (T1D), Metabolic Diseases, Glucose Metabolism Disorders, Endocrine System Diseases, Autoimmune Diseases, Immune System Diseases, Diabetes Melletus, Type 1, Nutritional and Metabolic Diseases, Combination Therapy
Interventions
AMX0035, Placebo
Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 69 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
29 Days to 6 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
San Francisco, California • Boston, Massachusetts • Worcester, Massachusetts + 2 more
Conditions
Urea Cycle Disorders
Interventions
HPN-100, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
7
States / cities
Los Angeles, California • Washington D.C., District of Columbia • New York, New York + 4 more
Conditions
Progressive Supranuclear Palsy, PSP, Neurodegenerative Diseases, Atypical Parkinsonism
Interventions
AMX0035, Placebo
Drug · Other
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
40 Years to 80 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
35
States / cities
Scottsdale, Arizona • Little Rock, Arkansas • Fountain Valley, California + 31 more
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
10 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Alzheimer Disease
Interventions
AMX0035, Placebo
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
55 Years to 89 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
11
States / cities
Jacksonville, Florida • Orlando, Florida • Palmetto Bay, Florida + 7 more
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
U.S. locations
20
States / cities
Los Angeles, California • Oakland, California • Fort Lauderdale, Florida + 14 more