- Conditions
- Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
- Interventions
- Ravicti
- Drug
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
Search Results
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Showing 1–24
of 51
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- US Department of Veterans Affairs
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 10
- States / cities
- Iowa City, Iowa • Lexington, Kentucky • Baltimore, Maryland + 7 more
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Interventions
- azacitidine, sodium phenylbutyrate
- Drug
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2000
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Beta Thalassemia
- Interventions
- 5-Azacytidine
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- Not listed
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 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
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Diseases, Neurodegenerative Diseases, Spinal Cord Diseases, TDP-43 Proteinopathies, Nervous System Diseases, Central Nervous System Diseases
- Interventions
- AMX0035, Placebo
- Drug · Other
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 137 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 25
- States / cities
- Phoenix, Arizona • Orange, California • San Francisco, California + 21 more
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, BUPHENYL®
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Houston, Texas
- Conditions
- Drug Toxicity
- Interventions
- HPN-100, HPN-100 or Placebo, Placebo, Moxifloxacin
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 98 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Spinal Muscular Atrophy Type I
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- Westat
- Other
- Eligibility
- 2 Months to 48 Months
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 5
- States / cities
- Stanford, California • Boston, Massachusetts • New York, New York + 2 more
- Conditions
- Combined D,L-2-hydroxyglutaric Aciduria
- Interventions
- Sodium phenylbutyrate
- Drug
- Lead sponsor
- Jerry Vockley, MD, PhD
- Other
- Eligibility
- Male only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
- Interventions
- Sodium Phenylbutyrate, Arginine
- Drug
- Lead sponsor
- Brendan Lee
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 1
- States / cities
- Houston, Texas
- 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
- Amyotrophic Lateral Sclerosis
- Interventions
- Placebo, AMX0035
- Other · Drug
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 664 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 28
- States / cities
- Phoenix, Arizona • Los Angeles, California • Orange, California + 23 more
- Conditions
- Maple Syrup Urine Disease
- Interventions
- Phenylbutyrate, Placebo powder
- Drug
- Lead sponsor
- Brendan Lee
- Other
- Eligibility
- 3 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Urea Cycle Disorder
- Interventions
- RAVICTI, NaPBA
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- Up to 99 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 6
- States / cities
- Gainesville, Florida • New York, New York • Cleveland, Ohio + 3 more
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Sodium phenylbutyrate
- Drug
- Lead sponsor
- University of Utah
- Other
- Eligibility
- 1 Day to 6 Months
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- 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, ALS
- Interventions
- AMX0035
- Drug
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 24
- States / cities
- Phoenix, Arizona • Orange, California • San Francisco, California + 20 more
- Conditions
- Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- Westat
- Other
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008
- U.S. locations
- 5
- States / cities
- Stanford, California • St Louis, Missouri • New York, New York + 2 more
- 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
- Conditions
- STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder, Developmental and Epileptic Encephalopathy
- Interventions
- Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]
- Drug
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 0 Months to 17 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • New York, New York
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 21
- States / cities
- Long Beach, California • Los Angeles, California • Stanford, California + 18 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