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ClinicalTrials.gov public records Last synced Oct 23, 2026, 9:25 AM EDT

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Showing 1–24 of 101 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Dehydrogenase Deficiency Clear all
Suspended Phase 2 Interventional

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

NCT06069375
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional

A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS

NCT00002257
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
12
States / cities
Berkeley, California • San Francisco, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 3 Interventional

Preventing Cytomegalovirus (CMV) Organ Damage With Valganciclovir in People With HIV

NCT00006145
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
56
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Smith-Lemli-Opitz Syndrome and Cholic Acid

NCT03720990
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholic Acid
Drug
Lead sponsor
University of Nebraska
Other
Eligibility
2 Years to 25 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Aurora, Colorado • Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated Apr 30, 2024 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

NCT01894165
View directory record Official record
Conditions
Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Interventions
ALXN1101, Placebo
Drug
Lead sponsor
Origin Biosciences
Industry
Eligibility
18 Years to 60 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 19, 2019 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional

The Safety and Effectiveness of Ganciclovir Used Alone or in Combination With Granulocyte-Macrophage Colony Stimulating Factor in the Treatment of Cytomegalovirus (CMV) of the Eye in Patients With AIDS

NCT00000989
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Sargramostim, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
4
States / cities
Los Angeles, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 23, 2026, 9:25 AM EDT
Terminated Phase 2 Interventional Results available

A Phase II Trial of the DNA Methyl Transferase Inhibitor, Guadecitabine (SGI-110), in Children and Adults With Wild Type GIST,Pheochromocytoma and Paraganglioma Associated With Succinate Dehydrogenase Deficiency and HLRCC-associated Kidney Cancer

NCT03165721
View directory record Official record
Conditions
Paraganglioma, Gastrointestinal Stromal Tumors, Carcinoma, Renal Cell, Renal Neoplasms, Pheochromocytoma
Interventions
SGI-110 (guadecitabine)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 99 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A Test to Predict the Hemolytic Potential of Drugs in G6PD Deficiency

NCT00076323
View directory record Official record
Conditions
Glucosephosphate Dehydrogenase Deficiency, Favism
Interventions
Primaquine
Drug
Lead sponsor
Walter Reed Army Institute of Research (WRAIR)
Federal
Eligibility
18 Years to 60 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2006
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 27, 2010 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Validation of a Diagnostic Test for Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Anti-coagulated Blood

NCT04054661
View directory record Official record
Conditions
G6PD Deficiency
Interventions
SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
Diagnostic Test
Lead sponsor
PATH
Other
Eligibility
18 Years to 65 Years
Enrollment
229 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Reading, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 9, 2021 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 2 Interventional

CMV Retinitis Retreatment Trial

NCT00000766
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2011 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional

A Study of AZT Plus Ganciclovir in Patients With AIDS and Cytomegalovirus (CMV) Infection

NCT00000995
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Zidovudine, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1990
U.S. locations
3
States / cities
San Diego, California • New York, New York • Rochester, New York
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 23, 2026, 9:25 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 1 Interventional

A Phase I Study of the Safety of Proleukin (Aldesleukin) in Combination With Ganciclovir and Antiretroviral Therapy in HIV Seropositive Patients With Cytomegalovirus (CMV) Retinitis

NCT00002321
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Aldesleukin
Drug
Lead sponsor
Chiron Corporation
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Toledo, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 1 Interventional

A Randomized Comparative Pharmacokinetic Study of Oral Ganciclovir After Treatment With Intravenous Ganciclovir for Cytomegalovirus Gastrointestinal Disease in AIDS Patients

NCT00000768
View directory record Official record
Conditions
Colitis, HIV Infections
Interventions
Glutamic acid hydrochloride, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
5
States / cities
Birmingham, Alabama • San Francisco, California • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Metabolism and Pharmacokinetics of Primaquine Enantiomers in Human Volunteers Receiving a Seven Day Dose Regimen

NCT03934450
View directory record Official record
Conditions
Malaria, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
RPQ, SPQ, Primaquine Phosphate, Placebo
Drug
Lead sponsor
University of Mississippi, Oxford
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
University, Mississippi
Source: ClinicalTrials.gov public record
Updated May 14, 2019 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional

Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections

NCT00002024
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 9:25 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Demographic, Metabolic, and Genomic Description of Neonates With Severe Hyperbilirubinemia

NCT00383318
View directory record Official record
Conditions
Hyperbilirubinemia, Jaundice
Interventions
Gene mutation sample
Procedure
Lead sponsor
Pediatrix
Other
Eligibility
Up to 6 Days
Enrollment
450 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2007
U.S. locations
1
States / cities
Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Jan 28, 2008 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Not applicable Interventional

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

NCT00002273
View directory record Official record
Conditions
Colitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
21 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 9:25 AM EDT
Recruiting Phase 2 Interventional

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

NCT06773026
View directory record Official record
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 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 23, 2026, 9:25 AM EDT
Recruiting No phase listed Observational

Natural History of MADD

NCT07734090
View directory record Official record
Conditions
Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 23, 2026, 9:25 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Primaquine Enantiomers in G6PD Deficient Human Volunteers

NCT04073953
View directory record Official record
Conditions
G6PD Deficiency
Interventions
RPQ, SPQ, Placebo
Drug
Lead sponsor
University of Mississippi, Oxford
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
University, Mississippi
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 23, 2026, 9:25 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Comparative Evaluation of the FINDER Instrument and FINDER G6PD Cartridge in Adults and Neonates

NCT04146246
View directory record Official record
Conditions
G6PD, G6PD Deficiency
Interventions
G6PD assay
Diagnostic Test
Lead sponsor
Baebies, Inc.
Industry
Eligibility
35 Weeks to 100 Years
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Durham, North Carolina • Raleigh, North Carolina • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 23, 2026, 9:25 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

An Investigation of Kidney and Urothelial Tumor Metabolism in Patients Undergoing Surgical Resection and/or Biopsy

NCT04623502
View directory record Official record
Conditions
Kidney Cancer, Renal Cell Carcinoma, Clear Cell Carcinoma, Urothelial Carcinoma, Metastatic Kidney Cancer, Metastatic Urothelial Carcinoma, Papillary Renal Cell Carcinoma, Chromophobe Renal Cell Carcinoma, Fumarate Hydratase Deficiency, Succinate Dehydrogenase-Deficient Renal Cell Carcinoma, HLRCC, Hereditary Leiomyomatosis and Renal Cell Cancer
Interventions
13C-Glucose, 13C-Acetate, 13C-Lactate, 13C-Glutamine, 13C-Fructose
Other
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years and older
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 23, 2026, 9:25 AM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 23, 2026, 9:25 AM EDT