Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 4, 2026, 12:49 PM EDT

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Showing 1–24 of 101 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Zidovudine, Didanosine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
3
States / cities
Irvine, California • Washington D.C., District of Columbia • Galveston, Texas
Conditions
Hereditary Neuropathy Caused by SORD Deficiency
Interventions
AT-007, Placebo
Drug
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Coral Gables, Florida • Iowa City, Iowa + 4 more
Conditions
Brain Neoplasm, Neoplasm Metastasis
Interventions
Cytovene (Ganciclovir Sodium), G1TKSVNa.53 Producer Cell Line
Drug · Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
19 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Interferon beta-1b, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 60 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
6
States / cities
Los Angeles, California • Orange, California • San Diego, California + 3 more
Conditions
Molybdenum Cofactor Deficiency, Type A
Interventions
ORGN001 (formerly ALXN1101)
Drug
Lead sponsor
Origin Biosciences
Industry
Eligibility
Not listed
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Roche Global Development
Other
Eligibility
3 Months to 12 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 3 more
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1993
U.S. locations
6
States / cities
Los Angeles, California • San Francisco, California • St Louis, Missouri + 3 more
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
Not yet recruiting No phase listed Observational

Natural History of MADD

NCT07734090
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
Completed No phase listed Observational Accepts healthy volunteers

Taurine Therapy for SSADH Deficiency

NCT01608178
Conditions
Succinic Semialdehyde Dehydrogenase
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
12 Years and older
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
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
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
Conditions
Pyruvate Dehydrogenase Complex Deficiency Disease
Interventions
No intervention
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Completed Early Phase 1 Interventional Accepts healthy volunteers

Treatment of Asian Flushing Syndrome With Topical Alpha Agonists

NCT03497442
Conditions
Flushing, Alcohol-Related Disorders, Aldehyde Dehydrogenase Deficiency
Interventions
Brimonidine Tartrate, Placebo Vehicle Gel
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
21 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
San Francisco, California
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Fomivirsen sodium, Ganciclovir
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
18
States / cities
Los Angeles, California • Pasadena, California • San Diego, California + 14 more
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Globulin, Immune, Ganciclovir
Drug
Lead sponsor
American National Red Cross
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Completed Phase 1 Interventional Accepts healthy volunteers

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

NCT01894165
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
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
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 60 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1995
U.S. locations
5
States / cities
San Diego, California • San Francisco, California • New York, New York
Completed Phase 1Phase 2 Interventional Results available

Smith-Lemli-Opitz Syndrome and Cholic Acid

NCT03720990
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
Conditions
Metabolic Disease, Seizures
Interventions
SGS-742, Placebo
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
4 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Bethesda, Maryland • Pullman, Washington
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Oakland, California • San Francisco, California + 5 more
Completed Phase 1 Interventional Accepts healthy volunteers

Primaquine Enantiomers in G6PD Deficient Human Volunteers

NCT04073953
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
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2035
U.S. locations
13
States / cities
Phoenix, Arizona • San Francisco, California • Aurora, Colorado + 10 more