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ClinicalTrials.gov public records Last synced Oct 17, 2026, 11:18 PM EDT

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Showing 1–24 of 168 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Single Gene Disorders Clear all
Active, not recruiting Phase 1Phase 2 Interventional

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

NCT01687608
View directory record Official record
Conditions
Hemophilia B
Interventions
AskBio009
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
15
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of the Effectiveness of an HIV Vaccine (ALVAC vCP205) to Boost Immune Functions in HIV-Negative Volunteers Who Have Already Received an HIV Vaccine

NCT00001136
View directory record Official record
Conditions
HIV Infections, HIV Seronegativity
Interventions
ALVAC-HIV MN120TMG (vCP205)
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 60 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Ends 2001
U.S. locations
6
States / cities
Birmingham, Alabama • Baltimore, Maryland • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 17, 2026, 11:18 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
ABO-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 25, 2023 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 4 Interventional

D-Cycloserine for Serine Palmitoyltransferase Inhibition

NCT07542548
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
D-cycloserine
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
6 Years to 6 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)

NCT01023048
View directory record Official record
Conditions
Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Interventions
preimplantation diagnosis
Other
Lead sponsor
Natera, Inc.
Industry
Eligibility
Up to 40 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Apr 2, 2014 · Synced Oct 17, 2026, 11:18 PM EDT
Completed No phase listed Observational

Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers

NCT06667414
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 60 Years
Enrollment
574 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
13
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 2 Interventional Results available

Randomized Trial of Suicide Gene Therapy and Prostate Cancer

NCT00583492
View directory record Official record
Conditions
Prostate Cancer
Interventions
Ad5-yCD/mutTKSR39rep-ADP, IMRT
Biological · Radiation
Lead sponsor
Henry Ford Health System
Other
Eligibility
18 Years and older · Male only
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
2
States / cities
Baltimore, Maryland • Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 17, 2016 · Synced Oct 17, 2026, 11:18 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

NCT06948019
View directory record Official record
Conditions
HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions
BFB-101 (AAV9-CBh-AP4B1)
Biological
Lead sponsor
BlackfinBio Ltd
Industry
Eligibility
12 Months to 60 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Oct 17, 2026, 11:18 PM EDT
Recruiting Phase 3 Interventional

A Phase 3 Trial for Intravitreal Injection of 4D-150 in Adults With Diabetic Macular Edema (DME).

NCT07867080
View directory record Official record
Conditions
Diabetic Macular Edema (DME)
Interventions
4D-150 IVT (3x10^10 vg), EYELEA ® (aflibercept) Injection 2 mg (0.05mL)
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
514 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
49
States / cities
Scottsdale, Arizona • Encino, California • Fullerton, California + 44 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

PS-002 for the Treatment of IgA Nephropathy in Adults

NCT07182227
View directory record Official record
Conditions
Immunoglobulin A (IgA) Nephropathy
Interventions
PS-002
Genetic
Lead sponsor
Purespring Therapeutics Limited
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Miami, Florida • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

NCT04370054
View directory record Official record
Conditions
Hemophilia A
Interventions
PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
19
States / cities
Mountain View, California • Oakland, California • Palo Alto, California + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 3 Interventional Results available

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua, Factor IX (FIX)
Genetic · Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
19
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Active, not recruiting Phase 3 Interventional

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT05898620
View directory record Official record
Conditions
Rett Syndrome
Interventions
NGN-401
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years and older · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
13
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
No Longer Available No phase listed Expanded access

Canavan-Single Patient IND

NCT05317780
View directory record Official record
Conditions
Canavan Disease
Interventions
rAAV9-CB6-AspA
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Months to 24 Months · Male only
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 23, 2023 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 3 Interventional Results available

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03306277
View directory record Official record
Conditions
SMA - Spinal Muscular Atrophy, Gene Therapy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 180 Days
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
16
States / cities
Los Angeles, California • Stanford, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316
View directory record Official record
Conditions
Fabry Disease
Interventions
AMT-191
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 1 Interventional

Study of the Glutaminase Inhibitor CB-839 in Solid Tumors

NCT02071862
View directory record Official record
Conditions
Solid Tumors, Triple-Negative Breast Cancer, Non Small Cell Lung Cancer, Renal Cell Carcinoma, Mesothelioma, Fumarate Hydratase (FH)-Deficient Tumors, Succinate Dehydrogenase (SDH)-Deficient Gastrointestinal Stromal Tumors (GIST), Succinate Dehydrogenase (SDH)-Deficient Non-gastrointestinal Stromal Tumors, Tumors Harboring Isocitrate Dehydrogenase-1 (IDH1) and IDH2 Mutations, Tumors Harboring Amplifications in the cMyc Gene
Interventions
CB-839, Pac-CB, CBE, CB-Erl, CBD, CB-Cabo
Drug
Lead sponsor
Calithera Biosciences, Inc
Industry
Eligibility
18 Years and older
Enrollment
210 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
13
States / cities
San Francisco, California • Stanford, California • Sarasota, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2022 · Synced Oct 17, 2026, 11:18 PM EDT
Completed No phase listed Observational

Genotype Information and Functional Testing Study

NCT00992420
View directory record Official record
Conditions
Coronary Artery Disease
Interventions
Not listed
Lead sponsor
Scripps Health
Other
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Nov 23, 2022 · Synced Oct 17, 2026, 11:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

NCT06526923
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
SP-101 and doxorubicin Cohort 1, SP-101 and doxorubicin Cohort 2
Combination Product
Lead sponsor
Spirovant Sciences, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
4
States / cities
Kansas City, Kansas • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2024 · Synced Oct 17, 2026, 11:18 PM EDT
Available No phase listed Expanded access

BLESSED: Expanded Access for DNG64-CAR-V for Advanced Pancreatic Cancer, Sarcoma and Carcinoma of Breast

NCT04091295
View directory record Official record
Conditions
Pancreatic Cancer, Osteosarcoma, MPNST (Malignant Peripheral Nerve Sheath Tumor), Chondrosarcoma, Soft Tissue Sarcoma, Chordoma, Sarcoma, Carcinoma of Breast, Single Patient IND for Glioblastoma, Ovarian Carcinoma, Non-small Cell Lung Cancer , Prostate Cancer
Interventions
DNG64-CAR-V
Drug
Lead sponsor
Aveni Foundation
Other
Eligibility
12 Years to 100 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Santa Monica, California
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Oct 17, 2026, 11:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss

NCT07627971
View directory record Official record
Conditions
GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
Interventions
SKY-GJB2
Combination Product
Lead sponsor
Skylark Bio Inc.
Industry
Eligibility
9 Months to 7 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Bethesda, Maryland • Boston, Massachusetts • Oklahoma City, Oklahoma + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 17, 2026, 11:18 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Clinical Trial of an Alphavirus Replicon Vaccine for Cytomegalovirus (CMV)

NCT00439803
View directory record Official record
Conditions
Cytomegalovirus Infections
Interventions
AVX601, Placebo
Biological
Lead sponsor
AlphaVax, Inc.
Industry
Eligibility
18 Years to 45 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2008
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 10, 2008 · Synced Oct 17, 2026, 11:18 PM EDT