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ClinicalTrials.gov public records Last synced Oct 17, 2026, 1:15 PM EDT

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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Rocket Pharmaceuticals Inc. Clear all
Active, not recruiting No phase listed Observational

Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

NCT06282432
View directory record Official record
Conditions
Leukocyte Adhesion Deficiency
Interventions
Not listed
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
3 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2036
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 17, 2026, 1:15 PM EDT
Terminated Phase 1 Interventional

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

NCT04525352
View directory record Official record
Conditions
Infantile Malignant Osteopetrosis
Interventions
RP-L401
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Month and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 13, 2022 · Synced Oct 17, 2026, 1:15 PM EDT
Suspended Phase 2 Interventional

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

NCT06422351
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

NCT04105166
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 50 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Sep 5, 2025 · Synced Oct 17, 2026, 1:15 PM EDT
Recruiting No phase listed Observational

Danon Disease Natural History Study

NCT06214507
View directory record Official record
Conditions
Danon Disease
Interventions
No intervention
Other
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
9
States / cities
Phoenix, Arizona • La Jolla, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Recruiting Phase 2 Interventional

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
3
States / cities
La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Recruiting Phase 1 Interventional

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

NCT05885412
View directory record Official record
Conditions
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Interventions
RP-A601
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
La Jolla, California • Durham, North Carolina • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Not listed Phase 1 Interventional

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

NCT03814408
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 17, 2026, 1:15 PM EDT
Enrolling by invitation No phase listed Observational

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

NCT07527975
View directory record Official record
Conditions
Fanconi Anemia
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2038
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Recruiting Phase 1 Interventional

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

NCT07137338
View directory record Official record
Conditions
Dilated Cardiomyopathy (DCM)
Interventions
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
San Diego, California • Rochester, Minnesota • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Completed Phase 1Phase 2 Interventional

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

NCT03812263
View directory record Official record
Conditions
Leukocyte Adhesion Defect - Type I
Interventions
RP-L201
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
3 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 15, 2023 · Synced Oct 17, 2026, 1:15 PM EDT
Active, not recruiting Phase 2 Interventional

Gene Therapy for Fanconi Anemia, Complementation Group A

NCT04248439
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Stanford, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 17, 2026, 1:15 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Recruiting No phase listed Observational

PKP2-ACM Natural History Study

NCT06644742
View directory record Official record
Conditions
Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Genetic Diseases
Interventions
Not listed
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
12 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Boston, Massachusetts • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 17, 2026, 1:15 PM EDT
Not listed No phase listed Observational

The Natural History of Danon Disease

NCT03766386
View directory record Official record
Conditions
Danon Disease
Interventions
Not listed
Lead sponsor
University of California, San Diego
Other
Eligibility
0 Years to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated May 12, 2022 · Synced Oct 17, 2026, 1:15 PM EDT