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Clinical trials
catalyst calendar across sponsors →Studies where RCKT is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
12 interventional · 7 observational
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102NCT07527975observationalSurvival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118). | — | Enrolling by invitation | Biological: RP-L102 | Fanconi Anemia | 14 | May 2038 | ≤ 4,303 d | 2026-04-14 |
| Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)NCT06282432observationalHematopoietic stem cell transplant (HSCT) free survival | — | Active, not recruiting | — | Leukocyte Adhesion Deficiency | 9 | 2036-10-04 | in 3,699 d | 2025-12-18 |
| Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene TherapyNCT04437771observationalMonitor long term safety of patients through blood laboratory evaluations and general health status | — | Active, not recruiting | Other: Safety and efficacy assessments | Fanconi Anemia Complementation Group A, Fanconi Anemia | 9 | 2034-01-17 | in 2,708 d | 2026-04-13 |
| BAG3-DCM Natural History StudyNCT07486752observationalCardiac structure and function | — | Not yet recruiting | — | Dilated Cardiomyopathy (DCM), Cardiovascular Diseases, Heart Diseases, Genetic Diseases | 30 | Dec 2032 | ≤ 2,326 d | 2026-03-20 |
| PKP2-ACM Natural History StudyNCT06644742observationalHeart rhythm and rate monitoring measures | — | Recruiting | — | Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Genetic Diseases | 36 | Dec 2030 | ≤ 1,595 d | 2026-02-27 |
| Danon Disease Natural History StudyNCT06214507observationalLeft Ventricular Mass Index (LVMI) by echocardiogram | — | Recruiting | Other: No intervention | Danon Disease | 60 | Dec 2029 | ≤ 1,230 d | 2026-07-28 |
| A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated CardiomyopathyNCT07137338Incidence of Treatment-emergent Adverse Events (TEAE)Open-label · Treatment | Phase 1 | Recruiting | Genetic: 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) | Dilated Cardiomyopathy (DCM) | 8 | Jun 2029 | ≤ 1,046 d | 2026-06-30 |
| A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)NCT05885412Evaluation of safety associated with RP-A601Open-label · Treatment | Phase 1 | Recruiting | Genetic: RP-A601 | PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | 9 | Sep 2028 | ≤ 773 d | 2026-04-16 |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon DiseaseNCT06092034Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass indexOpen-label · Treatment | Phase 2 | Recruiting | Genetic: RP-A501 | Danon Disease | 14 | Apr 2028 | ≤ 620 d | 2026-05-04 |
| Gene Therapy for Fanconi Anemia, Complementation Group ANCT04248439Bone Marrow (BM) Colony-Forming Cell (CFC) Mitomycin-C (MMC) resistanceOpen-label · Treatment | Phase 2 | Active, not recruiting | Biological: RP-L102 | Fanconi Anemia Complementation Group A | 5 | 2026-05-05 | — | 2025-12-22 |
| Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase DeficiencyNCT06422351Improvement in AnemiaOpen-label · Treatment | Phase 2 | Suspendedsponsor's stated reason: Initiation of study has been paused, but may re-start in the future. | Biological: RP-L301 | Pyruvate Kinase Deficiency | 10 | Jan 2029 | ≤ 896 d | 2026-04-16 |
| Gene Therapy for Pyruvate Kinase Deficiency (PKD)NCT04105166Evaluation of the safety and toxicity of RP-L301: number of participants with treatment-related adverse eventsOpen-label · Treatment | Phase 1 | Completed | Biological: RP-L301 | Pyruvate Kinase Deficiency | 4 | 2025-06-09actual | — | 2025-09-05 |
| Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2BNCT03882437Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute Common Terminology Criteria (NCI CTCAE)Open-label · Treatment | Phase 1 | Unknown status | Biological: RP-A501 | Danon Disease | 7 | 2025-03-24 | — | 2023-03-03 |
| Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype ANCT04069533Phenotypic correction of bone marrow colony forming units after infusion of RP-L102Open-label · Treatment | Phase 2 | Unknown status | Biological: RP-L102 | Fanconi Anemia Complementation Group A | 7 | Feb 2025 | — | 2024-05-22 |
| Natural History of Danon DiseaseNCT05548855observationalCardiac Manifestations- LVPWd | — | Completed | Procedure: Heart Transplant · Device: Cardiac Assistive Device | Danon Disease | 59 | 2023-10-31actual | — | 2023-11-21 |
| A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-INCT03812263Phase I: Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.5.0Open-label · Treatment | Phase 1/2 | Completed | Biological: RP-L201 | Leukocyte Adhesion Defect - Type I | 9 | 2023-09-12actual | — | 2023-11-15 |
| A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-INCT03825783Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.5.0Open-label · Treatment | Phase 1 | Withdrawnsponsor's stated reason: Patients were enrolled under global Phase 1/2 protocol RP-L201-0318. | Biological: RP-L201 | Leukocyte Adhesion Defect - Type I | 0 | Oct 2021 | — | 2021-12-07 |
| A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant OsteopetrosisNCT04525352Number of participants with treatment-related adverse events as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0Open-label · Treatment | Phase 1 | Terminatedsponsor's stated reason: Study discontinued due to feasibility. | Biological: RP-L401 | Infantile Malignant Osteopetrosis | 1 | 2021-05-21actual | — | 2022-07-13 |
| A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype ANCT03814408Number of participants with treatment-related adverse events as assessed by CTCAE v.5.0Open-label · Treatment | Phase 1 | Unknown status | Biological: RP-L102 | Fanconi Anemia Complementation Group A | 2 | Dec 2020 | — | 2020-11-24 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19