TrialLentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
Trial details
- Registry ID
- NCT04069533
- Phase
- Phase 2
RCKT
Rocket Pharmaceuticals Inc.
Company identity checked against sources
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4Trials in this database
8Recorded changes · last 12 months
Partial coverageOnly the trials and history collected so far
Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.
Completion dates · Pro
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Pro summarizes how far estimated completion dates moved later and counts trials with edits to their main study measures, using the history available here. View Pro features
Included trials
Check company identity source| Trial | Sponsor | Phase | Status | Primary completion |
|---|---|---|---|---|
| Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype ANCT04069533 | RCKT | Phase 2 | Unknown | 2025-02Estimated |
| Gene Therapy for Fanconi Anemia, Complementation Group ANCT04248439 | RCKT | Phase 2 | Active not recruiting | 2026-05-05Estimated |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon DiseaseNCT06092034 | RCKT | Phase 2 | Recruiting | 2028-04Estimated |
| Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase DeficiencyNCT06422351 | RCKT | Phase 2 | Suspended | 2029-01Estimated |
Gene Therapy for Fanconi Anemia, Complementation Group A
Trial details
- Registry ID
- NCT04248439
- Phase
- Phase 2
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Trial details
- Registry ID
- NCT06092034
- Phase
- Phase 2
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
Trial details
- Registry ID
- NCT06422351
- Phase
- Phase 2
Latest recorded trial changes
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
NCT06422351 · Rocket Pharmaceuticals Inc.
Primary completion date changed
− Before
Nov 2026
+ After
Jan 2029
Recruitment status changed
− Before
Not yet recruiting
+ After
Suspended
Gene Therapy for Fanconi Anemia, Complementation Group A
NCT04248439 · Rocket Pharmaceuticals Inc.
Primary completion date changed
− Before
May 2026
+ After
5 May 2026
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
NCT06092034 · Rocket Pharmaceuticals Inc.
Main study measure wording changed
Text changed; whether the study measures changed has not been verified. Saved text may be shortened.
Compare wording
− Before
Measure: Evaluation of efficacy associated with RP-A501 via co-primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
+ After
Measure: Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
Participant count type changed
− Before
Actual
+ After
Estimated
Enrollment · participant count changed
− Before
12 participants (actual)
+ After
14 participants (planned)
Primary completion date changed
− Before
Sep 2025
+ After
Apr 2028
Recruitment status changed
− Before
Active not recruiting
+ After
Recruiting