Status:
TERMINATED
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
Lead Sponsor:
Rocket Pharmaceuticals Inc.
Collaborating Sponsors:
California Institute for Regenerative Medicine (CIRM)
Conditions:
Infantile Malignant Osteopetrosis
Eligibility:
All Genders
1+ years
Phase:
PHASE1
Brief Summary
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
Detailed Description
This is a non-randomized Phase 1 study to evaluate the preliminary safety and efficacy of hematopoietic gene therapy consisting of autologous CD34+ enriched hematopoietic cells transduced with the len...
Eligibility Criteria
Inclusion
- A confirmed diagnosis of IMO with documented TCIRG1 mutation.
- Age at least 1 month with minimum weight of 4 kg
- Absence of debilitating hydrocephalus (defined as hydrocephalus at NCI CTCAE v5.0 Grade 3 or higher persisting despite shunt or similar procedural intervention).
- Lansky Play Scale of at least 60%
- Preserved hepatic function (AST/ALT ≤3.0 ULN; bilirubin ≤1.5 ULN; to minimize potential for excessive toxicity from busulfan conditioning)
- No concomitant medical or other conditions that would represent a contraindication to autologous hematopoietic stem cell transplant.
- Absolute neutrophil count of ≥500/mm3 and platelet count of ≥25,000/mm3
- No prior allogeneic or other hematopoietic stem cell transplant.
- Availability of a non-autologous rescue (back-up) hematopoietic stem cell donor/source
Exclusion
- Availability of medically-feasible HLA-matched sibling donor for allogeneic HSCT.
- Any medical or other contraindication for either apheresis or autologous transplant as determined by the Investigator.
- Participation in another clinical trial with an investigational drug within 14 days before the informed consent signature. Participation in observational studies is allowed.
- Active hematologic or solid organ malignancy, not including non-melanoma skin cancer or another carcinoma in situ.
- Uncontrolled seizure disorder.
- Renal dysfunction as defined by a glomerular filtration rate \<30 mL/min/1.73m2 or dialysis dependence.
- Serious infections with persistent bloodstream pathogens at time of trial entry
- Pulmonary dysfunction as defined by either:
- Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection) or
- Oxygen saturation (by pulse oximetry) \<90% resulting from pulmonary conditions (intermittent hypoxia secondary to IMO-related choanal atresia will not be considered exclusionary)
Key Trial Info
Start Date :
November 19 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 21 2021
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT04525352
Start Date
November 19 2020
End Date
May 21 2021
Last Update
July 13 2022
Active Locations (1)
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1
University of California, Los Angeles
Los Angeles, California, United States, 90095