NCT04525352

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

A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

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 lentiviral vector (LV) carrying the human TCIRG1 transgene (RP-L401) in pediatric patients with IMO. Following myeloablative conditioning patients will receive an infusion of the genetically modified hematopoietic stem and progenitor cells (HSPCs).

Conditions

• Infantile Malignant Osteopetrosis

Interventions

Type	Name	Description
BIOLOGICAL	RP-L401	CD34+ enriched hematopoietic stem cells from pediatric subjects with infantile malignant osteopetrosis transduced ex vivo with lentiviral vector carrying the TCIRG1 transgene

Timeline

• Start date: 2020-11-19
• Primary completion: 2021-05-21
• Completion: 2021-05-21
• First posted: 2020-08-25
• Last updated: 2022-07-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study