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Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease

NCT06287086 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2024-06-03

No results posted yet for this study

Summary

This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)

Conditions

Interventions

DRUG

BRL-101 autologous hematopoietic stem and progenitor cells injection

CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A gene.

Sponsors & Collaborators

  • Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College

    collaborator UNKNOWN

  • Bioray Laboratories

    lead INDUSTRY

Principal Investigators

  • Jun shi, PhD · Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-14
Primary Completion
2025-08-20
Completion
2026-06-10

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06287086 on ClinicalTrials.gov