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Clinical Study of BRL-101 in Severe SCD

NCT06300723 · Status: ENROLLING_BY_INVITATION · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2024-07-31

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

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

Sponsors & Collaborators

  • First Affiliated Hospital of Guangxi Medical University

    collaborator OTHER

  • Bioray Laboratories

    lead INDUSTRY

Principal Investigators

  • Yongrong Lai, phD · First Affiliated Hospital of Guangxi Medical University

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
2024-07-29
Primary Completion
2026-03-20
Completion
2026-06-15

Countries

  • China

Study Locations

More Related Trials

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 NCT06300723 on ClinicalTrials.gov