Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease
NCT06287099 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 5
Last updated 2024-03-19
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
Subjects will receive a single infusion of BRL-101.
Sponsors & Collaborators
-
Nanfang Hospital, Southern Medical University
collaborator OTHER -
Bioray Laboratories
lead INDUSTRY
Principal Investigators
-
Xiaoqin Feng, PhD · Nanfang Hospital, Southern 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
- 2024-04-20
- Primary Completion
- 2025-10-20
- Completion
- 2026-05-10
More Related Trials
-
Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function
NCT06145282 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
CS-206 in Patients With Sickle Cell Disease
NCT06565026 ·Status: RECRUITING ·Phase: EARLY_PHASE1
-
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
NCT05329649 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care
NCT03167450 ·Status: COMPLETED
-
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
NCT03745287 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
Gene Editing For Sickle Cell Disease
NCT06506461 ·Status: RECRUITING ·Phase: PHASE1
-
A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3
-
An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
NCT04053803 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2
-
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
NCT02380079 ·Status: UNKNOWN ·Phase: PHASE1
-
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
NCT04925206 ·Status: TERMINATED ·Phase: PHASE1
-
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
NCT06975865 ·Status: RECRUITING ·Phase: PHASE3
-
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
NCT00445978 ·Status: COMPLETED ·Phase: PHASE2
-
A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
NCT03132324 ·Status: TERMINATED ·Phase: PHASE1
-
A Study of IMR-687 in Subjects With Sickle Cell Disease
NCT04474314 ·Status: TERMINATED ·Phase: PHASE2
-
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
NCT06024876 ·Status: COMPLETED ·Phase: EARLY_PHASE1
-
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
NCT04628585 ·Status: ENROLLING_BY_INVITATION
-
An Assessment of Prasugrel on Healthy Adults and Sickle Cell Adults
NCT01178099 ·Status: COMPLETED ·Phase: PHASE1
-
Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study
NCT02090296 ·Status: WITHDRAWN ·Phase: PHASE2
-
Crizanlizumab Improves Tissue Oxygen Supply Demand Matching in Patients With Sickle Cell Anemia
NCT05469828 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1/PHASE2
-
Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
NCT06399107 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders
NCT01316796 ·Status: COMPLETED ·Phase: PHASE1
-
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
NCT06041620 ·Status: RECRUITING ·Phase: NA
-
CS-101 in Patients With β-thalassemia
NCT06328764 ·Status: ENROLLING_BY_INVITATION ·Phase: EARLY_PHASE1
-
Gene Transfer for Sickle Cell Disease
NCT03282656 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1
-
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
NCT04819841 ·Status: RECRUITING ·Phase: PHASE1/PHASE2