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NCT06287086

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

Not yet recruiting NA Last updated 3 June 2024
What this trial tests

NA trial testing BRL-101 autologous hematopoietic stem and progenitor cells injection in Sickle Cell Disease in 1 participant. Not yet recruiting.

Timeline
14 June 2024
Primary endpoint
20 August 2025
10 June 2026

Quick facts

Lead sponsorBioray Laboratories
PhaseNA
StatusNot yet recruiting
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment1
Start date14 June 2024
Primary completion20 August 2025
Estimated completion10 June 2026

Drugs / interventions tested

Conditions studied

Sponsor

Bioray Laboratories — full company profile →

Who can join

Adults 3 to 35, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Recent advances in therapeutic gene-editing technologies.
    Liu D, Cao D, Han R. · · 2025 · cited 17× · PMID 40119516 · DOI 10.1016/j.ymthe.2025.03.026
  2. Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.
    Ball J, Bradley A, Le A, Tisdale JF, et al · · 2025 · cited 5× · PMID 40083162 · DOI 10.1016/j.ymthe.2025.03.016
  3. CRISPR CLIP: comprehensive reviews on interventional studies using precision recombinant technologies: clinical landmarks, implications, and prospects.
    Chodnekar SY, Tsetskhladze Z. · · 2024 · cited 1× · PMID 39659430 · DOI 10.1093/oxfimm/iqae013
  4. Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies.
    O'Hanlon Cohrt K, O'Dea S. · · 2026 · PMID 42074029 · DOI 10.3390/ijms27083384
  5. Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges.
    Yang P, Khoshandam M, Bhia I, Raji S, et al · · 2025 · PMID 41809894 · DOI 10.1016/j.ajps.2025.101068
  6. Therapeutic applications of CRISPR-Cas9 gene editing.
    Bharti A, Mudge J. · · 2025 · PMID 41476860 · DOI 10.3389/fgeed.2025.1724291
  7. Emerging trends in gene and cell therapy: CRISPR in DNA editing and beyond.
    Ji R, Chen Q, Zhang Y. · · 2026 · PMID 41418774 · DOI 10.1016/j.xcrm.2025.102459
  8. Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.
    Aprile A, Lidonnici MR, Ferrari G. · · 2025 · PMID 41253147 · DOI 10.1016/j.xcrm.2025.102419

Verify or expand the search:

Other recruiting trials for Sickle Cell Disease

Currently open trials in the same condition.

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