A Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations

NCT03728322 EARLY_PHASE1 UNKNOWN

This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.

Details

Lead sponsor	Allife Medical Science and Technology Co., Ltd.
Phase	EARLY_PHASE1
Status	UNKNOWN
Enrolment	12
Start date	2019-01
Completion	2021-01

Conditions

• Thalassemia

Interventions

• iHSCs treatment group

Primary outcomes

• Occurrence of treatment related adverse events as assessed by CTCAE v4.0 — 1 year
  Defined as \>= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment