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A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants

NCT05757245 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2023-04-18

No results posted yet for this study

Summary

This is a non-randomized, open label, single-site, single-dose, phase 1 study in up to 5 participants (between 5 and 35 years of age, inclusive) with Transfusion-dependent α-thalassemia. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using GMCN-508A Drug Product \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].

Conditions

  • Transfusion-dependent α-Thalassemia

Interventions

GENETIC

GMCN-508A Drug Product

Transplant of autologous hematopoietic stem and progenitor cells transduced with GMCN-508A lentiviral vector.

Sponsors & Collaborators

  • Genmedicn Biopharma Ltd.

    collaborator UNKNOWN

  • First Affiliated Hospital of Guangxi Medical University

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2023-05-08
Primary Completion
2028-08-01
Completion
2030-12-31

Countries

  • China

Study Locations

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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 NCT05757245 on ClinicalTrials.gov