Actively Recruiting

Phase 1
Age: 5Years - 35Years
All Genders
NCT05757245

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

Led by First Affiliated Hospital of Guangxi Medical University · Updated on 2023-04-18

5

Participants Needed

1

Research Sites

399 weeks

Total Duration

On this page

Sponsors

F

First Affiliated Hospital of Guangxi Medical University

Lead Sponsor

G

Genmedicn Biopharma Ltd.

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 5Years - 35Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • The participant or their legal guardian understands the study and has signed informed consent.
  • Age between 5 and 35 years, any gender.
  • Clinical diagnosis of transfusion-dependent -thalassemia requiring 6 or more units of blood transfusions in the past 24 weeks without more than 56 days without transfusion.
  • Karnofsky or Lansky performance score of 70 or higher.
  • Determined to undergo autologous hematopoietic stem cell transplantation and conditioning by the principal investigator.
  • Willingness to comply with the study protocol.
  • Fertile participants agree to use effective contraception during the study.
Not Eligible

You will not qualify if you...

  • Diagnosed with mild -thalassemia, Hb Bart's edema, ATRx -thalassemia, hemoglobin S/-thalassemia, myelodysplastic anemia subtype, HbE homozygous  gene mutation, or any -thalassemia.
  • Uncorrected bleeding disorders with frequent bleeding.
  • Clinically significant bacterial, fungal, parasitic, or viral infection.
  • Severe iron overload.
  • Any prior or current cancer, myeloproliferative disorder, or immunodeficiency.
  • Major medical disease, abnormal lab tests, or mental illness making participation unsuitable.
  • Immediate family history of familial cancer syndrome.
  • Prior gene therapy or allogeneic bone marrow or stem cell transplant.
  • Participation in another investigational drug study within 3 months before screening.
  • Pregnancy, planning pregnancy, or breastfeeding.
  • Known allergy to study drug ingredients.
  • Eligible for allogeneic transplant with a known donor.
  • Any other condition deemed exclusionary by the investigator.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

The affiliated hospital of guangxi medical university

Nanning, Guangxi, China, 530021

Actively Recruiting

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How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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