Actively Recruiting
Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease
Led by The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army · Updated on 2024-06-03
5
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
Sponsors
T
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Lead Sponsor
G
Guangzhou Reforgene Medicine Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and efficacy of RM-004, a gene-edited autologous hematopoietic stem cell treatment, for people aged 12 to 35 with Hemoglobin H-Constant Spring disease. This rare blood disorder is characterized by specific genetic mutations and requires frequent blood transfusions. The study aims to assess how well RM-004 works and its safety profile in this patient group. The study is an open-label, non-randomized trial where all participants receive a single intravenous infusion of RM-004 after undergoing conditioning with busulfan to prepare their body for the treatment. This treatment involves using the participant's own genetically modified stem cells to potentially improve their condition. Participants will be closely monitored for up to 24 months after the infusion. Researchers will track safety, the ability of the infused cells to engraft within 42 days, and whether participants achieve independence from blood transfusions. Additional assessments include mortality rates, duration of transfusion independence, and the proportion of participants who stop transfusions for six months or more. Follow-up visits and evaluations will help researchers understand the long-term effects of RM-004.
CONDITIONS
Brief Title
Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subjects voluntarily sign informed consent and agree to complete study procedures, follow-up, and treatment.
- Age between 12 and 35 years at screening, any gender.
- History of at least 100 mL/kg/year of packed red blood cell transfusions in the 2 years before screening.
- Diagnosed with Hemoglobin H-Constant Spring disease (--/\u03b1\u03b1CS) with HBA2 c.427T>C mutation.
You will not qualify if you...
- Availability of an HLA-matched or well-matched donor for allogeneic hematopoietic stem cell transplantation.
- Prior hematopoietic stem cell transplant or gene therapy.
- History of severe bleeding disorders.
- Active bacterial, viral, fungal, or parasitic infections judged significant by the investigator at screening.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 42 days after infusion
Participants receive a single intravenous infusion of RM-004 cells following myeloablative conditioning with busulfan.
Multiple visits for treatment and engraftment monitoring up to 42 days
Duration - Up to 24 months after RM-004 infusion
Participants are monitored for safety and efficacy outcomes including transfusion independence for up to 24 months after RM-004 infusion.
Regular monitoring visits during follow-up period
Trial Site Locations
Total: 1 location
1
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Nanning, Guangxi, China
Actively Recruiting
Research Team
X
Xinhua Zhang, MD
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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