Evaluating RM-004 Cells for Treating Hemoglobin H-Constant Spring Disease

Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease

This study is testing a new stem cell treatment to see if it can help young people with Hemoglobin H-Constant Spring disease feel better and stay safe.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Ages: 12 Years to 35 Years
• Sex: All
• Sponsor: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army (Academic / other)
• Locations: 1 site (Nanning, Guangxi)
• Trial ID: NCT06107400 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and efficacy of RM-004, an autologous hematopoietic stem cell transplantation therapy, for patients with Hemoglobin H-Constant Spring disease. It is a non-randomized, one-arm, open-label trial that will recruit five subjects aged 12 to 35 years. Participants will be monitored for their response to the treatment and any potential side effects throughout the study period.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 to 35 years with a diagnosis of Hemoglobin H-Constant Spring disease and a history of significant blood transfusions.

Not a fit: Patients who have an available HLA-matched donor for allogeneic HSCT or those who have previously undergone HSCT or gene therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the need for blood transfusions in patients with Hemoglobin H-Constant Spring disease.

How similar studies have performed: While gene therapy approaches for hemoglobinopathies are being explored, this specific application of RM-004 is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

1. Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment.
2. At the time of screening, subjects should be aged from 12 to 35 years old, regardless of gender.
3. History of at least 100 mL/kilograms (kg)/year of packed red blood cells (pRBC) transfusions in the prior 2 years before screening.
4. Subjects diagnosed with Hemoglobin H-Constant Spring disease (--/ααCS) with HBA2 c.427T\>C mutation.

Exclusion Criteria:

1. Subject who has an available HLA-matched/well-matched HSCT donor for allogeneic hematopoietic stem cell transplantation (HSCT).
2. Prior HSCT or gene therapy.
3. History of severe hemorrhagic disease.
4. Clinically significant active bacterial, viral, fungal or parasitic infections per investigator's judgement at the time of screening.

Where this trial is running

Nanning, Guangxi

• The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army — Nanning, Guangxi, China (Recruiting)

Study contacts

• Principal investigator: Xinhua Zhang, MD — The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
• Study coordinator: Xinhua Zhang, MD

Conditions Alpha Thalassemia Hemoglobin H Constant Spring; Hemoglobinopathies; Hereditary Diseases; RM-004; Hemoglobin H-Constant Spring disease; Alpha-Thalassemia; Gene therapy; CBE