Clinical trial NCT06107400: Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease
Official title: Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: EARLY_PHASE1
Conditions: Alpha Thalassemia Hemoglobin H Constant Spring
Hemoglobinopathies
Hereditary Diseases
Keywords: RM-004
Hemoglobin H-Constant Spring disease
Alpha-Thalassemia
Gene therapy
CBE
Lead sponsor: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Sponsor class: OTHER
Collaborators: name: Guangzhou Reforgene Medicine Co., Ltd.
First posted: 2023-10-30
Enrollment: 5
Sex: ALL
Minimum age (years): 12
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.

Detailed description: This is a non-randomized, one-arm, open label study to evaluate the safety and efficacy of RM-004 for autologous hematopoietic stem cell transplantation (HSCT) for the treatment of Hemoglobin H-Constant Spring disease. Five subjects aged from 12 to 35 years will be recruited in this study.

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.

Primary outcomes:
measure: Proportion of subjects with engraftment
timeFrame: Within 42 days after RM-004 infusion
measure: Safety of RM-004 infusion
timeFrame: From signing of informed consent up to 24 months after RM-004 infusion
measure: Proportion of subjects who achieve transfusion independence
timeFrame: Up to 24 months after RM-004 infusion
Secondary outcomes:
measure: Incidence of all-cause mortality
timeFrame: From signing of informed consent up to 24 months after RM-004 infusion
measure: Proportion of subjects who stop receiveing transfusion ≥ 6 months
timeFrame: Up to 24 months after RM-004 infusion
measure: Duration of transfusion independence
timeFrame: Up to 24 months after RM-004 infusion
Other outcomes:
Locations:
city: Nanning
country: China
facility: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
state: Guangxi
Study officials:
affiliation: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
name: Xinhua Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: