Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Early Phase 1RecruitingNCT06107400

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army5 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Alpha Thalassemia Hemoglobin H Constant Spring Hemoglobinopathies Hereditary Diseases

Interventions

RM-004GENETIC

Intravenous infusion of gene-edited CD34+autologous hematopoietic stem cells (RM-004) after myeloablative conditioning with busulfan.

Eligibility

Sex: ALLMin age: 12 YearsMax age: 35 Years

Medical Language ↔ Plain English

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.

Locations (1)

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

Nanning, Guangxi, China

RECRUITING

Outcomes

Primary Outcomes

Proportion of subjects with engraftment

Proportion of subjects with successful neutrophil engraftment (first day of 3 consecutive measurements of absolute neutrophil count \[ANC\] ≥0.5×10\^9/L on 3 different days) after RM-004 infusion.

Time frame: Within 42 days after RM-004 infusion

Safety of RM-004 infusion

Number of subjects with adverse events (AEs) and serious adverse events (SAEs)

Time frame: From signing of informed consent up to 24 months after RM-004 infusion

Proportion of subjects who achieve transfusion independence

Transfusion independence (TI) was defined as a weighted average hemoglobin (Hb) ≥ 9 g/dL without any red blood cells transfusions for a continuous period of ≥12 months at any time during the study after RM-004 infusion.

Time frame: Up to 24 months after RM-004 infusion

Secondary Outcomes

Incidence of all-cause mortality

Time frame: From signing of informed consent up to 24 months after RM-004 infusion

Proportion of subjects who stop receiveing transfusion ≥ 6 months

Time frame: Up to 24 months after RM-004 infusion

Duration of transfusion independence

Duration of TI was calculated as the time from the start of TI up to the last available Hb at which the TI criteria are still met. Time period of TI will start when subjects achieve a Hb ≥ 9 g/dL with no transfusions in the preceding 60 days

Time frame: Up to 24 months after RM-004 infusion

Central Contacts

Xinhua Zhang, MD

CONTACT

+8613321717386 zxh303@163.com

Data from ClinicalTrials.gov

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