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

Hemoglobinopathies Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06107400
• Other study ID #: IIT-RM-004-01
• Status: Recruiting
• Phase: Early Phase 1
• Start date: October 8, 2023
• Est. completion date: October 31, 2026

Study information

• Verified date: October 2023
• Source: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
• Contact: Xinhua Zhang, MD
• Phone: +8613321717386
• Email: zxh303[@]163.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: October 31, 2026
• Est. primary completion date: October 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 35 Years

Eligibility

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 (--/aaCS) 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.

Related Conditions & MeSH terms

• alpha-Thalassemia
• Genetic Diseases, Inborn
• Hemoglobinopathies
• Hereditary Diseases
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
China	The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army	Nanning	Guangxi

Sponsors (2)

Lead Sponsor	Collaborator
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army	Guangzhou Reforgene Medicine Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Within 42 days after RM-004 infusion
Primary	Safety of RM-004 infusion	Number of subjects with adverse events (AEs) and serious adverse events (SAEs)	From signing of informed consent up to 24 months after RM-004 infusion
Primary	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.	Up to 24 months after RM-004 infusion
Secondary	Incidence of all-cause mortality	Incidence of all-cause mortality	From signing of informed consent up to 24 months after RM-004 infusion
Secondary	Proportion of subjects who stop receiveing transfusion = 6 months	Proportion of subjects who stop receiveing transfusion = 6 months	Up to 24 months after RM-004 infusion
Secondary	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	Up to 24 months after RM-004 infusion