Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06107400

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

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

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Alpha Thalassemia Hemoglobin H Constant Spring Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Hereditary Diseases Thalassemia alpha-Thalassemia

Age range

12 year–35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

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

Nanning, Guangxi, China

Location status: Recruiting

Location contact

Xinhua Zhang, MD

CONTACT

[email protected]

+8613321717386

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

RM-004

Genetic

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

Primary outcomes

  1. Proportion of subjects with engraftment

    Time frame: Within 42 days after RM-004 infusion

    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.

  2. Safety of RM-004 infusion

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

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

  3. Proportion of subjects who achieve transfusion independence

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

    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.

Secondary outcomes

  1. Incidence of all-cause mortality

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

    Incidence of all-cause mortality

  2. Proportion of subjects who stop receiveing transfusion ≥ 6 months

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

    Proportion of subjects who stop receiveing transfusion ≥ 6 months

  3. Duration of transfusion independence

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

    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

Study contacts

Contact information is provided by the study sponsor or research team.

Xinhua Zhang, MD

CONTACT

[email protected]

+8613321717386

Sponsors and collaborators

Lead sponsor

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

Other

Collaborators

  • Guangzhou Reforgene Medicine Co., Ltd.

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2025
Study completion
2026
First posted
Oct 30, 2023
Registry last updated
Jun 3, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.