NCT06107400

Brief Summary

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for early_phase_1

Timeline
6mo left

Started Oct 2023

Typical duration for early_phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress84%
Oct 2023Oct 2026

Study Start

First participant enrolled

October 8, 2023

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

October 25, 2023

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 30, 2023

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2025

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2026

Expected
Last Updated

June 3, 2024

Status Verified

October 1, 2023

Enrollment Period

2.1 years

First QC Date

October 25, 2023

Last Update Submit

May 30, 2024

Conditions

Alpha Thalassemia Hemoglobin H Constant SpringHemoglobinopathiesHereditary Diseases

Keywords

RM-004Hemoglobin H-Constant Spring diseaseAlpha-ThalassemiaGene therapyCBE

Outcome Measures

Primary Outcomes (3)

  • 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

  • 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

  • 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 Outcomes (3)

  • Incidence of all-cause mortality

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

  • Proportion of subjects who stop receiveing transfusion ≥ 6 months

    Up to 24 months after RM-004 infusion

  • Duration of transfusion independence

    Up to 24 months after RM-004 infusion

Study Arms (1)

RM-004

EXPERIMENTAL

RM-004 (ex vivo gene-edited CD34+ autologous hematopoietic stem cells) will be administered as a single intravenous infusion

Genetic: RM-004

Interventions

RM-004GENETIC

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

RM-004

Eligibility Criteria

Age12 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Nanning, Guangxi, China

RECRUITING

MeSH Terms

Conditions

HemoglobinopathiesGenetic Diseases, Inbornalpha-Thalassemia

Condition Hierarchy (Ancestors)

Hematologic DiseasesHemic and Lymphatic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemia

Study Officials

  • Xinhua Zhang, MD

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

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Xinhua Zhang, MD

CONTACT

+8613321717386zxh303@163.com

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 25, 2023

First Posted

October 30, 2023

Study Start

October 8, 2023

Primary Completion

October 31, 2025

Study Completion (Estimated)

October 31, 2026

Last Updated

June 3, 2024

Record last verified: 2023-10

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)