Alpha Thalassemia Hemoglobin H Constant SpringEarly Phase 1NCT06107400
Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease
A Early Phase 1 study testing RM-004 in people with Alpha Thalassemia Hemoglobin H Constant Spring and Hemoglobinopathies, run by The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army at 1 site in China.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease. Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies, Hereditary Diseases
- What is being tested?
- RM-004
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 8 Oct 2023to 31 Oct 2026
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Nanning, Guangxi
Xinhua Zhang, MD, +8613321717386, zxh303@163.com
About this study
Official title: Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of 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.
Read the 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.
- Being tested
- RM-004
- Keywords
- RM-004, Hemoglobin H-Constant Spring disease, Alpha-Thalassemia, Gene therapy, CBE
- Medical subject headings
- Hemoglobinopathies, Genetic Diseases, Inborn, alpha-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RM-004Genetic
Intravenous infusion of gene-edited CD34+autologous hematopoietic stem cells (RM-004) after myeloablative conditioning with busulfan.
| Study group | Role | What they receive |
|---|---|---|
| RM-004 | Experimental | RM-004 (ex vivo gene-edited CD34+ autologous hematopoietic stem cells) will be administered as a single intravenous infusion Receives: Genetic: RM-004 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Within 42 days after RM-004 infusion
Safety of RM-004 infusion
Number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Measured: 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.
Measured: Up to 24 months after RM-004 infusion
Secondary outcomes (3)
Incidence of all-cause mortality
Incidence of all-cause mortality
Measured: From signing of informed consent up to 24 months after RM-004 infusion
Proportion of subjects who stop receiveing transfusion ≥ 6 months
Proportion of subjects who stop receiveing transfusion ≥ 6 months
Measured: 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
Measured: Up to 24 months after RM-004 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2023
- Study start8 Oct 2023
- Primary completion31 Oct 2025 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha Thalassemia Hemoglobin H Constant SpringStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Sponsor type
- University, hospital or other
- Collaborators
- Guangzhou Reforgene Medicine Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Xinhua Zhang, MD, Principal investigator, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06107400
- Sponsor's protocol ID
- IIT-RM-004-01
- Status last verified
- Oct 2023
- First submitted
- 25 Oct 2023
- Last updated
- 3 Jun 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease
- ClinicalTrials.gov ID
- NCT06107400
- Acronym / sponsor ID
- IIT-RM-004-01
- Keywords
- RM-004, Hemoglobin H-Constant Spring disease, Alpha-Thalassemia, Gene therapy, CBE
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation ArmyUniversity, hospital or other
- Collaborators
- Guangzhou Reforgene Medicine Co., Ltd.
- Study start
- 8 Oct 2023 (actual)
- Primary completion
- 31 Oct 2025 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 30 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RM-004 | Experimental | Genetic: RM-004RM-004 (ex vivo gene-edited CD34+ autologous hematopoietic stem cells) will be administered as a single intravenous infusion |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RM-004Intravenous infusion of gene-edited CD34+autologous hematopoietic stem cells (RM-004) after myeloablative conditioning with busulfan. | Genetic | RM-004 |
Primary outcome
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.
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)
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.
3 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
Time frame: Up to 24 months after RM-004 infusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2024.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Xinhua Zhang, MD principal investigator
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing RM-004 in people with Alpha Thalassemia Hemoglobin H Constant Spring and Hemoglobinopathies, run by The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xinhua Zhang, MD (+8613321717386, zxh303@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with engraftment (time frame: Within 42 days after RM-004 infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2024.