BridgeMD
Tests treatment safety and results for Alpha Thalassemia Hemoglobin H Constant Spring
Official title Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease
ClinicalTrials.gov ID: NCT06107400
What this study is testing
What is RM-004?
RM-004 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for alpha thalassemia hemoglobin h constant spring.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 35
You may be able to join if
- people voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and...
- At the time of screening, people 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.
- people diagnosed with Hemoglobin H-Constant Spring disease (--/ααCS) with HBA2 c.427T\>C mutation.
You likely can't join 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.
The study team makes the final eligibility decision.
Where it's taking place
- Nanning, Guangxi, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Nanning, Guangxi, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.