A Phase I Clinical Trial to Evaluate Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (RM-001) in Transfusion-Dependent beta Thalassemia Patients

A Phase I Clinical Trial to Evaluate Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (RM-001) in Transfusion-Dependent beta-Thalassemia Patients

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

TCTR

Registry ID

TCTR20250828001

Enrollment

Registered

2025-08-28

Start date

2026-01-01

Completion date

Unknown

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transfusion-Dependent beta-Thalassemia Thalassemia

Interventions

Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells

Experimental Genetic

RM001

Eligibility

Sex/Gender

All

Age

6 Years to 35 Years

Inclusion criteria

Inclusion criteria: 1.Full understanding and voluntary signing of informed consent (signed by themselves or their legal guardians). 2.Age 6 to 35 years. 3.Diagnosed with transfusion-dependent Beta-thalassemia (TDT), also known as beta-thalassemia major, with a history of at least 100 mL/kg/year of PRBC transfusions or 8 times of PRBC transfusions per year in the 2 years prior to the consent. 4.No available 10/10 human leukocyte antigen (HLA)-matched related donor. 5. Patients have no access to or are deemed unsuitable for haploidentical transplantation by the study physician. 6. Lansky score (for subjects less than16 years) / Karnofsky score (for subjects more than16 years) more than 80. 7. Subjects assessed by investigators as suitable candidates for HSCT.

Exclusion criteria

Exclusion criteria: An available 10/10 HLA-matched related donor. 2. Prior allogeneic HSCT. 3. Patients with positive results in any of the following pathogen tests: human immunodeficiency virus (HIV-1/2), human cytomegalovirus (HCMV), Epstein-Barr virus (EBV), COVID-19, hepatitis B surface antigen (HBsAg), hepatitis B e-antigen (HBeAg), hepatitis B e-antibody (HBeAb), hepatitis B core antibody (HBcAb), hepatitis C virus antibody (HCV-Ab), and anti-Treponema pallidum antibody (TP-Ab). 4. Clinically significant and active bacterial or fungal infections as determined by the investigator. 5. Coagulation abnormalities

Design outcomes

Primary

Measure	Time frame
Safety Endpoints 42 days post dose neutrophil engraftment	—

Secondary

Measure	Time frame
Efficacy Endpoint 12 months post dose of subjects achieving transfusion independence	—

Countries

Thailand

Contacts

Public ContactWinat Khoungtip

Siriraj Hospital

winat.khoungtip@outlook.com0800958889

Outcome results

None listed

Source: TCTR (via WHO ICTRP) · Data processed: Aug 10, 2026