Actively Recruiting
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2023-10-17
2
Participants Needed
1
Research Sites
147 weeks
Total Duration
On this page
Sponsors
I
Institute of Hematology & Blood Diseases Hospital, China
Lead Sponsor
S
Shanghai Vitalgen BioPharma Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
CONDITIONS
Official Title
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 3-35 years old, male or female
- Signed informed consent by the participant or legal guardian
- Clinical diagnosis of transfusion-dependent beta-thalassemia with blood transfusion records showing at least 8 times per year or 10 units/kg/year in the past 2 years
- Karnofsky score (if 16 years or older) or Lansky score (if younger than 16 years) of 80 or higher
- Stable disease eligible for hematopoietic stem cell transplantation as judged by the investigator
- Access to diagnosis and treatment records from medical institutions within the past 2 years
- Willing and able to comply with study procedures and complete at least 2 years of follow-up
- Use of effective contraception for subjects of childbearing potential within 12 months of treatment
You will not qualify if you...
- Diagnosis of associated alpha-thalassemia with more than 1 alpha chain deletion or alpha gene defect
- Availability of fully matched HLA donors suitable for allogeneic hematopoietic stem cell transplantation
- Positive for irregular or platelet antibodies
- Prior allogeneic bone marrow transplantation or gene therapy
- Clinically significant and active infections including HIV, HCMV, EBV, syphilis, or prior hepatitis B or C infection
- Major organ injuries
- Contraindications for hematopoietic stem cell collection or poor collection efficiency
- Contraindications to investigational product, G-CSF, plerixafor, busulfan, or other related drugs
- Participation in another interventional clinical study within 3 months prior to screening
- History or family history of malignancy or myeloproliferative disorders
- History of uncontrolled epilepsy, mental or psychiatric disorders
- Abuse of psychoactive substances, drugs, or alcohol within 6 months prior to enrollment
- Pregnant or breastfeeding females
- Any other disease or condition interfering with study procedures or deemed unsuitable by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
Actively Recruiting
Research Team
J
Jun Shi, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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