iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

NCT03728322 | Unknown Early Phase 1

• 1 other identifier: HBB HSC-01
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.

Conditions

Thalassemia

Outcome Measures

Primary Outcomes (1)

• Occurrence of treatment related adverse events as assessed by CTCAE v4.0

  Defined as \>= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment

  1 year

Study Arms (1)

iHSCs treatment group

EXPERIMENTAL

Biological: iHSCs treatment group

Interventions

iHSCs treatment group BIOLOGICAL

iHSCs intravenous injection

iHSCs treatment group

Eligibility Criteria

• Age: 2 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects ≥ 2 and ≤ 60 years of age
• Subjects was confirmed the transfusion-dependent β-thalassemia
• Adequate organ function, as defined by:
• Serum creatinine ≤ 1.5 mg/dl ； Serum ALT/AST）≤2.5×ULN；ALB≥25g/L； Serum total bilirubin \< 1.5x ULN Left ventricular ejection fraction≥50%
• Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases
• Subjects survival was expected≥6 months
• Adult patients were willing to use reliable contraceptives (such as condoms) and not to donate sperm throughout the study period and within three months of discharge
• Subjects and the guardians able to undergo post-physical therapy/rehabilitation

You may not qualify if:

• Subjects allergic to macromolecular biological agents such as antibodies or cytokines
• Subjects receipt of any investigational clinical trials within 3 months.
• Subjects previous treatment with any hematopoietic stem cell transplantation or other organ transplantation
• Uncontrolled bleeding symptoms
• Severe cardiovascular disease is known, including any of the following:
• Myocardial infarction or thrombosis has occurred in the past six months Subjects with unstable angina pectoris Subjects with Class III/IV cardiovascular disability according to the New York Heart Association Classification
• Subjects have one kinds of tumors within 5 years
• Active hepatitis B (HBV DNA\>1000copy/mL), hepatitis C or HIV infection.
• Subjects have an infectious diseases that cannot be controlled within 4 weeks
• subjects have severe central nervous system disease or epilepsy
• Subjects are Suffering from mental illness; Patients with alcohol dependence, drug abuse, drug addiction, and medical, psychological or social conditions that may interfere with research or have an impact on the evaluation of research results
• Women in pregnancy (positive urine/blood pregnancy test) or lactation
• Subjects who have other conditions that were not appropriate for the group determined by the researchers.

Sponsors & Collaborators

• Allife Medical Science and Technology Co., Ltd.: lead

MeSH Terms

Conditions

Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: early phase 1
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 31, 2018
• First Posted: November 2, 2018
• Study Start: January 1, 2019
• Primary Completion: January 1, 2020
• Study Completion: January 1, 2021
• Last Updated: November 5, 2018

Record last verified: 2018-10