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Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old

NCT05709288 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2025-02-21

No results posted yet for this study

Summary

This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.

Conditions

  • Hemophilia B

Interventions

DRUG

BBM-H901

Single dose intravenous infusion of BBM-H901, an adeno-associated viral (AAV) vector designed to drive expression of an hyper active human factor IX mutant(FIX Padua) transgene in liver. The dose of BBM-H901 is 5x10'12 vg/Kg.

Sponsors & Collaborators

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Principal Investigators

  • Lei Zhang, MD · Insitute of haematology and blood diseases hospital, chinese academy of medical sciences

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-03-23
Primary Completion
2035-06-30
Completion
2035-11-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05709288 on ClinicalTrials.gov