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

Status Recruiting

Clinical Trial Summary

Clinical Trial Description

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. Nine subjects will be enrolled and administered with single infusion of BBM-H901, an AAV at one dose level of 5x10'12 vg/Kg. Subjects and statutory guardian must provide informed consent and then undergo screening assessments up to 4-8weeks prior administration of BBM-H901. All subjects will undergo 52(+- 2) weeks safety observation and will be continuously followed up to evaluate long- term safety and efficacy of BBM-H901 up to ten years. The first subject will be dosed at 5x10'12 vg/Kg and undergo 8 weeks safety observation of which the data will undergo review by an independent safety committee. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT05709288
Study type	Interventional
Source	Institute of Hematology & Blood Diseases Hospital, China
Contact	Feng Xue, MD
Phone	+862223909240
Email	xuefeng@ihcams.ac.cn
Status	Recruiting
Phase	Phase 1
Start date	March 23, 2023
Completion date	November 2035

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04072237` - Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia	Phase 1
Completed	`NCT02199717` - An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia	N/A
Completed	`NCT01662531` - A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B	Phase 3
Completed	`NCT01335061` - Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B	Phase 3
Completed	`NCT01217255` - Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Completed	`NCT00037557` - Study Evaluating rFIX; BeneFIX in Severe Hemophilia B	Phase 3
Completed	`NCT02554773` - An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B	Phase 1/Phase 2
Terminated	`NCT02807753` - The Hemophilia Ultrasound Project
Active, not recruiting	`NCT03901755` - An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
Not yet recruiting	`NCT05980377` - Patterns of Hemophilia Care in Assiut Children Patients
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Terminated	`NCT03248141` - Understanding Hemophilia A and B Drug Dosage Administration Patterns
Terminated	`NCT01460147` - Osteoporosis and MRI Study in Hemophilia	N/A
Completed	`NCT03818529` - ATHN 8: Previously Untreated Patients (PUPs) Matter Study
Completed	`NCT02571569` - A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors	Phase 1
Terminated	`NCT01620801` - Hemophilia B Gene Therapy With AAV8 Vector	Phase 1
Completed	`NCT01233440` - Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B	Phase 1
Active, not recruiting	`NCT04135300` - Gene Therapy for Chinese Hemophilia B	N/A
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Terminated	`NCT00947193` - Study of Ataluren (PTC124) in Hemophilia A and B	Phase 2