Gene Therapy for Chinese Hemophilia B
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|ClinicalTrials.gov Identifier: NCT04135300|
Recruitment Status : Recruiting
First Posted : October 22, 2019
Last Update Posted : July 31, 2020
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Genetic: Single dose intravenous injection of BBM-H901||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Hemophilia B patients|
|Masking:||None (Open Label)|
|Official Title:||Gene Therapy for Chinese Hemophilia B With Adeno-associated Virus (AAV) Vector|
|Actual Study Start Date :||October 16, 2019|
|Estimated Primary Completion Date :||November 2021|
|Estimated Study Completion Date :||November 2021|
Experimental: Arm of BBM-H901
Subjects will be dosed with single dose of BBM-H901 at 5x10·12 vg/kg via intravenous infusion.
Genetic: Single dose intravenous injection of BBM-H901
Single dose intravenous infusion of BBM-H901, an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene in liver. The dose of BBM-H901 will be 5x10'12 vg/Kg.
- Incidence of treatment- related adverse events [ Time Frame: Infusion to the end of study, average 1 year. ]Number of patients experiencing treatment-related adverse events. Including inhibitor development.
- Change from baseline alanine aminotransferase ans aspartate amino transferase [ Time Frame: At multiple timepoints from pre-dose through up to 1 years post-dose ]liver function tests include ALT, AST.
- Antibody against AAV capsid protein [ Time Frame: from screening through up to 1 years ]Immune response against AAV capsid will be evaluated by measurement of the total antibody and neutralizing antibody against AAV capsid protein in plasma samples collected at multiple timepoints after dosing up to 1 year.
- Vector- derived FIX:C and FIX antigen levels. [ Time Frame: At multiple timepoints from pre-dose through up to 1 years post-dose ]Vector- derived FIX:C and FIX antigen levels will be measured after dosing.
- Vector shedding of BBM-H901 [ Time Frame: From date of infusion until the date of 3 consecutive documented negative results, assessed up to 1 year ]Serum and semen will be collected to assess clearance of vector genomes
- annualized bleeding rate changes from baseline [ Time Frame: through study completion, an average of 1 year ]annualized bleeding rate changes from baseline
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04135300
|Contact: Feng XUE, MDfirstname.lastname@example.org|
|Contact: lei zhang, MDemail@example.com|
|Institute of Hematology & Blood Diseases Hospital||Recruiting|
|Tianjin, Tianjin, China, 300020|
|Contact: Lei Zhang, MD firstname.lastname@example.org|
|Principal Investigator:||Lei Zhang, MD||Institute of Hematology & Blood Diseases Hospital|