Gene Therapy for Chinese Hemophilia B (NCT04135300) | Clinical Trial Compass
CompletedNot Applicable
Gene Therapy for Chinese Hemophilia B
China10 participantsStarted 2019-10-16
Plain-language summary
GT2019001 is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ā¤2IU/dl residual FIX levels. 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.
Who can participate
Age range18 Years
SexMALE
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Inclusion criteria
ā. Be able to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations;
ā. Be male and ā„18 years of age;
ā. Have hemophilia B with ā¤2 IU/dL (ā¤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is \>2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ā¤2% FIX coagulant activity (FIX:C) ;
ā. Have had ā„100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subject's record/history;
ā. a. Prophylaxis subjects: have had bleeding events and/or infusions with FIX protein products during the last 12 weeks documented in the subjects' medical records; OR b. On-demand subjects: have had ā„4 bleeding events in the last 52 weeks and/or chronic hemophilic arthropathy (pain, joint destruction, and loss of range of motion) in one or more joints;
ā. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
ā. Have no measurable FIX inhibitor as assessed by laboratory; or documented no prior history of FIX inhibitor after 50 EDs (family history of inhibitors will not exclude the subject) and no clinical signs or symptoms of decreased response to FIX administration;
ā. Have acceptable laboratory values:
Exclusion criteria
What they're measuring
1
Incidence of treatment- related adverse events
Timeframe: Infusion to the end of study, average 1 year.
2
Change from baseline alanine aminotransferase ans aspartate amino transferase
Timeframe: At multiple timepoints from pre-dose through up to 1 years post-dose
3
Antibody against AAV capsid protein
Timeframe: from screening through up to 1 years
Trial details
NCT IDNCT04135300
SponsorInstitute of Hematology & Blood Diseases Hospital, China
ā. Have active hepatitis B or C, and HBsAg, hepatitis B core antibody, hepatitis B virus-DNA positivity or hepatitis C virus-RNA viral load positivity, respectively. Negative viral assays in two samples, collected at least six months apart, will be required to be considered negative. Both natural clearers and those who have cleared hepatitis C virus on antiviral therapy are eligible;
ā. Currently on antiviral therapy for hepatitis B or C;
ā. Have significant underlying liver disease, as defined by a preexisting diagnosis of portal hypertension, splenomegaly, encephalopathy, reduction below normal limits of serum albumin or evidence of significant liver fibrosis (fibrosis stage ā„ 3) within the past 6 months prior to or at Screening as determined by any of the following diagnostic modalities: AST-to-Platelet Ratio Index (APRI) \>1;
ā. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ā¤200/mm3. Subjects who are HIV-positive and stable, with an adequate CD4 count (\>200/mm3) and undetectable viral load (\<50 gc/mL) measured twice in the six months prior to enrollment, on an antiretroviral drug regimen are eligible to enroll;
ā. Have anti-BBM-H901 neutralizing antibody titers ā„1:5;
ā. Have history of chronic infection or other chronic disease that the Investigator considers to constitute an unacceptable risk;
ā. Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the last 12 weeks;
ā. Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study;