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Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexMale
Age18+
SponsorShanghai Xinzhi BioMed Co., Ltd.

About this trial

This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.

BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Subjects voluntarily sign informed consent form;

Males ≥ 18 years;

Subjects are clinically diagnosed with severe hemophilia A;

Have > = 150 documented exposure days to a Factor VIII protein product

Disqualifiers

Being positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus-DNA (HBV-DNA). Being positive for hepatitis C virus antibody (HCV-Ab) or hepatitis C virus RNA (HCV-RNA).

Currently on antiviral therapy for hepatitis B or C;

Suffer from coagulation disorders other than hemophilia A;

In addition to glucocorticoids, any other immunosuppressants are being used before selection;

Trial design

Design model

Single group

Treatments tested in this trial

  • Single dose intravenous injection of BBM-H803

    Genetic

    Single dose intravenous infusion of BBM-H803, an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene in liver. The dose of BBM-H803 will be calculated according to participant's weight.

Treatment groups

55 Participants
are divided into 1 treatment group
Group A: Arm of BBM-H803Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Phase 1/2: Incidence of dose limiting toxicity (DLT) events

To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-H803 injection infusion

Time frame
6 weeks
2

Phase 1/2: The incidence of adverse events (AEs) and serious adverse events (SAEs)

To assess the safety of BBM-H803 Injection by AEs and SAEs.

Time frame
6 weeks
3

Phase 1/2: Number of participants with changes in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels [liver function]

To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST).

Time frame
6 weeks
4

Phase 3: Annualized bleeding rate (ABR)

To assess ABR, including spontaneous bleeding, traumatic bleeding and joint bleeding after administration.

Time frame
52 weeks

Secondary outcomes

1

Phase 1/2/3: Plasma FVIII activity level

All samples collected from participants for plasma FVIII activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FVIII activity levels.

Time frame
52 weeks
2

Phase 1/2/3: The incidence of adverse events (AEs) and serious adverse events (SAEs)

To assess the safety of BBM-H803 Injection by AEs and SAEs.

Time frame
52 weeks
3

Phase 1/2/3: Number of participants with changes in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels [liver function]

To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT)and aspartate aminotransferase (AST).

Time frame
52 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shanghai Xinzhi BioMed Co., Ltd.

Lead sponsor

Shanghai Mianyi Biopharmaceutical Co., Ltd.

Collaborator

Source ClinicalTrials.gov
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