Brief Title
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
Official Title
Phase 1 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Ascending Doses of Subcutaneous Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
Brief Summary
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor.
Detailed Description
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor. The study will enroll at least 8 adult male subjects with moderate or severe Hemophilia A or B with or without an inhibitor, in each dosing stage. Each subject will receive escalating doses of MarzAA for each stage of the study (except for Stage 5, where subjects receive the same dose as in Stage 4 split between two anatomical sites).
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last
Secondary Outcome
Comparative MarzAA Activity of Intravenous and Subcutaneous - Cmax
Condition
Hemophilia A
Intervention
MarzAA (marzeptacog alfa [activated])
Study Arms / Comparison Groups
Study Population
Description: MarzAA (Coagulation Factor VIIa variant) 18 µg/kg intravenously (Stage 1) followed by MarzAA 30 µg/kg subcutaneously (SC) (Stage 2), MarzAA 45 µg/kg SC (Stage 3), MarzAA 60 µg/kg SC (Stage 4), MarzAA 2x30 µg/kg SC (Stage 5), MarzAA 90 µg/kg SC (Stage 6), MarzAA 120 µg/kg SC (Stage 7), MarzAA 2×60 µg/kg SC (Stage 8), MarzAA 3x60 µg/kg SC (Stage 9)
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
11
Start Date
September 24, 2019
Completion Date
June 17, 2020
Primary Completion Date
April 30, 2020
Eligibility Criteria
Inclusion Criteria: - Moderate or severe congenital Hemophilia A or B, with or without an inhibitor - Male, age 18 or older - Affirmation of informed consent with signature confirmation before any trial related activities Exclusion Criteria: - Inability to discontinue and washout prophylaxis treatment 72 hours prior to dosing. - Previous participation in a trial involving SC Administration of rFVIIa or any trial using a modified amino-acid sequence FVIIa - Known positive antibody to FVII or FVIIa detected by central laboratory at screening - Have a coagulation disorder other than hemophilia A or B, with or without an inhibitor - Significant contraindication to participate
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Howard Levy, MD, PhD, MMM, ,
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT04072237
Organization ID
MAA-102
Responsible Party
Sponsor
Study Sponsor
Catalyst Biosciences
Study Sponsor
Howard Levy, MD, PhD, MMM, Study Director, Sponsor GmbH
Verification Date
September 2021