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Clinical Trials/CTRI/2021/03/032089
CTRI/2021/03/032089
Other
Phase 1

Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog alfa (activated) in Treatment of Episodic Bleeding in Subjects with Inherited Bleeding Disorders.

Catalyst Biosciences Inc0 sites8 target enrollmentTBD
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Overview

Phase
Phase 1
Intervention
Not specified
Conditions
Not specified
Sponsor
Catalyst Biosciences Inc
Enrollment
8
Status
Other
Last Updated
3 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
TBD
End Date
December 6, 2021
Last Updated
3 years ago
Study Type
Interventional

Investigators

Eligibility Criteria

Inclusion Criteria

  • •Study candidates must meet all the following inclusion criteria to be eligible for participation in this study:
  • •1\) Confirmed diagnosis of cohort:
  • •a) Confirmed diagnosis of congential Factor VII deficiency (FVIID)
  • •b) Confirmed diagnosis of congenital Glanzmann thrombasthenia (GT) (ie, platelet function analyzer, mutational analysis)
  • •c) Confirmed diagnosis of congenital Hemophilia A with inhibitors on emicizumab (HAwI\-E) treated with the same dose of emicizumab
  • •2\) History of bleeding with an (a) Annualized bleeding rate (ABR) of ââ?°Â¥8 for FVIID. (b) Annualized bleeding rate (ABR) of ââ?°Â¥8 for GT. (c) Annualized bleeding rate (ABR) of ââ?°Â¥1 for HAwI\-E
  • •3\) Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
  • •4\) If female, the subject must meet the following criteria (a) Not currently be breastfeeding (b) Not plan on becoming pregnant during the study. (c) Be surgically sterile, or at least 2 years postmenopausal, or have a negative serum pregnancy test during Screening.
  • •5\) Subjectââ?¬•s ability to rapidly assess a bleeding episode and respond appropriately
  • •6\) Affirmation of informed consent with signature confirmation and assent for children from age 12 to 17 years before any study\-related activities

Exclusion Criteria

  • •Subjects who meet any of the following criteria will not be eligible for participation in this study:
  • •1\) Cohort 1: genotype of FVIID subjects with following mutations:
  • •a) P.A354V\-p.464Hfs
  • •b) P.Ser112\-Stop (homozygous)
  • •c) Ala294Val \+ Del C
  • •d) 100GLN ARG shift
  • •e) Ser103 Gly
  • •Note: documentation of historic genotype would be acceptable.
  • •2\) Inability to discontinue and washout any prophylactic (except Hemlibra) or episodic treatment for 5 days and 10 days for platelet transfusion prior to dosing
  • •3\) Previous participation in a clinical study involving SC administration of wt\-rFVIIa (NovoSeven or MOD\-5014\) or any study using a modified amino\-acid sequence FVIIa (other than MarzAA) such as: NN1731 or BAY86\-6150\.

Outcomes

Primary Outcomes

Not specified

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