EUCTR2020-003371-18-IT
Active, not recruiting
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 - MAA-202
ConditionsFactor VII deficiency, Glanzmann thrombasthenia (GT) and Hemophilia A with inhibitors on emicizumab prophylaxis (HAwI-E)MedDRA version: 20.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.0Level: PTClassification code 10016079Term: Factor VII deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Factor VII deficiency, Glanzmann thrombasthenia (GT) and Hemophilia A with inhibitors on emicizumab prophylaxis (HAwI-E)
- Sponsor
- Catalyst Biosciences, Inc.
- Enrollment
- 24
- Status
- Active, not recruiting
- Last Updated
- 3 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\) Confirmed diagnosis of cohort:
- •a) Confirmed diagnosis of congential FVIID
- •b) Confirmed diagnosis of congenital GT (ie, platelet function analyzer, mutational analysis)
- •c) Confirmed diagnosis of congenital HAwI\-E treated with the same dose of emicizumab for at least 4 weeks and with one of the following:
- •i) Titer \=5 BU
- •ii) Titer \=0\.6 BU but expected to have a high anamnestic response to FVIII, as demonstrated from the subject’s medical history, precluding the use of FVIII products to treat bleeding as documented by the Investigator
- •iii) Titer \=0\.6 BU but expected to be refractory to increased dosing of FVIII, as demonstrated from the subject’s medical history, precluding the use of FVIII products to treat bleeding as documented by in the Investigator
- •Note: documentation of highest historic titer should be recorded.
- •2\) History of bleeding with an
- •a) ABR of \=8 for FVIID
Exclusion Criteria
- •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\.
- •Note: Prior participation in a study of intravenous (IV) LR769, rFVIIa\-FP (CSL689\), or MarzAA is permissible
Outcomes
Primary Outcomes
Not specified
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