Factor VII DeficiencyPhase 1/2NCT04548791
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
A Phase 1/2 study testing Coagulation Factor VIIa variant in people with Factor VII Deficiency and Glanzmann Thrombasthenia, run by Catalyst Biosciences at 20 sites in 5 countries.
Trial summary
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
- What is being tested?
- Coagulation Factor VIIa variant
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 20 sitesin 5 countries
- Study dates
- 17 May 2021to 3 Dec 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of cohort: FVII deficiency, Glanzmann Thrombasthenia, or hemophilia A with inhibitors
- Male or female, age 12 or older
- History of frequent bleeding episodes
- Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study related activities
- Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
Who cannot join
- Genotype of FVIID subjects with identified mutations by central lab at screening
- Previous participation in a clinical trial evaluating a modified rFVIIa agent
- Received an investigational drug within 30 days or 5 half-lives or absence of clinical effect, whichever is longer
- Known hypersensitivity to trial or related product
- Known positive antibody to FVII or FVIIa detected by central lab at screening
- Be immunosuppressed
- Significant contraindication to participate
Trial locations and contacts
20 sites in 5 countries.
20 sites in 5 countries
United States7 sites
University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
Rush University
Chicago, Illinois, 60612
Children's Hospital of Michigan
Detroit, Michigan, 48201
Michigan State University Center for Bleeding Disorders & Clotting Disorders
East Lansing, Michigan, 49805
East Carolina University
Greenville, North Carolina, 27858
UC Davis Medical Center
Sacramento, California, 95817
University of California -San Francisco
San Francisco, California, 94143
India5 sites
Mazumdar Shaw Medical Centre
Bengaluru
St. John's Medical College Hospital
Bengaluru
Amrita Institute of Medical Sciences and Research Centre
Kochi
K. J. Somaiya Hospital and Research Centre
Mumbai
Sahyadri Super Speciality Hospital
Pune
Italy5 sites
Careggi University Hospital
Florence
Center for Thrombosis and Haemorrhagic Diseases
Milan
Maggiore Polyclinic Hospital, IRCCS Ca' Granda
Milan
Children's Hospital BambiNo Gesù, IRCCS (PEDS)
Roma
City of Health and Science of Turin
Turin
Russia2 sites
Territorial Clinical Hospital
Barnaul
National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation
Moscow
Ukraine1 site
Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology
Lviv
About this study
Official title: 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
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
- Being tested
- Coagulation Factor VIIa variant
- Medical subject headings
- Factor VII Deficiency, Thrombasthenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Coagulation Factor VIIa variantBiological
Also called MarzAA
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 10 μg/kg, 20 μg/kg, 30 μg/kg, 40 μg/kg, and 60 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 20 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis. Receives: Biological: Coagulation Factor VIIa variant |
| Cohort 2 | Experimental | For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis. Receives: Biological: Coagulation Factor VIIa variant |
| Cohort 3 | Experimental | For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis. Receives: Biological: Coagulation Factor VIIa variant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Measured: Dosing period for each stage in a cohort will be approximately 5 to 11 days
Bleeding episode treatment success
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment
Measured: 24 hours after the first administration of study drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2020
- Study start17 May 2021
- Primary completion15 Nov 2021
- Study completion3 Dec 2021
Why it stopped: Company decision (not a safety issue)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Factor VII DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catalyst Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04548791
- Sponsor's protocol ID
- MAA-202
- Status last verified
- Dec 2021
- First submitted
- 8 Sep 2020
- Last updated
- 21 Dec 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04548791
- Acronym / sponsor ID
- MAA-202
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company decision (not a safety issue)
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Catalyst BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 17 May 2021 (actual)
- Primary completion
- 15 Nov 2021 (actual)
- Study completion
- 3 Dec 2021 (actual)
- First posted
- 16 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: Coagulation Factor VIIa variantFor Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 10 μg/kg, 20 μg/kg, 30 μg/kg, 40 μg/kg, and 60 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 20 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis. |
| Cohort 2 | Experimental | Biological: Coagulation Factor VIIa variantFor Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis. |
| Cohort 3 | Experimental | Biological: Coagulation Factor VIIa variantFor Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of cohort: FVII deficiency, Glanzmann Thrombasthenia, or hemophilia A with inhibitors
- Male or female, age 12 or older
- History of frequent bleeding episodes
- Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study related activities
- Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
Key exclusion criteria (7 listed)
- Genotype of FVIID subjects with identified mutations by central lab at screening
- Previous participation in a clinical trial evaluating a modified rFVIIa agent
- Received an investigational drug within 30 days or 5 half-lives or absence of clinical effect, whichever is longer
- Known hypersensitivity to trial or related product
- Known positive antibody to FVII or FVIIa detected by central lab at screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Coagulation Factor VIIa variantAlso: MarzAASingle intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes | Biological | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Time frame: Dosing period for each stage in a cohort will be approximately 5 to 11 days
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Bleeding episode treatment success
Time frame: 24 hours after the first administration of study drug
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment
Results and safety
The study was stopped early (reason given: Company decision (not a safety issue)). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Coagulation Factor VIIa variant in people with Factor VII Deficiency and Glanzmann Thrombasthenia, run by Catalyst Biosciences at 20 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 21 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, India, Italy, Russia, Ukraine. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Company decision (not a safety issue)). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose (time frame: Dosing period for each stage in a cohort will be approximately 5 to 11 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Dec 2021.