Hemophilia A With InhibitorPhase 3NCT04489537
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B
A Phase 3 study testing MarzAA in people with Hemophilia A With Inhibitor and Hemophilia B With Inhibitor, run by Catalyst Biosciences at 43 sites in 16 countries.
Trial summary
The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
- What is being tested?
- MarzAA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 43 sitesin 16 countries
- Study dates
- 4 May 2021to 1 Dec 2021
- Phase
- Phase 3
- 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 congenital hemophilia A or B 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
Who cannot join
- 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
- Known hypersensitivity to trial or related product
- Known positive antibody to FVII or FVIIa detected by central lab at screening
- Have a coagulation disorder other than hemophilia A or B
- Be immunosuppressed
- Significant contraindication to participate
Trial locations and contacts
43 sites in 16 countries.
43 sites in 16 countries
India8 sites
Nirmal Hospital
Gujrāt
K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre
Kārnād
Sanjay Gandhi Postgraduate Institute of Medical Sciences
Lucknow
K.J Somaiya Hospital and Research Centre
Mumbai
MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre
Mumbai
Sahyadri Specialty Hospital
Mumbai
Grant Medical Foundation, Ruby Hall Clinic
Pune
All India Institute of Medical Sciences, Rishikesh
Rishikesh
Malaysia5 sites
Hospital Ampang
Ampang
Raja Perempuan Zainab II Hospital
Kota Bharu
Queen Elizabeth Hospital Malaysia
Kota Kinabalu
Hospital Tunku Azizah Kuala Lumpur
Kuala Lumpur
Tuanku Ja'afar Hospital, Seremban
Sungai Petani
Italy4 sites
Azienda Ospedaliero-Universitaria Careggi
Florence
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Milan
Azienda Ospedaliera Universitaria Federico II
Naples
Citta della Salute e della Scienza di Torino - Ospedale le Molinette
Torino
Turkey (Türkiye)4 sites
Akdeniz University Medical Faculty Hospital
Antalya
Istanbul University Faculty of Medicine
Istanbul
Ege University Medical Faculty
Izmir
Özel Acibadem Adana Hastanesi
Seyhan
Mexico3 sites
Instituto de Investigaciones Aplicada a la Neurociencia A.C.
Durango
Instituto Nacional de Pediatria
Mexico City
Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.
Mexico City
Ukraine3 sites
"Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology
Cherkasy
"Kyiv City Clinical Hospital #9" of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment
Kyiv
"Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department
Lviv
United Kingdom3 sites
Queen Elizabeth Hospital Birmingham
Birmingham
Royal Liverpool University Hospital
Liverpool
University Hospital Southampton NHS Foundation Trust
Southampton
United States2 sites
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Lombardi Comprehensive Cancer Center
Washington D.C., District of Columbia, 20007
Russia2 sites
Kirov Research Institute of Hematology and Blood Transfusion
Kirov
Medis, Llc
Nizhny Novgorod
Spain2 sites
Hospital Universitario Vall d'Hebron
Barcelona
Hospital de Malaga
Málaga
Taiwan2 sites
Changhua Christian Hospital (CCH)
Changhua
Taichung Veterans General Hospital
Taichung
Georgia1 site
JSC "K.Eristavi National Center of Experimental and Clinical Surgery"
Tbilisi
Hungary1 site
Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ
Budapest
About this study
Official title: Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On Demand Treatment and Control of Bleeding Episodes in Subjects With Hemophilia A or Hemophilia B, With Inhibitors: The Crimson 1 Study
The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC).
- Being tested
- MarzAA
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MarzAABiological
A cross over design to assess the efficacy of a dosing regimen of 60 µg/kg of MarzAA compared with standard of care for the treatment of bleeding episodes.
| Study group | Role | What they receive |
|---|---|---|
| MarzAA | Experimental | Coagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis Receives: Biological: MarzAA |
| Standard of Care | Active comparator | Standard of care administered on-demand during bleeding episodes Receives: Biological: MarzAA |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 compared with standard of care.
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 posted28 Jul 2020
- Study start4 May 2021
- Primary completion15 Nov 2021
- Study completion1 Dec 2021
Why it stopped: Sponsor decision (not a safety decision)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 18 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
- NCT04489537
- Sponsor's protocol ID
- MAA-304
- Status last verified
- Dec 2021
- First submitted
- 24 Jul 2020
- Last updated
- 17 Dec 2021
- Results posted
- No
- Data monitoring committee
- Yes
- 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 17 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On Demand Treatment and Control of Bleeding Episodes in Subjects With Hemophilia A or Hemophilia B, With Inhibitors: The Crimson 1 Study
- ClinicalTrials.gov ID
- NCT04489537
- Acronym / sponsor ID
- MAA-304
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision (not a safety decision)
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Catalyst BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 4 May 2021 (actual)
- Primary completion
- 15 Nov 2021 (actual)
- Study completion
- 1 Dec 2021 (actual)
- First posted
- 28 Jul 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 safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MarzAA | Experimental | Biological: MarzAACoagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis |
| Standard of Care | Active comparator | Biological: MarzAAStandard of care administered on-demand during bleeding episodes |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of congenital hemophilia A or B 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
Key exclusion criteria (7 listed)
- 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
- Known hypersensitivity to trial or related product
- Known positive antibody to FVII or FVIIa detected by central lab at screening
- Have a coagulation disorder other than hemophilia A or B
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 |
|---|---|---|
| MarzAAA cross over design to assess the efficacy of a dosing regimen of 60 µg/kg of MarzAA compared with standard of care for the treatment of bleeding episodes. | Biological | MarzAA, Standard of Care |
Primary outcome
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 compared with standard of care.
Results and safety
The study was stopped early (reason given: Sponsor decision (not a safety decision)). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 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.
43 sites in 16 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MarzAA in people with Hemophilia A With Inhibitor and Hemophilia B With Inhibitor, run by Catalyst Biosciences at 43 sites in 16 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 4 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 17 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 43 sites in India, Malaysia, Italy, Turkey (Türkiye), Mexico and 11 more countries. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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: Sponsor decision (not a safety decision)). 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?
Bleeding episode treatment success (time frame: 24 hours after the first administration of study drug). 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 17 Dec 2021.