Hemophilia APhase 1NCT04072237

Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia

A Phase 1 study testing MarzAA (marzeptacog alfa [activated]) in people with Hemophilia A and Hemophilia B, run by Catalyst Biosciences at 5 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older, men only
Conditions the study is for: Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
What is being tested?: MarzAA (marzeptacog alfa [activated])
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 11 participants
Where: 5 sitesin 2 countries
Study dates: 24 Sep 2019to 17 Jun 2020
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 18 years and older
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

5 sites in 2 countries.

Country

City, postcode or hospital

5 sites in 2 countries

Russia3 sites

Kirov Research Institute of Hematology and Blood Transfusion
Kirov
National Medical Hematology Research Center
Moscow
Municipal Policlinic # 37, City Center for Hemophilia Treatment
Saint Petersburg

Other trials in Russia

Bulgaria2 sites

Medical Center "Hippocrates - N"
Plovdiv
Specialized Hospital for Active Treatment of Hematological Diseases
Sofia

Other trials in Bulgaria

About this study

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

Read the 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).

Conditions: Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Hemophilia A Without Inhibitor, Hemophilia B Without Inhibitor
Being tested: MarzAA (marzeptacog alfa [activated])
Medical subject headings: Hemophilia A, Hemophilia B

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MarzAA (marzeptacog alfa [activated])Biological
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA (Coagulation Faction VIIa Variant)

Study groups in NCT04072237
Study group	Role	What they receive
Study Population	Experimental	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) Receives: Biological: MarzAA (marzeptacog alfa [activated])

What is being measured

The primary outcome is the main question the study is designed to answer.

Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last
Comparative pharmacokinetics (PK) by dose level/stage based on examination of AUC frequencies of these for each of the dose groups
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity by Dose Level/Stage - AUCT1-T2 Normalized by Dose = AUC0-last/Dose
Comparative pharmacokinetics by dose level/stage based on examination of AUC frequency of these for each of the dose groups
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

Secondary outcomes (21)

Comparative MarzAA Activity of Intravenous and Subcutaneous - Cmax
Change in Cmax at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - Tmax
Change in Tmax at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2eqα
Change in T1/2eqα at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2λ-z
Change in T1/2λ-z at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - CL
Change in CL at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - Vd1
Change in Vd1 at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - BAabs
Change in BAabs at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - Mean Residence Time
Change in Mean Residence Time at each stage for each dose group
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC From T1 to T2 Norm by Dose
PK analysis by route of administration, dose level/stage of the study based on examination of AUC for each of the dose groups.
Split dose (2*30 µg/kg) vs. (60 µg/kg)
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC Infinity Obs and AUC to Last Nonzero Conc
PK analysis by route of administration, dose level/stage of the study based on examination of AUC for each of the dose groups.
Split dose (2*30 µg/kg) vs. (60 µg/kg)
Measured: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Change in Coagulation Parameters - Prothrombin Time (PT)
Maximum change in PT from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-7 (SC).
Change in Coagulation Parameters - Activated Partial Thromboplastin Time (aPTT)
Maximum change in aPTT from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Peak
Maximum change in TGT parameter from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Lag and TGT-Time to Peak
Maximum change in TGT parameters from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Endogenous Thrombin Potential
Maximum change in TGT parameter from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - Fibrinogen
Maximum change in thrombogenicity parameter from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - Prothrombin Fragments 1 + 2
Maximum change in thrombogenicity parameter from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - Thrombin/Antithrombin
Maximum change in thrombogenicity parameter from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - D-Dimer
Maximum change in thrombogenicity parameter from pre-dose
Measured: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Occurrence of an Antibody Response to MarzAA
Occurrence of an antibody response to MarzAA and whether it is inhibitory and cross-reactive to wild-type recombinant coagulation FVII (wt-rFVII) or wt-FVIIa
Measured: From time of first dose of MarzAA until date of first occurrence of clinical event, assessed up to End of Study. Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing.
Occurrence of Clinical Thrombotic Event
Occurrence of clinical thrombotic event not attributable to another cause
Measured: From the date of first dose of MarzAA until date of first occurrence of clinical event, assessed up to End of Study. Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Sep 201917 Jun 2020

First posted28 Aug 2019
Study start24 Sep 2019
Primary completion30 Apr 2020
Study completion17 Jun 2020

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Hemophilia AStudies 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
Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Sequential
Masking: None (open label)
Enrolment: 11 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04072237
Sponsor's protocol ID: MAA-102
Status last verified: Sep 2021
First submitted: 22 Aug 2019
Last updated: 13 Sep 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No: This is an open label study so each investigator will have full access to all study subject data that is entered into the database.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 13 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04072237
Acronym / sponsor ID: MAA-102
Conditions: Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Hemophilia A Without Inhibitor, Hemophilia B Without Inhibitor
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 11 participants (actual)
Lead sponsor: Catalyst BiosciencesIndustry (pharma, biotech, devices)
Study start: 24 Sep 2019 (actual)
Primary completion: 30 Apr 2020 (actual)
Study completion: 17 Jun 2020 (actual)
First posted: 28 Aug 2019 (actual)
Results: Posted 16 Jul 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Sequential
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Study Population	Experimental	Biological: MarzAA (marzeptacog alfa [activated])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)

Who may qualify

Ages: 18 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

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

Key exclusion criteria (5 listed)

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

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
MarzAA (marzeptacog alfa [activated])Single intravenous dose and ascending doses of subcutaneous injection of MarzAA (Coagulation Faction VIIa Variant)	Biological	Study Population

Primary outcome

Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative pharmacokinetics (PK) by dose level/stage based on examination of AUC frequencies of these for each of the dose groups
Comparative MarzAA Activity by Dose Level/Stage - AUCT1-T2 Normalized by Dose = AUC0-last/Dose
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative pharmacokinetics by dose level/stage based on examination of AUC frequency of these for each of the dose groups

21 secondary outcomes

Comparative MarzAA Activity of Intravenous and Subcutaneous - Cmax
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - Tmax
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2eqα
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2λ-z
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - CL
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - Vd1
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - BAabs
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Comparative MarzAA Activity of Intravenous and Subcutaneous - Mean Residence Time
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC From T1 to T2 Norm by Dose
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC Infinity Obs and AUC to Last Nonzero Conc
Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Change in Coagulation Parameters - Prothrombin Time (PT)
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-7 (SC).
Change in Coagulation Parameters - Activated Partial Thromboplastin Time (aPTT)
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Peak
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Lag and TGT-Time to Peak
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Endogenous Thrombin Potential
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - Fibrinogen
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - Prothrombin Fragments 1 + 2
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - Thrombin/Antithrombin
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Change in Thrombogenicity Parameter - D-Dimer
Time frame: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).
Occurrence of an Antibody Response to MarzAA
Time frame: From time of first dose of MarzAA until date of first occurrence of clinical event, assessed up to End of Study. Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing.
Occurrence of Clinical Thrombotic Event
Time frame: From the date of first dose of MarzAA until date of first occurrence of clinical event, assessed up to End of Study. Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing.

Results and safety

Results posted to the registry on 16 Jul 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Overall Study Population	11	10

Primary outcome: Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last

Time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.. Mean (Standard Deviation). Unit: h*ng/mL

Measure	Stage 1 MarzAA 18 µg/kg IV n = 10	Stage 2 MarzAA 30 µg/kg SC n = 8	Stage 3 MarzAA 45 µg/kg SC n = 8	Stage 4 MarzAA 60 µg/kg SC n = 8	Stage 5 MarzAA 2×30 µg/kg SC n = 8	Stage 6 MarzAA 90 µg/kg SC n = 8	Stage 7 MarzAA 120 µg/kg SC n = 8	Stage 8 2×60 µg/kg SC Q3H n = 8	Stage 9 3×60 µg/kg SC Q3H n = 8
AUC0-∞	1390.0 (433.99)	516.4 (170.01)	849.4 (275.00)	1060.0 (205.86)	1025.6 (328.31)	1487.9 (429.39)	2087.6 (648.85)	2108.0 (409.00)	3235.5 (735.43)
AUC0-last	1382.6 (431.45)	429.5 (178.30)	692.9 (232.53)	922.1 (192.38)	934.1 (305.32)	1322.6 (440.03)	1868.1 (651.04)	1939.1 (357.25)	3038.1 (760.56)

Primary outcome: Comparative MarzAA Activity by Dose Level/Stage - AUCT1-T2 Normalized by Dose = AUC0-last/Dose

Measure	Stage 1 MarzAA 18 µg/kg IV n = 10	Stage 2 MarzAA 30 µg/kg SC n = 8	Stage 3 MarzAA 45 µg/kg SC n = 8	Stage 4 MarzAA 60 µg/kg SC n = 8	Stage 5 MarzAA 2×30 µg/kg SC n = 8	Stage 6 MarzAA 90 µg/kg SC n = 8	Stage 7 MarzAA 120 µg/kg SC n = 8	Stage 8 2×60 µg/kg SC Q3H n = 8	Stage 9 3×60 µg/kg SC Q3H n = 8
Value	76.81 (23.97)	14.32 (5.94)	15.40 (5.17)	15.37 (3.206)	15.57 (5.09)	14.70 (4.89)	15.57 (5.43)	16.16 (2.98)	16.88 (4.23)

21 secondary outcomes are reported in the full results.

Adverse events

Time frame: Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Stage 1 MarzAA 18 µg/kg SC	0 of 11 (0%)	0 of 11 (0%)	0 of 11
Stage 2 MarzAA 30 µg/kg SC	0 of 8 (0%)	3 of 8 (37.5%)	0 of 8
Stage 3 MarzAA 45 µg/kg SC	0 of 8 (0%)	0 of 8 (0%)	0 of 8
Stage 4 MarzAA 60 µg/kg SC	0 of 8 (0%)	1 of 8 (12.5%)	0 of 8
Stage 5 MarzAA 2×30 µg/kg SC	0 of 8 (0%)	1 of 8 (12.5%)	0 of 8
Stage 6 MarzAA 90 µg/kg SC	0 of 8 (0%)	0 of 8 (0%)	0 of 8
Stage 7 MarzAA 120 µg/kg SC	0 of 8 (0%)	2 of 8 (25%)	0 of 8
Stage 8 MarzAA 2×60 µg/kg SC Q3H	0 of 8 (0%)	3 of 8 (37.5%)	0 of 8
Stage 9 MarzAA 3×60 µg/kg SC Q3H	0 of 8 (0%)	2 of 8 (25%)	0 of 8

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No. This is an open label study so each investigator will have full access to all study subject data that is entered into the database.

Related trials for Hemophilia A

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
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Active, not recruitingPhase 3
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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.

Howard Levy, MD, PhD, MMM study director

Sponsor GmbH

5 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing MarzAA (marzeptacog alfa [activated]) in people with Hemophilia A and Hemophilia B, run by Catalyst Biosciences at 5 sites in 2 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 3 inclusion and 5 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: finished, as of the sponsor's last update on 13 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Russia, Bulgaria. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Jul 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 - AUC0-∞ and AUC0-last (time frame: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.). 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?

On the ClinicalTrials.gov record for NCT04072237.

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 13 Sep 2021.