Hemophilia APhase 1NCT02418793
A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B
A Phase 1 study testing MOD-5014 in people with Hemophilia A and Hemophilia B, run by OPKO Health, Inc. at 11 sites in United States.
Trial summary
The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- MOD-5014
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 11 sitesUnited States
- Study dates
- May 2015to Sep 2018
- Phase
- Phase 1
- 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 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of moderate or severe congenital Hemophilia A or B with or without inhibitors
Who cannot join
- Diagnosis of any coagulation disorder other than Hemophilia A or B
- Receipt of any immunomodulatory therapy within 3 months prior to screening, with the exception of Hepatitis C or HIV therapy
- Have had, within one month prior to study drug administration, a major surgical procedure (e.g. orthopedic, abdominal) or have an elective surgery planned within the study period
- Use of any anticoagulant for arterial/venous obstructions and/or atrial fibrillation within 7 days prior to first study drug administration
- Malignancy within past 5 years (excluding non-melanoma skin cancer)
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Rush University Medical Center
Chicago, Illinois
UT Southwestern Medical Center
Dallas, Texas
City of Hope
Duarte, California
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania
The Gulf States Hemophilia and Thrombophilia Center
Houston, Texas
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana
Children's Hospital Los Angeles
Los Angeles, California, 90027
Orthopaedic Hemophilia Treatment Center
Los Angeles, California
Bleeding & Clotting Disorders Institute
Peoria, Illinois
UPMC Presbyterian Shadyside
Pittsburgh, Pennsylvania
University of California Davis Medical Center
Sacramento, California
About this study
Official title: A Phase 1/2a, Open-Label, Multicenter, Dose Escalation Study to Assess the Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) Profile of a Long Acting Recombinant FVIIa (MOD-5014) in Adult Men With Hemophilia A or B
The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- MOD-5014
- Keywords
- Hemophilia, Factor VII, Factor VIIa, Inhibitors, Long Acting
- 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.
MOD-5014Drug
Long Acting Factor VIIa
| Study group | Role | What they receive |
|---|---|---|
| Dose Cohort 1 | Experimental | Lowest MOD-5014 dose tested in the study Receives: Drug: MOD-5014 |
| Dose Cohort 2 | Experimental | MOD-5014 Dose cohort 2 Receives: Drug: MOD-5014 |
| Dose Cohort 3 | Experimental | MOD-5014 Dose cohort 3 Receives: Drug: MOD-5014 |
| Dose Cohort 4 | Experimental | MOD-5014 Dose cohort 4 Receives: Drug: MOD-5014 |
| Dose Cohort 5 | Experimental | MOD-5014 Dose cohort 5 Receives: Drug: MOD-5014 |
| Dose Cohort 6 | Experimental | Highest MOD-5014 dose tested in the study Receives: Drug: MOD-5014 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
To assess the acute safety and tolerability of single intravenous (IV) administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
Measured: 30 days
Secondary outcomes (1)
Pharmacokinetic Profile
To evaluate the pharmacokinetic profile (MOD-5014 serum levels; Area Under the Curve (AUC); Cmax; Tmax; T1/2) of single IV administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
Measured: 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2015 (estimated)
- Study startMay 2015
- Primary completionSep 2018
- Study completionSep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- OPKO Health, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Guy Ben-Bashat, Study director, OPKO Health, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02418793
- Sponsor's protocol ID
- CP-5-001
- Status last verified
- May 2021
- First submitted
- 31 Mar 2015
- Last updated
- 20 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2a, Open-Label, Multicenter, Dose Escalation Study to Assess the Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) Profile of a Long Acting Recombinant FVIIa (MOD-5014) in Adult Men With Hemophilia A or B
- ClinicalTrials.gov ID
- NCT02418793
- Acronym / sponsor ID
- CP-5-001
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- Hemophilia, Factor VII, Factor VIIa, Inhibitors, Long Acting
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- OPKO Health, Inc.Industry (pharma, biotech, devices)
- Study start
- May 2015
- Primary completion
- Sep 2018 (actual)
- Study completion
- Sep 2018 (actual)
- First posted
- 16 Apr 2015 (estimated)
- 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 current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Dose Cohort 1 | Experimental | Drug: MOD-5014Lowest MOD-5014 dose tested in the study |
| Dose Cohort 2 | Experimental | Drug: MOD-5014MOD-5014 Dose cohort 2 |
| Dose Cohort 3 | Experimental | Drug: MOD-5014MOD-5014 Dose cohort 3 |
| Dose Cohort 4 | Experimental | Drug: MOD-5014MOD-5014 Dose cohort 4 |
| Dose Cohort 5 | Experimental | Drug: MOD-5014MOD-5014 Dose cohort 5 |
| Dose Cohort 6 | Experimental | Drug: MOD-5014Highest MOD-5014 dose tested in the study |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Diagnosis of moderate or severe congenital Hemophilia A or B with or without inhibitors
Key exclusion criteria (5 listed)
- Diagnosis of any coagulation disorder other than Hemophilia A or B
- Receipt of any immunomodulatory therapy within 3 months prior to screening, with the exception of Hepatitis C or HIV therapy
- Have had, within one month prior to study drug administration, a major surgical procedure (e.g. orthopedic, abdominal) or have an elective surgery planned within the study period
- Use of any anticoagulant for arterial/venous obstructions and/or atrial fibrillation within 7 days prior to first study drug administration
- Malignancy within past 5 years (excluding non-melanoma skin cancer)
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 |
|---|---|---|
| MOD-5014Long Acting Factor VIIa | Drug | Dose Cohort 1, Dose Cohort 2, Dose Cohort 3, Dose Cohort 4, Dose Cohort 5, Dose Cohort 6 |
Primary outcome
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
Time frame: 30 days
To assess the acute safety and tolerability of single intravenous (IV) administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
1 secondary outcome
Pharmacokinetic Profile
Time frame: 14 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2021.
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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.
Guy Ben-Bashat study director
OPKO Health, Inc.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MOD-5014 in people with Hemophilia A and Hemophilia B, run by OPKO Health, Inc. at 11 sites in United States.
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 1 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 20 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions (time frame: 30 days). 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 20 May 2021.