NCT02418793

Brief 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. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started May 2015

Typical duration for phase_1

Geographic Reach
1 country

11 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 31, 2015

Completed
16 days until next milestone

First Posted

Study publicly available on registry

April 16, 2015

Completed
15 days until next milestone

Study Start

First participant enrolled

May 1, 2015

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2018

Completed
Last Updated

May 20, 2021

Status Verified

May 1, 2021

Enrollment Period

3.3 years

First QC Date

March 31, 2015

Last Update Submit

May 18, 2021

Conditions

Hemophilia AHemophilia B

Keywords

HemophiliaFactor VIIFactor VIIaInhibitorsLong Acting

Outcome Measures

Primary Outcomes (1)

  • 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.

    30 days

Secondary Outcomes (1)

  • Pharmacokinetic Profile

    14 days

Other Outcomes (1)

  • Pharmacodynamic Profile (evaluate the pharmacodynamic response (FVIIa clotting activity, coagulation parameters) of single IV administration of escalating MOD-5014 doses)

    14 days

Study Arms (6)

Dose Cohort 1

EXPERIMENTAL

Lowest MOD-5014 dose tested in the study

Drug: MOD-5014

Dose Cohort 2

EXPERIMENTAL

MOD-5014 Dose cohort 2

Drug: MOD-5014

Dose Cohort 3

EXPERIMENTAL

MOD-5014 Dose cohort 3

Drug: MOD-5014

Dose Cohort 4

EXPERIMENTAL

MOD-5014 Dose cohort 4

Drug: MOD-5014

Dose Cohort 5

EXPERIMENTAL

MOD-5014 Dose cohort 5

Drug: MOD-5014

Dose Cohort 6

EXPERIMENTAL

Highest MOD-5014 dose tested in the study

Drug: MOD-5014

Interventions

MOD-5014DRUG

Long Acting Factor VIIa

Dose Cohort 1Dose Cohort 2Dose Cohort 3Dose Cohort 4Dose Cohort 5Dose Cohort 6

Eligibility Criteria

Age18 Years - 65 Years
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of moderate or severe congenital Hemophilia A or B with or without inhibitors

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

City of Hope

Duarte, California, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Orthopaedic Hemophilia Treatment Center

Los Angeles, California, United States

Location

University of California Davis Medical Center

Sacramento, California, United States

Location

Rush University Medical Center

Chicago, Illinois, United States

Location

Bleeding & Clotting Disorders Institute

Peoria, Illinois, United States

Location

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Location

UPMC Presbyterian Shadyside

Pittsburgh, Pennsylvania, United States

Location

UT Southwestern Medical Center

Dallas, Texas, United States

Location

The Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, United States

Location

MeSH Terms

Conditions

Hemophilia AHemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • Guy Ben-Bashat

    OPKO Health, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 31, 2015

First Posted

April 16, 2015

Study Start

May 1, 2015

Primary Completion

September 1, 2018

Study Completion

September 1, 2018

Last Updated

May 20, 2021

Record last verified: 2021-05

Locations

US(11)