A Phase 1 interventional study of MOD-5014 and MOD-5014 Placebo in Hemophilia A or B With Inhibitors, sponsored by OPKO Health, Inc.. Completed at 1 site in Israel. Open to male participants aged 18 Years to 50 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2019-09-30.
Sponsored by OPKO Health, Inc. · Phase 1, Interventional, and Other
A Phase 1, randomized, single-blind, placebo-controlled, single dose, dose-escalation study to assess the safety, pharmacokinetic and pharmacodynamic profile of subcutaneous administration of a long-acting recombinant factor VIIa (MOD-5014) in healthy adult males.
This will be a single-dose, randomized, single-blind, placebo-controlled, dose-escalating study.
The study will include four escalating dose groups, with eight subjects in each dose group. Subjects will be randomized in 3:1 ratio to receive a single SC injection of MOD-5014 (n=6) or a placebo (n=2), and will be followed up for 30 days. The initial MOD-5014 dose group will receive 100 µg/kg followed by single doses of 200, 400 and 600 µg/kg administered to subsequent subject cohorts.
The decision to proceed to the higher dose level will be made by a Data Safety Monitoring Board (DSMB) after review of relevant safety data (including adverse events, clinical laboratory and vital signs), collected up to and including 7 days after the last subject of the previous dose group has been dosed.
Common Terminology Criteria for Adverse Events (CTCAE) guidelines will be used to determine maximum tolerated dose (MTD) and dose limiting toxicity (DLT). Dose escalation will be permitted if the prior dose is well tolerated, and there are no safety or tolerability concerns raised by the investigator, sponsor, medical monitor or DSMB over 7 days post-dosing.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 32 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →OPKO Health, Inc. is the lead sponsor of 48 studies on the registry; 2 are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 6 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
MOD-5014 longevity is the result of fusion of three consecutive C-terminal peptide (CTP) domains to the C-terminus of FVII. CTP technology is based on a natural peptide, the C-terminal peptide of the beta chain of human chorionic gonadotropin (hCG), which provides hCG with the required longevity to maintain pregnancy (initial half-life \[t1/2\] \~10 h, terminal t1/2 \~37 h). The beta chain of luteinizing hormone (LH), a gonadotropin that triggers ovulation, is almost identical to hCG but does not include the CTP. As a result, LH has a significantly shorter half-life in blood (initial t1/2 \~1 h, terminal t1/2 \~10 h) (Fares et al., 1992).
Biological: MOD-5014
Placebo solution for SC injection containing the same inactive ingredients used in the active drug product at matching volumes
Other: MOD-5014 Placebo
MOD-5014, a long-acting modified recombinant Factor VIIa
Placebo solution for SC injection containing the same inactive ingredients used in the active drug product at matching volumes
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), Immunogenicity, laboratory results, vital signs and injection site reactions
Time frame: within 30 days of injection
Cmax of MOD-5014
Time frame: within 30 days of injection
Tmax of MOD-5014
Time frame: within 30 days of injection
AUC(0-t) of MOD-5014
Time frame: within 30 days of injection
AUC(inf) of MOD-5014
Time frame: within 30 days of injection
T(½) of MOD-5014
Time frame: within 30 days of injection
Clearance of MOD-5014
Time frame: within 30 days of injection
Plan to share: Undecided
This study is completed, as verified in Sep 2019. You cannot join it, but the record below documents what was studied.
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OPKO Health, Inc.