NCT03528551|CompletedPhase 3ResultsFDA Drug

A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A

pathfinder8

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated Patients With Severe Haemophilia A

Novo Nordisk A/S ClinicalTrials.gov

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4 other identifiers

NN7088-4410U1111-1202-27802017-003788-36JapicCTI-183952

Study Type

interventional

Target

160

Locations

26 countries

Sites

Timeline

RegisteredMay 2018

StartedApr 2018

CompletionDec 2020

Brief Summary

This study will look at how a known study medicine N8-GP works in previously N8-GP treated people with haemophilia A. The aim is to look at how N8-GP works during regular use. Participants will get N8-GP. N8-GP has been tested in more than 200 people with haemophilia A for several years. Participants will get an injection of N8-GP into a blood vessel, one, two or three times weekly. Participants will get more doses if they bleed or if they will need a surgery. The study will last for about 2 years. Participants will have at least 9 visits with the study doctor. If participants agree to be in this study, they will get their first injection (in this study) at the first visit. Participants will also get an injection at visit 3, 5 and 7. Participants will be trained to give all other injections themselves. Participants must not use any clotting factors other than N8-GP or any anticoagulants (blood thinners) during the study.

Trial Health

On Track

Trial Health Score

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

Enrollment

160

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Apr 2018

Typical duration for phase_3

Geographic Reach

26 countries

67 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

2.6 years study duration

First Submitted

Initial submission to the registry

April 18, 2018

Completed

12 days until next milestone

Study Start

First participant enrolled

April 30, 2018

Completed

18 days until next milestone

First Posted

Study publicly available on registry

May 18, 2018

Completed

2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 3, 2020

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 3, 2020

Completed

12 months until next milestone

Results Posted

Study results publicly available

November 26, 2021

Completed

Last Updated

December 22, 2022

Status Verified

December 1, 2022

Enrollment Period

2.6 years

First QC Date

April 18, 2018

Results QC Date

July 22, 2021

Last Update Submit

December 20, 2022

Conditions

Congenital Bleeding Disorder Haemophilia A

Outcome Measures

Primary Outcomes (1)

Number of Adverse Events Reported
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All AEs mentioned here are treatment emergent adverse events (TEAEs). The TEAE is defined as an event reported from date of first trial product administration until end of the treatment visit (week 104) or follow-up visit if relevant (1 month after end of the treatment).
Week 0 to week 108

Secondary Outcomes (11)

Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU)
Week 0 to week 104
Number of Bleeding Episodes on Prophylaxis
Week 0 to week 104
Number of Spontaneous Bleeding Episodes on Prophylaxis
Week 0 to week 104
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None
Week 0 to week 104
Mean Number of N8-GP Injections Required Per Bleeding Episode
Week 0 to week 104
+6 more secondary outcomes

Study Arms (3)

N8-GP, once weekly

EXPERIMENTAL

All participants will receive turoctocog alfa pegol (N8-GP) once weekly.

Drug: Turoctocog alfa pegol

N8-GP, twice weekly

EXPERIMENTAL

All participants will receive N8-GP twice weekly.

Drug: Turoctocog alfa pegol

N8-GP, three times weekly

EXPERIMENTAL

All participants will receive N8-GP three times weekly.

Drug: Turoctocog alfa pegol

Interventions

Turoctocog alfa pegolDRUG

Turoctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years.

N8-GP, once weekly

Eligibility Criteria

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Male patients of all ages with the diagnosis of severe congenital haemophilia A (coagulation Factor VIII \[FVIII\] activity less than 1%) based on medical records
On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer

You may not qualify if:

Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol - Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or non-approved investigational medicinal product

Contact the study team to confirm eligibility.