Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial

Phase 3

Terminated

• Conditions: Haemophilia A
• Interventions: Drug: Turoctocog alfa

• Registration Number: NCT03588741
• Lead Sponsor: Novo Nordisk A/S

Brief Summary

This trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate safety of immune tolerance induction (ITI) treatment with turoctocog alfa (a recombinant factor VIII) in patients who have developed neutralising antibodies against factor VIII after exposure to subcutaneous turoctocog alfa pegol during participation in NN7170-4213 (NCT02994407)

Detailed Description

Not available

Study Timeline

• Study Start: 2018-06-12
• Study First Submitted: 2018-06-12
• Study First Submitted QC: 2018-07-03
• Study First Posted: 2018-07-17
• Primary Completion: 2019-06-19
• Study Completion: 2019-06-19
• Results First Submitted: 2020-06-12
• Status Verified: 2020-07
• Results First Submitted QC: 2020-07-02
• Last Update Submitted: 2020-07-02
• Results First Posted: 2020-07-07
• Last Update Posted: 2020-07-07

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 1

Inclusion Criteria

• Previous participation in the NN7170-4213 trial (male, age at least 18 years (part A) and age at least 12 years (part B))
• Development of a confirmed high titre neutralising antibody towards factor VIII (greater than 5 Bethesda Unit) after exposure to subcutaneous turoctocog alfa pegol in the NN7170-4213 trial or development of a confirmed clinically relevant low titre inhibitor (at least 0.6 to below or equal to 5 Bethesda Unit), defined as factor VIII activity measures (recovery) and/or bleedpattern indicating a lack of clinical response to factor VIII treatment

Exclusion Criteria

• Known or suspected hypersensitivity to trial product(s) or related products, defined as allergic reactions
• Participation in another clinical trial within 1 month before screening (except participation in NN7170-4213)
• Any disorder, except for conditions associated with Haemophilia A which in the investigator's opinion might jeopardise patients' safety or compliance with the protocol
• Currently receiving immune tolerance induction treatment with a factor VIII containing product other than turoctocog alfa

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Turoctocog alfa	Turoctocog alfa	-

Primary Outcome Measures

Name	Time	Method
Number of Adverse Events	Month 0 - up to month 12	An adverse event (AE) was any untoward medical occurrence in a participant administered a medicinal product, and which does not necessarily have a causal relationship with this treatment.

Secondary Outcome Measures

Name	Time	Method
Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other)	Month 12	ITI treatment response was categorized as: 1. Success: Undetectable inhibitor titre \<0.6 bethesda units (BU) (or lower limit of quantification \[LLoQ\] if above 0.6 BU); Normalised FVIII in vivo recovery, defined as ≥0.013 international units (IU) per milliliter per IU per kilogram ((IU/ml)/(IU/kg)) (66% of expected incremental recovery); turoctocog alfa half-life ≥7 hours (based on FVIII activity) after 72 hours treatment-free washout period. 2. Partial success: Inhibitor titre ≤5 BU; Clinical effect of turoctocog alfa therapy as judged by the investigator. 3. Failure (one criterion had to be fulfilled): Failure to attain defined success or partial success after 24 months of ITI treatment with turoctocog alfa; Decrease in inhibitor titre after 12 months of ITI treatment \<20% compared to peak titre. 4. Other: Participants not fulfilling the above criteria e.g. early withdrawal from ITI treatment, lack of adherence to recommended ITI protocol etc.

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇬🇧 Sheffield, United Kingdom