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

Phase 3TerminatedNCT03588741

Novo Nordisk A/S1 enrolled

Overview

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)

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Haemophilia A

Interventions

Turoctocog alfaDRUG

Intravenous (i.v., under the skin) administration.A maximum dose of 200 IU/kg daily. The maximum treatment period for this trial is 24 months and the patient(s) will be called for visit to the clinic every 3rd month.

Eligibility

Sex: MALEMin age: 12 Years

Medical Language ↔ Plain English

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

Locations (14)

Novo Nordisk Investigational Site

Vienna, Austria

Novo Nordisk Investigational Site

Sofia, Bulgaria

Novo Nordisk Investigational Site

Nantes, France

Outcomes

Primary Outcomes

Number of Adverse Events

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.

Time frame: Month 0 - up to month 12

Secondary Outcomes

Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other)

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.

Time frame: Month 12

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.