Trial Outcomes & Findings for Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial (NCT NCT03588741)

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

1 participants

Primary outcome timeframe

Month 0 - up to month 12

Results posted on

2020-07-07

Participant Flow

The trial was conducted at 1 trial site in Germany.

Previously treated participants with severe haemophilia A (FVIII activity \<1% according to medical records) who had developed clinically relevant FVIII inhibitors in trial NN7170-4213 were offered immune tolerance induction (ITI) treatment with turoctocog alfa.

Participant milestones

Participant milestones
Measure	Turoctocog Alfa The participant received intravenous (i.v.) injection of 65 international units per kilogram (IU/kg) turoctocog alfa 3 times per week. The planned treatment duration was for at least 12 months and up to a maximum period of 24 months.
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Turoctocog Alfa The participant received intravenous (i.v.) injection of 65 international units per kilogram (IU/kg) turoctocog alfa 3 times per week. The planned treatment duration was for at least 12 months and up to a maximum period of 24 months.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Turoctocog Alfa n=1 Participants The participant received intravenous (i.v.) injection of 65 international units per kilogram (IU/kg) turoctocog alfa 3 times per week. The planned treatment duration was for at least 12 months and up to a maximum period of 24 months.
Age, Categorical <=18 years	0 Participants n=1 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=1 Participants
Age, Categorical >=65 years	0 Participants n=1 Participants
Sex: Female, Male Female	0 Participants n=1 Participants
Sex: Female, Male Male	1 Participants n=1 Participants

PRIMARY outcome

Timeframe: Month 0 - up to month 12

Population: Safety analysis set (SAS) comprised of the participant(s) who initiated ITI treatment with turoctocog alfa.

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.

Outcome measures

Outcome measures
Measure	Turoctocog Alfa n=1 Participants The participant received intravenous (i.v.) injection of 65 international units per kilogram (IU/kg) turoctocog alfa 3 times per week. The planned treatment duration was for at least 12 months and up to a maximum period of 24 months.
Number of Adverse Events	6 Adverse events

SECONDARY outcome

Timeframe: Month 12

Population: FAS comprised of the participant(s) who initiated ITI treatment with turoctocog alfa.

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.

Outcome measures

Outcome measures
Measure	Turoctocog Alfa n=1 Participants The participant received intravenous (i.v.) injection of 65 international units per kilogram (IU/kg) turoctocog alfa 3 times per week. The planned treatment duration was for at least 12 months and up to a maximum period of 24 months.
Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other) Success	0 Participants
Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other) Partial success	0 Participants
Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other) Failure	0 Participants
Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other) Other	1 Participants

Adverse Events

Turoctocog Alfa

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Turoctocog Alfa n=1 participants at risk The participant received intravenous (i.v.) injection of 65 international units per kilogram (IU/kg) turoctocog alfa 3 times per week. The planned treatment duration was for at least 12 months and up to a maximum period of 24 months.
Infections and infestations Nasopharyngitis	100.0% 1/1 • Number of events 3 • Month 0 - up to month 12 Results are based on the safety analysis set (SAS), which comprised the participant(s) who initiated ITI treatment with turoctocog alfa.
Musculoskeletal and connective tissue disorders Muscle disorder	100.0% 1/1 • Number of events 2 • Month 0 - up to month 12 Results are based on the safety analysis set (SAS), which comprised the participant(s) who initiated ITI treatment with turoctocog alfa.
Nervous system disorders Headache	100.0% 1/1 • Number of events 1 • Month 0 - up to month 12 Results are based on the safety analysis set (SAS), which comprised the participant(s) who initiated ITI treatment with turoctocog alfa.

Additional Information

Clinical Reporting Anchor and Disclosure (1452)

Novo Nordisk A/S

Phone: (+1) 866-867-7178

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
Publication restrictions are in place

Restriction type: OTHER