Haemophilia APhase 3NCT03588741
Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial
A Phase 3 study testing Turoctocog alfa in people with Haemophilia A, run by Novo Nordisk A/S at 14 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Haemophilia A
- What is being tested?
- Turoctocog alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 14 sitesin 7 countries
- Study dates
- 12 Jun 2018to 19 Jun 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
14 sites in 7 countries.
14 sites in 7 countries
Serbia4 sites
Novo Nordisk Investigational Site
Belgrade, 11000
Novo Nordisk Investigational Site
Belgrade, 11070
Novo Nordisk Investigational Site
Niš, 18000
Novo Nordisk Investigational Site
Novi Sad, 21000
Germany3 sites
Novo Nordisk Investigational Site
Berlin, 10249
Novo Nordisk Investigational Site
Duisburg, 47051
Novo Nordisk Investigational Site
Homburg, 66421
United Kingdom3 sites
Novo Nordisk Investigational Site
London, NW3 2QG
Novo Nordisk Investigational Site
Oxford, OX3 7LJ
Novo Nordisk Investigational Site
Sheffield, S10 2JF
Turkey (Türkiye)1 site
Novo Nordisk Investigational Site
Bornova-IZMIR, 35100
About this study
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)
- Conditions
- Haemophilia A
- Being tested
- Turoctocog alfa
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| Turoctocog alfa | Experimental | Not described Receives: Drug: Turoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Month 0 - up to month 12
Secondary outcomes (1)
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.
Measured: Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2018
- Study start12 Jun 2018
- Primary completion19 Jun 2019
- Study completion19 Jun 2019
Why it stopped: The trial was terminated as the participant withdrew from the trial.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor and Disclosure (1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03588741
- Sponsor's protocol ID
- NN7170-4345
- Other IDs
- U1111-1187-7323, 2016-003821-40, 2016
- Status last verified
- Jul 2020
- First submitted
- 12 Jun 2018
- Last updated
- 7 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial
- ClinicalTrials.gov ID
- NCT03588741
- Acronym / sponsor ID
- NN7170-4345
- Conditions
- Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was terminated as the participant withdrew from the trial.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 12 Jun 2018 (actual)
- Primary completion
- 19 Jun 2019 (actual)
- Study completion
- 19 Jun 2019 (actual)
- First posted
- 17 Jul 2018 (actual)
- Results
- Posted 7 Jul 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Turoctocog alfa | Experimental | Drug: Turoctocog alfa |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Turoctocog alfaIntravenous (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. | Drug | Turoctocog alfa |
Primary outcome
Number of Adverse Events
Time frame: 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.
1 secondary outcome
Response to FVIII ITI Treatment (Success, Partial Success, Failure, Other)
Time frame: Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Reporting Anchor and Disclosure (1452) study director
Novo Nordisk A/S
14 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Turoctocog alfa in people with Haemophilia A, run by Novo Nordisk A/S at 14 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Serbia, Germany, United Kingdom, Austria, Bulgaria and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jul 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse Events (time frame: Month 0 - up to month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2020.