Congenital Bleeding DisorderPhase 3pathfinder™5NCT01731600
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
A Phase 3 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 51 sites in 16 countries.
Trial summary
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products. Read more about this study
- Who is it for?
- Ages 0 to 11 years, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A
- What is being tested?
- turoctocog alfa pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 68 participants
- Where
- 51 sitesin 16 countries
- Study dates
- 20 Feb 2013to 28 Sep 2018
- 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 0 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
- Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years
Who cannot join
- Any history of FVIII inhibitors
Trial locations and contacts
51 sites in 16 countries.
51 sites in 16 countries
United States24 sites
Novo Nordisk Investigational Site
Boise, Idaho, 83712
Novo Nordisk Investigational Site
Boston, Massachusetts, 02115
Novo Nordisk Investigational Site
Charleston, South Carolina, 29425
Novo Nordisk Investigational Site
Charlotte, North Carolina, 28204
Novo Nordisk Investigational Site
Charlottesville, Virginia, 22903
Novo Nordisk Investigational Site
Cincinnati, Ohio, 45229
Novo Nordisk Investigational Site
Dallas, Texas, 75235
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
Novo Nordisk Investigational Site
Houston, Texas, 77030
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Long Beach, California, 90806
Novo Nordisk Investigational Site
Minneapolis, Minnesota, 55404
Novo Nordisk Investigational Site
Nashville, Tennessee, 37232-9830
Novo Nordisk Investigational Site
New Hyde Park, New York, 11040
Novo Nordisk Investigational Site
New Orleans, Louisiana, 70118-5720
Novo Nordisk Investigational Site
Omaha, Nebraska, 68198-6828
Novo Nordisk Investigational Site
Orange, California, 92868
Novo Nordisk Investigational Site
Orlando, Florida, 32827
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19104
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19134
Novo Nordisk Investigational Site
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Spokane, Washington, 99204
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Novo Nordisk Investigational Site
Washington D.C., District of Columbia, 20010-2978
Turkey (Türkiye)4 sites
Novo Nordisk Investigational Site
Antalya, 01010
Novo Nordisk Investigational Site
Bornova-IZMIR, 35100
Novo Nordisk Investigational Site
İzmit, 41380
Novo Nordisk Investigational Site
Samsun
France3 sites
Novo Nordisk Investigational Site
Bron, 69677
Novo Nordisk Investigational Site
Lille, 59097
Novo Nordisk Investigational Site
Paris, 75015
Switzerland3 sites
Novo Nordisk Investigational Site
Bellinzona, 6500
Novo Nordisk Investigational Site
Lucerne, 6000
Novo Nordisk Investigational Site
Zurich, 8032
United Kingdom3 sites
Novo Nordisk Investigational Site
Leicester, LE1 5WW
Novo Nordisk Investigational Site
London, SE1 7EH
Novo Nordisk Investigational Site
Oxford, OX3 9DU
Greece2 sites
Novo Nordisk Investigational Site
Athens, GR-11527
Novo Nordisk Investigational Site
Thessaloniki, GR 54642
Japan2 sites
Novo Nordisk Investigational Site
Kitakyusyu, Fukuoka, 807 8555
Novo Nordisk Investigational Site
Tokyo, 167-0035
Ukraine2 sites
Novo Nordisk Investigational Site
Donetsk, 83045
Novo Nordisk Investigational Site
Lviv, 79044
About this study
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Being tested
- turoctocog alfa pegol
- 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 alfa pegolDrug
Also called NNC 0129-0000-1003, N8-GP
Fixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight.
| Study group | Role | What they receive |
|---|---|---|
| N8-GP | Experimental | Not described Receives: Drug: turoctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units
The number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) ≥0.6 Bethesda units was presented.
Measured: During the main phase of the trial (from 0-26 weeks of treatment)
Secondary outcomes (16)
Frequency of Adverse Events Including Serious Adverse Events Reported During the Trial Period
The frequency of adverse events including serious adverse events reported during the main and extension phase of the trial. The data presented is the rate of AE i.e. number of AEs per patient years of exposue.
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes and Assessed as: Excellent, Good, Moderate, or None
Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by patient and/or parent(s)/caregiver 8 hours after first injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hours after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms.
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Number of Bleeding Episodes During Prophylactic Treatment With N8-GP (Annualised Bleeding Rate)
The number of bleeding episodes per year reported during the prophylactic treatment with N8-GP.
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP Per Bleeding Episode (Number of Injections)
The mean number of injections of N8-GP used for treatment of a bleed from start to stop of a bleed.
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP Per Bleeding Episode (U/kg)
The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed.
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP During Prophylaxis (Number of Injections)
The mean number of injections of N8-GP used for treatment of a bleed from start to stop of a bleed during prophylaxis.
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP During Prophylaxis (U/kg Per Month)
The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed during prophylaxis (per month per subject). Consumption used for treatment includes all doses given (prophylaxis, treatment of bleed, minor surgery and pharmacokinetics [PK])
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP During Prophylaxis (U/kg Per Year)
The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed during prophylaxis (per year per subject). Consumption used for treatment includes all doses given (prophylaxis, treatment of bleed, minor surgery and pharmacokinetics)
Measured: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for Previous FVIII Product
The incremental recovery was defined as the increase in plasma FVIII activity per IU/kg of factor administered recorded 60 minutes after end of injection. It was calculated as (Factor VIII procoagulant [FVIII:C] activity measured in plasma 60 min after dosing - FVIII:C activity measured in plasma immediately before dosing) / (dose injected at time 0 min), where the dose was expressed as U FVIII product per kg body weight. A chromogenic assay with normal human plasma (NHP) as calibrator was used.
Measured: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for N8-GP
The incremental recovery was defined as the peak level recorded 60 min after end of injection and dose-normalised. It was calculated as (FVIII:C activity measured in plasma 60 min after dosing - FVIII:C activity measured in plasma immediately before dosing) / (dose injected at time 0 min), where the dose was expressed as U FVIII product per kg body weight. A chromogenic assay with product specific calibrator (PSS) as calibrator was used.
Measured: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Area Under the Curve Evaluated for Previous FVIII Product
Area under the curve (AUC) versus time from zero to infinity. This is calculated as AUC = AUClast + (C(t) / λz), where C(t) is the last measurable concentration. A chromogenic assay with NHP as calibrator was used.
Measured: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Area Under the Curve Evaluated for N8-GP
Area under the curve versus time from zero to infinity. This is calculated as AUC = AUClast + (C(t) / λz), where C(t) is the last measurable concentration. A chromogenic assay with product specific standard (PSS) as calibrator was used.
Measured: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Terminal Half-life Evaluated for Previous FVIII Product
t½ = ln(2) / λz, where t½ is terminal half-life and λz is the terminal elimination rate. The terminal elimination rate was planned estimated using linear regression on the terminal part of the time versus log(concentration) curve. A population-based method simultaneously estimating individual t½ values for all patients was applied, including patients with few values above the lower limit of quantification (LLOQ). This was estimated using time points from 1h to 30h. A chromogenic assay with PSS as calibrator was used.
Measured: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Terminal Half-life Evaluated for N8-GP
t½ = ln(2) / λz, where λz is the terminal elimination rate. The terminal elimination rate was planned estimated using linear regression on the terminal part of the time versus log(concentration) curve. A population-based method simultaneously estimating individual t½ values for all patients was applied, including patients with few values above the LLOQ. This was estimated using time points from 6h to 96h. A chromogenic assay with PSS as calibrator was used.
Measured: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Clearance Evaluated for Previous FVIII Product
Total plasma clearance of drug after intravenous administration measured as actual dose/AUC. A chromogenic assay with NHP as calibrator was used.
Measured: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Clearance Evaluated for N8-GP
Total plasma clearance of drug after intravenous administration measured as actual dose/AUC. A chromogenic assay with PSS as calibrator was used.
Measured: From 1 hour prior to and up to 96 hours after initial administration of N8-GP.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2012 (estimated)
- Study start20 Feb 2013
- Primary completion15 Sep 2014
- Study completion28 Sep 2018
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Bleeding DisorderStudies 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
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01731600
- Sponsor's protocol ID
- NN7088-3885
- Other IDs
- U1111-1129-6009, 2012-001711-23, JapicCTI-132214
- Acronym
- pathfinder™5
- Status last verified
- Nov 2020
- First submitted
- 9 Nov 2012
- Last updated
- 23 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Tosetto A, Neff A, Lentz SR, Santagostino E, Nemes L, Sathar J, Meijer K, Chowdary P, Shen C, Landorph A, Hampton K. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials. Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.
PubMed 32293786Saulyte Trakymiene S, Economou M, Kenet G, Landorph A, Shen C, Kearney S. Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5. J Thromb Haemost. 2020 Sep;18 Suppl 1(Suppl 1):15-25. doi: 10.1111/jth.15036.
PubMed 32940955
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
- ClinicalTrials.gov ID
- NCT01731600
- Acronym / sponsor ID
- pathfinder™5 / NN7088-3885
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 20 Feb 2013 (actual)
- Primary completion
- 15 Sep 2014 (actual)
- Study completion
- 28 Sep 2018 (actual)
- First posted
- 22 Nov 2012 (estimated)
- Results
- Posted 30 Oct 2019
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 globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| N8-GP | Experimental | Drug: turoctocog alfa pegol |
Who may qualify
- Ages
- 0 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
- Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years
Key exclusion criteria (1 listed)
- Any history of FVIII inhibitors
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 alfa pegolAlso: NNC 0129-0000-1003, N8-GPFixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight. | Drug | N8-GP |
Primary outcome
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units
Time frame: During the main phase of the trial (from 0-26 weeks of treatment)
The number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) ≥0.6 Bethesda units was presented.
16 secondary outcomes
Frequency of Adverse Events Including Serious Adverse Events Reported During the Trial Period
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes and Assessed as: Excellent, Good, Moderate, or None
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Number of Bleeding Episodes During Prophylactic Treatment With N8-GP (Annualised Bleeding Rate)
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP Per Bleeding Episode (Number of Injections)
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP Per Bleeding Episode (U/kg)
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP During Prophylaxis (Number of Injections)
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP During Prophylaxis (U/kg Per Month)
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Consumption of N8-GP During Prophylaxis (U/kg Per Year)
Time frame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for Previous FVIII Product
Time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for N8-GP
Time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Area Under the Curve Evaluated for Previous FVIII Product
Time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Area Under the Curve Evaluated for N8-GP
Time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Terminal Half-life Evaluated for Previous FVIII Product
Time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Terminal Half-life Evaluated for N8-GP
Time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Clearance Evaluated for Previous FVIII Product
Time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
Clearance Evaluated for N8-GP
Time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 32293786Tosetto A, Neff A, Lentz SR, Santagostino E, Nemes L, Sathar J, Meijer K, Chowdary P, Shen C, Landorph A, Hampton K. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials. Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.
- Results publication: PubMed 32940955Saulyte Trakymiene S, Economou M, Kenet G, Landorph A, Shen C, Kearney S. Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5. J Thromb Haemost. 2020 Sep;18 Suppl 1(Suppl 1):15-25. doi: 10.1111/jth.15036.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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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.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
51 sites in 16 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 51 sites in 16 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 1 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: finished, as of the sponsor's last update on 23 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in United States, Turkey (Türkiye), France, Switzerland, United Kingdom and 11 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 30 Oct 2019). 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 Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (time frame: During the main phase of the trial (from 0-26 weeks of treatment)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01731600, which also lists 2 linked publications.
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 23 Nov 2020.