Congenital Bleeding DisorderPhase 1alleviate 1NCT02994407
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
A Phase 1 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 19 sites in 6 countries.
Trial summary
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A. Read more about this study
- Who is it for?
- Ages 12 years and older, 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
- 50 participants
- Where
- 19 sitesin 6 countries
- Study dates
- 30 Jan 2017to 15 Oct 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Male, age above or equal to 18 years at the time of signing informed consent,(part A).
- Male, age above or equal to 12 years at the time of signing informed consent,(part B).
- Diagnosis of congenital haemophilia A based on medical records (FVIII activity <1%).
- History of more than 150 exposure days to any FVIII containing products.
Who cannot join
- Previous participation in this trial. Participation is defined as signed informed consent.
- (Patients who have completed part A are allowed to also participate in part B. If so, a separate informed consent covering part B must be signed.)
- Immune compromised patients due to human immunodeficiency virus (HIV) infection (defined as viral load greater than or equal to 400.000 copies/mL and/or cluster of differentiation 4+ (CD4+) lymphocyte count less than or equal to 200/μL performed at screening or defined by medical records no older than 6 months)
- Any history of FVIII inhibitors (defined by medical records within 8 years of randomisation)
- Inhibitors to FVIII (greater than or equal to 0.6 Bethesda unit (BU)) at screening, measured by Nijmegen modified Bethesda method at central laboratory.
Trial locations and contacts
19 sites in 6 countries.
19 sites in 6 countries
United States8 sites
Novo Nordisk Investigational Site
Charleston, South Carolina, 29425-0001
Novo Nordisk Investigational Site
Cleveland, Ohio, 44106
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
Novo Nordisk Investigational Site
East Lansing, Michigan, 48823
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Milwaukee, Wisconsin, 53226
Novo Nordisk Investigational Site
Norfolk, Virginia, 23507
Novo Nordisk Investigational Site
Orlando, Florida, 32827
Germany3 sites
Novo Nordisk Investigational Site
Berlin, 10249
Novo Nordisk Investigational Site
Duisburg, 47051
Novo Nordisk Investigational Site
Homburg, 66421
Serbia3 sites
Novo Nordisk Investigational Site
Belgrade, 11000
Novo Nordisk Investigational Site
Belgrade, 11070
Novo Nordisk Investigational Site
Novi Sad, 21000
Austria2 sites
Novo Nordisk Investigational Site
Innsbruck, A 6020
Novo Nordisk Investigational Site
Vienna, 1090
Japan2 sites
Novo Nordisk Investigational Site
Shinjuku-ku, Tokyo, 160 0023
Novo Nordisk Investigational Site
Tokyo, 167-0035
About this study
Official title: Safety, Tolerability, and Pharmacokinetics Study of Single and Multiple Subcutaneous Doses of Turoctocog Alfa Pegol in Patients With Haemophilia A
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
- 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
Part A: Participants will receive a single dose of turoctocog alfa pegol, administered subcutaneously (under the skin), at a dose of 12.5, 25 or 50 U/kg.
Part B: Participants will receive a daily dose of turoctocog alfa pegol, as identified in Part A, as a subcutaneous (under the skin) injection for a period of 3 months.
| Study group | Role | What they receive |
|---|---|---|
| N8-GP s.c. | 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 adverse events
Count and % of Adverse events
Measured: Day 0-Day 28
Secondary outcomes (25)
Cmax
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Incidence of FVIII inhibitors above or equal to 0.6 BU
Count of presence of inhibitors
Measured: Day 0-Day 28
Area under the activity time curve from 0 to infinity
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Area under the activity time curve from 0 to t
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Area under the activity time curve from 0 to last
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
tmax- time to maximal FVIII activity
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Cmin -the minimal FVIII activity
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
tmin - time to minimal FVIII activity
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Css, min - the minimum FVIII activity at steady state
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Css, max - the maximal FVIII activity at steady state
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Css - the mean FVIII activity at steady state
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Racc - accumulation ratio
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
t½ - terminal half-life
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
CL - total plasma clearance of drug after intravenous administration
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Vz -apparent volume of distribution during terminal phase
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Vss - apparent volume of distribution during steady state
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
MRT - mean residence time
Calculated based on plasma FVIII activity measured in blood.
Measured: 0-144 hours
Injection site reactions
Count of reactions
Measured: Day 0 - day 28
Number of treatment requiring bleeding episodes
Count of episodes
Measured: Day 0 - day 120
Consumption of FVIII
Measured in IU
Measured: Day 0 - day 120
Change in Coagulation parameters, fibrinogen
Measured in g/L
Measured: Day 0, day 7
Change in Coagulation parameters, antithrombin
Measured in %
Measured: Day 0, day 7
Change in Coagulation parameters, international normalised ratio
Measured in INR
Measured: Day 0, day 7
Change in Coagulation parameters, activated partial thromboplastin time
Measured in sec.
Measured: Day 0, day 7
Change in Coagulation parameters, von Willebrand Factor
Measured in %
Measured: Day 0, day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2016 (estimated)
- Study start30 Jan 2017
- Primary completion15 Oct 2018
- Study completion15 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02994407
- Sponsor's protocol ID
- NN7170-4213
- Other IDs
- U1111-1183-5111, 2016-002396-99, JapicCTI-173683, 2016
- Acronym
- alleviate 1
- Status last verified
- Jan 2020
- First submitted
- 13 Dec 2016
- Last updated
- 5 Feb 2020
- Results posted
- No
- 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
Publications
Klamroth R, Feistritzer C, Friedrich U, Lentz SR, Reichwald K, Zak M, Chowdary P. Pharmacokinetics, immunogenicity, safety, and preliminary efficacy of subcutaneous turoctocog alfa pegol in previously treated patients with severe hemophilia A (alleviate 1). J Thromb Haemost. 2020 Feb;18(2):341-351. doi: 10.1111/jth.14660. Epub 2019 Nov 15.
PubMed 31618804
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Tolerability, and Pharmacokinetics Study of Single and Multiple Subcutaneous Doses of Turoctocog Alfa Pegol in Patients With Haemophilia A
- ClinicalTrials.gov ID
- NCT02994407
- Acronym / sponsor ID
- alleviate 1 / NN7170-4213
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 30 Jan 2017 (actual)
- Primary completion
- 15 Oct 2018 (actual)
- Study completion
- 15 Oct 2018 (actual)
- First posted
- 15 Dec 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
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 s.c. | Experimental | Drug: turoctocog alfa pegol |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male, age above or equal to 18 years at the time of signing informed consent,(part A).
- Male, age above or equal to 12 years at the time of signing informed consent,(part B).
- Diagnosis of congenital haemophilia A based on medical records (FVIII activity <1%).
- History of more than 150 exposure days to any FVIII containing products.
Key exclusion criteria (5 listed)
- Previous participation in this trial. Participation is defined as signed informed consent.
- (Patients who have completed part A are allowed to also participate in part B. If so, a separate informed consent covering part B must be signed.)
- Immune compromised patients due to human immunodeficiency virus (HIV) infection (defined as viral load greater than or equal to 400.000 copies/mL and/or cluster of differentiation 4+ (CD4+) lymphocyte count less than or equal to 200/μL performed at screening or defined by medical records no older than 6 months)
- Any history of FVIII inhibitors (defined by medical records within 8 years of randomisation)
- Inhibitors to FVIII (greater than or equal to 0.6 Bethesda unit (BU)) at screening, measured by Nijmegen modified Bethesda method at central laboratory.
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 pegolPart A: Participants will receive a single dose of turoctocog alfa pegol, administered subcutaneously (under the skin), at a dose of 12.5, 25 or 50 U/kg. Part B: Participants will receive a daily dose of turoctocog alfa pegol, as identified in Part A, as a subcutaneous (under the skin) injection for a period of 3 months. | Drug | N8-GP s.c. |
Primary outcome
Number of adverse events
Time frame: Day 0-Day 28
Count and % of Adverse events
25 secondary outcomes
Cmax
Time frame: 0-144 hours
Incidence of FVIII inhibitors above or equal to 0.6 BU
Time frame: Day 0-Day 28
Area under the activity time curve from 0 to infinity
Time frame: 0-144 hours
Area under the activity time curve from 0 to t
Time frame: 0-144 hours
Area under the activity time curve from 0 to last
Time frame: 0-144 hours
tmax- time to maximal FVIII activity
Time frame: 0-144 hours
Cmin -the minimal FVIII activity
Time frame: 0-144 hours
tmin - time to minimal FVIII activity
Time frame: 0-144 hours
Css, min - the minimum FVIII activity at steady state
Time frame: 0-144 hours
Css, max - the maximal FVIII activity at steady state
Time frame: 0-144 hours
Css - the mean FVIII activity at steady state
Time frame: 0-144 hours
Racc - accumulation ratio
Time frame: 0-144 hours
t½ - terminal half-life
Time frame: 0-144 hours
CL - total plasma clearance of drug after intravenous administration
Time frame: 0-144 hours
Vz -apparent volume of distribution during terminal phase
Time frame: 0-144 hours
Vss - apparent volume of distribution during steady state
Time frame: 0-144 hours
MRT - mean residence time
Time frame: 0-144 hours
Injection site reactions
Time frame: Day 0 - day 28
Number of treatment requiring bleeding episodes
Time frame: Day 0 - day 120
Consumption of FVIII
Time frame: Day 0 - day 120
Change in Coagulation parameters, fibrinogen
Time frame: Day 0, day 7
Change in Coagulation parameters, antithrombin
Time frame: Day 0, day 7
Change in Coagulation parameters, international normalised ratio
Time frame: Day 0, day 7
Change in Coagulation parameters, activated partial thromboplastin time
Time frame: Day 0, day 7
Change in Coagulation parameters, von Willebrand Factor
Time frame: Day 0, day 7
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2020.
- Derived from this study: PubMed 31618804Klamroth R, Feistritzer C, Friedrich U, Lentz SR, Reichwald K, Zak M, Chowdary P. Pharmacokinetics, immunogenicity, safety, and preliminary efficacy of subcutaneous turoctocog alfa pegol in previously treated patients with severe hemophilia A (alleviate 1). J Thromb Haemost. 2020 Feb;18(2):341-351. doi: 10.1111/jth.14660. Epub 2019 Nov 15.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
19 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 19 sites in 6 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 4 inclusion and 5 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 5 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Germany, Serbia, Austria, Japan and 1 more country. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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: Day 0-Day 28). 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 NCT02994407, which also lists 1 linked publication.
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 5 Feb 2020.