Severe Hemophilia APhase 3NCT01992549

Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

A Phase 3 study testing Human-cl rhFVIII in people with Severe Hemophilia A, run by Octapharma at 15 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A. Read more about this study

Who is it for?: Any age, men only
Conditions the study is for: Severe Hemophilia A
What is being tested?: Human-cl rhFVIII
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 48 participants
Where: 15 sitesin 9 countries
Study dates: Apr 2014to 27 Dec 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 Any age
Men only
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

1. Patients who completed GENA-05 in accordance with the study protocol

Who cannot join

Severe liver or kidney disease
Concomitant treatment with any systemic immunosuppressive drug;
Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

Trial locations and contacts

15 sites in 9 countries.

Country

City, postcode or hospital

15 sites in 9 countries

Canada4 sites

University of Alberta
Edmonton, Alberta
McMaster Children's Hospital
Hamilton, Ontario, L8S4K1
Hospital for Sick Children
Toronto
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4

Other trials in Canada

France2 sites

Hopital de la Timone
Marseille
Hôpital Kremlin Bicètre
Paris

Other trials in France

India2 sites

Sahyadri Speciality Hospital
Pune, 411004
Christian Medical College
Vellore, 632004

Other trials in India

Ukraine2 sites

The National Children Specialized Hospital "OHMATDET"
Kiev
Danylo Halytsky Lviv National Medical University
Lviv

Other trials in Ukraine

United States1 site

UC Davis Medical Center
Sacramento, California, 95817

Other trials in United States

Georgia1 site

Institute of Hematology and Transfusiology
Tbilisi

Other trials in Georgia

Moldova1 site

IMSP Mother and Child Institute
Chisinau

Other trials in Moldova

Poland1 site

University Medical School
Warsaw

Other trials in Poland

United Kingdom1 site

Great Ormond Street Hospital for Children
London, WC1N 3JH

Other trials in United Kingdom

About this study

Official title: Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

Conditions: Severe Hemophilia A
Being tested: Human-cl rhFVIII
Medical subject headings: Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Human-cl rhFVIIIBiological

Study groups in NCT01992549
Study group	Role	What they receive
Human-cl rhFVIII	Experimental	Not described Receives: Biological: Human-cl rhFVIII

What is being measured

The primary outcome is the main question the study is designed to answer.

Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.
Measured: Maximum two years

Secondary outcomes (4)

Frequency of Spontaneous Break-through Bleeds
The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).
Measured: Maximum 2 years
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.
Measured: Maximum 2 years
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".
Measured: Maximum 2 years
The Occurrence of Any Adverse Event (AE)
The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.
Measured: Maximum 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Apr 201427 Dec 2018

First posted25 Nov 2013 (estimated)
Study startApr 2014
Primary completion27 Dec 2018
Study completion27 Dec 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.
Other trials for Severe Hemophilia AStudies of the same condition, open and under way.
More trials run by OctapharmaThe 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: 48 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01992549
Sponsor's protocol ID: GENA-15
Status last verified: Dec 2020
First submitted: 19 Nov 2013
Last updated: 19 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
Not yet recruitingObservational study
Post Approval Phase 4 Study to Learn More About How Safe Octocog Alfa is, and How Well it Works in Patients With Severe Hemophilia A in India
Severe Hemophilia A

All Severe Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
ClinicalTrials.gov ID: NCT01992549
Acronym / sponsor ID: GENA-15
Conditions: Severe Hemophilia A
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 48 participants (actual)
Lead sponsor: OctapharmaIndustry (pharma, biotech, devices)
Study start: Apr 2014
Primary completion: 27 Dec 2018 (actual)
Study completion: 27 Dec 2018 (actual)
First posted: 25 Nov 2013 (estimated)
Results: Posted 17 Dec 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Human-cl rhFVIII	Experimental	Biological: Human-cl rhFVIII

Who may qualify

Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (1 listed)

1. Patients who completed GENA-05 in accordance with the study protocol

Key exclusion criteria (3 listed)

Severe liver or kidney disease
Concomitant treatment with any systemic immunosuppressive drug;
Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

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
Human-cl rhFVIII	Biological	Human-cl rhFVIII

Primary outcome

Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
Time frame: Maximum two years
The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.

4 secondary outcomes

Frequency of Spontaneous Break-through Bleeds
Time frame: Maximum 2 years
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
Time frame: Maximum 2 years
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
Time frame: Maximum 2 years
The Occurrence of Any Adverse Event (AE)
Time frame: Maximum 2 years

Results and safety

Results posted to the registry on 17 Dec 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Human-cl rhFVIII	48	44

Primary outcome: Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors

Time frame: Maximum two years. Number (95% Confidence Interval). Unit: participants

Measure	Human-cl rhFVIII n = 48
Value	0 (0.000 to 7.549)

Population analysed: The analysis was performed for the safety (SAF) population which includes all patients who received at least 1 infusion of Human-cl rhFVIII (N=48)

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Maximum 2 years. Other adverse events reported at 2.08% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
SAF Population	5 of 48 (10.4%)	29 of 48 (60.4%)	0 of 48

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Severe Hemophilia A

RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
Not yet recruitingObservational study
Post Approval Phase 4 Study to Learn More About How Safe Octocog Alfa is, and How Well it Works in Patients With Severe Hemophilia A in India
Severe Hemophilia A

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.

Sigurd Knaub, PhD study director

Octapharma

15 sites in 9 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Human-cl rhFVIII in people with Severe Hemophilia A, run by Octapharma at 15 sites in 9 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 1 inclusion and 3 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 19 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in Canada, France, India, Ukraine, United States and 4 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 17 Dec 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?

Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors (time frame: Maximum two years). 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 NCT01992549.

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 19 Jan 2021.