Hemophilia ANCT02962765
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
An observational study of Hemophilia A, run by Octapharma at 39 sites in 13 countries.
Trial summary
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 80 participants
- Where
- 39 sitesin 13 countries
- Study dates
- Jan 2015to 20 Aug 2020
- Phase
- Observational
- 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
Study population: The goal is to collect data on 200 previously treated male patients of any age with haemophilia (FVIII:C ≤ 2%). Patients from pre-authorisation studies can be followed up to at least 100 EDs. Newly enrolled patients have to be treated and followed for at least 100 EDs. * Of the 200 enrolled patients, at least 100 patients should have severe haemophilia A (FVIII:C < 1%). * Of the 200 enrolled patients, approx. 60 patients should be < 12 years of age. At least 10 patients should be aged between 14-18 years. * Patients with severe haemophilia A after successful immune tolerance induction (ITI) can also be included; the proportion of these ITI patients should not exceed 25% of the entire cohort.
Who may be able to join
- Haemophilia A (FVIII:C ≤ 2%) based on medical history; at least 100 patients should have severe haemophilia A (FVIII:C < 1%)
- Male patients of any age
- Previous treatment with a FVIII concentrate for more than 150 EDs
- Availability of detailed documentation (patient diary, log book, etc.) covering either the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e., prophylaxis, on-demand, recent surgery, or immune tolerance induction)
- Inhibitor negative (< 0.6 BU) at study entry as confirmed by a recovery test with previous FVIII product and inhibitor test in a central laboratory
- Immunocompetence (CD4+ count > 200/µL), HIV-negative, or having a viral load < 200 particles/µL or < 400,000 copies/mL
- Decision to prescribe Human-cl rhFVIII before enrolment into the study
- Written informed consent by the patient or the patient's parent or legal guardian
Who cannot join
- Patients treated with any investigational medicinal product (IMP) except FVIII IMP within 30 days prior to the Screening Visit or patients planning to undergo treatment with any IMP other than Human-cl rhFVIII are not eligible for enrolment into the study.
Trial locations and contacts
39 sites in 13 countries.
39 sites in 13 countries
Italy13 sites
L'Azienda Ospedaliero Universitaria Consorziale Policlinico, U.O. di Medicina Trasfusionale, Centro Emofilia e Trombosi
Bari
U.O.C. Ematologia, Ospedale San Giacomo Apostolo
Castelfranco Veneto
UOC Malattie emorragiche e della coagulazione, Azienda Ospedaliera Universitaria Careggi
Florence
Fondazione IRCCS Ca Granda
Milan
AOU Federico II - Dipartimento di Medicina Clinica e Chirurgica
Naples
Azienda Sanitaria Locale Napoli 1 Centro
Naples
Azienda Ospedaliera di Padova
Padova
AOU Policlinico di Palermo
Palermo
Ospedale ARNAS Civico
Palermo
Dipartimento Di Medicina dell'Universita degli Studi di Perugia
Perugia
Dipartimento di Biotecnologie Cellulari ed Ematologia -"Sapienza" Università di Roma
Rome
A.O. Città della Salute e della Scienza di Torino - Ospedale Regina Margherita
Torino
S.C. Ematologia U, A.O.U. Città della Salute e della Scienza di Torino
Torino
United States5 sites
University of Florida
Gainesville, Florida, 32610
Gulf States Hemophilia and Thrombophilia
Houston, Texas, 77030
Hemophilia Treatment Center of Nevada
Las Vegas, Nevada, 89109
Nicklaus Children's Hospital
Miami, Florida, 33155
Tulane University
New Orleans, Louisiana, 70112
Argentina4 sites
Centro de Tratamiento de la Hemofilia Cordoba
Córdoba
CTH Centro de Tratamiento de Hematologia y Hemoterapia Córdoba S.A.
Córdoba
Fundación de Hemofilia de Salta
Salta
Centro Mayo
Santiago del Estero
France4 sites
CHU Hôtel Dieu
Nantes
Hopital Pontchaillou
Rennes
CHRU Hôpital Nord
Saint-Priest-en-Jarez
CRTH, Hopital Purpan
Toulouse
United Kingdom4 sites
Great Ormond Street Hospital (GOSH)
London
St. Thomas' Hospital
London
The Royal London Hospital
London
Nottingham University Hospitals NHS Trust
Nottingham
Czechia2 sites
Fakultní nemocnice Brno
Brno
Blood Centre, University Hospital
Ostrava
Belarus1 site
Belarusian Research Center for Pediatric Oncology, Hematology and Immunology
Borovlyany
Ecuador1 site
Hospital de Especialidades Teodoro Maldonado Carbo
Guayaquil, 090203
Lithuania1 site
Vilnius University Hospital, Santariskiu Klinikos-Children's Hospital
Vilnius
About this study
Official title: Prospective, Multinational, Non-interventional Post-authorisation Study to Document the Long-term Immunogenicity, Safety, and Efficacy of Human-cl rhFVIII (Simoctocog Alfa) in Patients With Haemophilia A Treated in Routine Clinical Practice
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
- Conditions
- Hemophilia A
- Medical subject headings
- Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With FVIII Inhibitors
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Measured: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Number of Patients With Adverse Drug Reactions
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.
Measured: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Secondary outcomes (4)
Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment
Total number of bleeding episodes under prophylaxis treatment divided by the duration of prophylactic phase (in years)
Measured: Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months)
Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent,' 'good,' moderate,' and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Measured: Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians
At the end of the postoperative period, an overall assessment of the efficacy of treatment in the pre-, peri-, and postoperative periods using the 'excellent,' 'good,' moderate,' and 'none' scale will be done jointly by the surgeon and the hematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Measured: From start of surgery until end of post-operative period
Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent', 'good', 'moderate', and 'none.'
Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Measured: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2016 (estimated)
- Study startJan 2015
- Primary completion20 Aug 2020
- Study completion20 Aug 2020
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 Hemophilia AStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kate Khair, PhD, Principal investigator, Great Ormond Street Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02962765
- Sponsor's protocol ID
- GENA-99
- Status last verified
- Sep 2021
- First submitted
- 8 Nov 2016
- Last updated
- 21 Oct 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Multinational, Non-interventional Post-authorisation Study to Document the Long-term Immunogenicity, Safety, and Efficacy of Human-cl rhFVIII (Simoctocog Alfa) in Patients With Haemophilia A Treated in Routine Clinical Practice
- ClinicalTrials.gov ID
- NCT02962765
- Acronym / sponsor ID
- GENA-99
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Jan 2015
- Primary completion
- 20 Aug 2020 (actual)
- Study completion
- 20 Aug 2020 (actual)
- First posted
- 11 Nov 2016 (estimated)
- Results
- Posted 28 Sep 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The goal is to collect data on 200 previously treated male patients of any age with haemophilia (FVIII:C ≤ 2%). Patients from pre-authorisation studies can be followed up to at least 100 EDs. Newly enrolled patients have to be treated and followed for at least 100 EDs. * Of the 200 enrolled patients, at least 100 patients should have severe haemophilia A (FVIII:C < 1%). * Of the 200 enrolled patients, approx. 60 patients should be < 12 years of age. At least 10 patients should be aged between 14-18 years. * Patients with severe haemophilia A after successful immune tolerance induction (ITI) can also be included; the proportion of these ITI patients should not exceed 25% of the entire cohort.
Key inclusion criteria (8 listed)
- Haemophilia A (FVIII:C ≤ 2%) based on medical history; at least 100 patients should have severe haemophilia A (FVIII:C < 1%)
- Male patients of any age
- Previous treatment with a FVIII concentrate for more than 150 EDs
- Availability of detailed documentation (patient diary, log book, etc.) covering either the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e., prophylaxis, on-demand, recent surgery, or immune tolerance induction)
- Inhibitor negative (< 0.6 BU) at study entry as confirmed by a recovery test with previous FVIII product and inhibitor test in a central laboratory
Key exclusion criteria (1 listed)
- Patients treated with any investigational medicinal product (IMP) except FVIII IMP within 30 days prior to the Screening Visit or patients planning to undergo treatment with any IMP other than Human-cl rhFVIII are not eligible for enrolment into the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of Patients With FVIII Inhibitors
Time frame: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Number of Patients With Adverse Drug Reactions
Time frame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.
4 secondary outcomes
Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment
Time frame: Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months)
Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
Time frame: Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians
Time frame: From start of surgery until end of post-operative period
Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
Time frame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Kate Khair, PhD principal investigator
Great Ormond Street Hospital
39 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Octapharma at 39 sites in 13 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 8 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 21 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in Italy, United States, Argentina, France, United Kingdom and 8 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Sep 2021). 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 Patients With FVIII Inhibitors (time frame: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)). The study lists 2 primary outcomes in all. 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 21 Oct 2021.