Clinical trial NCT02962765: Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2016-11-11
Enrollment: 80
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Number of Patients With FVIII Inhibitors
timeFrame: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)
measure: Number of Patients With Adverse Drug Reactions
timeFrame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Secondary outcomes:
measure: Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment
timeFrame: Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months)
measure: Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
timeFrame: Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
measure: Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians
timeFrame: From start of surgery until end of post-operative period
measure: Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
timeFrame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: New Orleans
country: United States
facility: Tulane University
state: Louisiana
city: Las Vegas
country: United States
facility: Hemophilia Treatment Center of Nevada
state: Nevada
city: Houston
country: United States
facility: Gulf States Hemophilia and Thrombophilia
state: Texas
city: Córdoba
country: Argentina
facility: Centro de Tratamiento de la Hemofilia Cordoba
city: Córdoba
country: Argentina
facility: CTH Centro de Tratamiento de Hematologia y Hemoterapia Córdoba S.A.
city: Salta
country: Argentina
facility: Fundación de Hemofilia de Salta
city: Santiago del Estero
country: Argentina
facility: Centro Mayo
city: Borovlyany
country: Belarus
facility: Belarusian Research Center for Pediatric Oncology, Hematology and Immunology
city: Brno
country: Czechia
facility: Fakultní nemocnice Brno
city: Ostrava
country: Czechia
facility: Blood Centre, University Hospital
city: Guayaquil
country: Ecuador
facility: Hospital de Especialidades Teodoro Maldonado Carbo
city: Nantes
country: France
facility: CHU Hôtel Dieu
city: Rennes
country: France
facility: Hopital Pontchaillou
city: Saint-Priest-en-Jarez
country: France
facility: CHRU Hôpital Nord
city: Toulouse
country: France
facility: CRTH, Hopital Purpan
city: Guatemala
country: Guatemala
facility: Pedias Inc. Centro Hospitalario La Paz
city: Bari
country: Italy
facility: L'Azienda Ospedaliero Universitaria Consorziale Policlinico, U.O. di Medicina Trasfusionale, Centro Emofilia e Trombosi
city: Castelfranco Veneto
country: Italy
facility: U.O.C. Ematologia, Ospedale San Giacomo Apostolo
city: Firenze
country: Italy
facility: UOC Malattie emorragiche e della coagulazione, Azienda Ospedaliera Universitaria Careggi
city: Milan
country: Italy
facility: Fondazione IRCCS Ca Granda
city: Naples
country: Italy
facility: AOU Federico II - Dipartimento di Medicina Clinica e Chirurgica
city: Naples
country: Italy
facility: Azienda Sanitaria Locale Napoli 1 Centro
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Palermo
country: Italy
facility: AOU Policlinico di Palermo
city: Palermo
country: Italy
facility: Ospedale ARNAS Civico
city: Perugia
country: Italy
facility: Dipartimento Di Medicina dell'Universita degli Studi di Perugia
city: Rome
country: Italy
facility: Dipartimento di Biotecnologie Cellulari ed Ematologia -"Sapienza" Università di Roma
city: Torino
country: Italy
facility: A.O. Città della Salute e della Scienza di Torino - Ospedale Regina Margherita
city: Torino
country: Italy
facility: S.C. Ematologia U, A.O.U. Città della Salute e della Scienza di Torino
city: Vilnius
country: Lithuania
facility: Vilnius University Hospital, Santariskiu Klinikos-Children's Hospital
city: Oslo
country: Norway
facility: Oslo University Hospital
city: Covilhã
country: Portugal
facility: Centro Hospitalar Cova da Beira
city: Bratislava
country: Slovakia
facility: National Haemophilia Center
city: London
country: United Kingdom
facility: Great Ormond Street Hospital (GOSH)
city: London
country: United Kingdom
facility: St. Thomas' Hospital
city: London
country: United Kingdom
facility: The Royal London Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals NHS Trust
Study officials:
affiliation: Great Ormond Street Hospital
name: Kate Khair, PhD
role: PRINCIPAL_INVESTIGATOR
References: