MedDataX Logo

Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ

NCT ID: NCT02962765

Last Updated: 2021-10-21

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

80 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-01-31

Study Completion Date

2020-08-20

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hemophilia A

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Octapharma

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Kate Khair, PhD

Role: PRINCIPAL_INVESTIGATOR

Great Ormond Street Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of Florida

Gainesville, Florida, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Tulane University

New Orleans, Louisiana, United States

Site Status

Hemophilia Treatment Center of Nevada

Las Vegas, Nevada, United States

Site Status

Gulf States Hemophilia and Thrombophilia

Houston, Texas, United States

Site Status

Centro de Tratamiento de la Hemofilia Cordoba

Córdoba, , Argentina

Site Status

CTH Centro de Tratamiento de Hematologia y Hemoterapia Córdoba S.A.

Córdoba, , Argentina

Site Status

Fundación de Hemofilia de Salta

Salta, , Argentina

Site Status

Centro Mayo

Santiago del Estero, , Argentina

Site Status

Belarusian Research Center for Pediatric Oncology, Hematology and Immunology

Borovlyany, , Belarus

Site Status

Fakultní nemocnice Brno

Brno, , Czechia

Site Status

Blood Centre, University Hospital

Ostrava, , Czechia

Site Status

Hospital de Especialidades Teodoro Maldonado Carbo

Guayaquil, , Ecuador

Site Status

CHU Hôtel Dieu

Nantes, , France

Site Status

Hopital Pontchaillou

Rennes, , France

Site Status

CHRU Hôpital Nord

Saint-Priest-en-Jarez, , France

Site Status

CRTH, Hopital Purpan

Toulouse, , France

Site Status

Pedias Inc. Centro Hospitalario La Paz

Guatemala City, , Guatemala

Site Status

L'Azienda Ospedaliero Universitaria Consorziale Policlinico, U.O. di Medicina Trasfusionale, Centro Emofilia e Trombosi

Bari, , Italy

Site Status

U.O.C. Ematologia, Ospedale San Giacomo Apostolo

Castelfranco Veneto, , Italy

Site Status

UOC Malattie emorragiche e della coagulazione, Azienda Ospedaliera Universitaria Careggi

Florence, , Italy

Site Status

Fondazione IRCCS Ca Granda

Milan, , Italy

Site Status

AOU Federico II - Dipartimento di Medicina Clinica e Chirurgica

Naples, , Italy

Site Status

Azienda Sanitaria Locale Napoli 1 Centro

Naples, , Italy

Site Status

Azienda Ospedaliera di Padova

Padua, , Italy

Site Status

AOU Policlinico di Palermo

Palermo, , Italy

Site Status

Ospedale ARNAS Civico

Palermo, , Italy

Site Status

Dipartimento Di Medicina dell'Universita degli Studi di Perugia

Perugia, , Italy

Site Status

Dipartimento di Biotecnologie Cellulari ed Ematologia -"Sapienza" Università di Roma

Rome, , Italy

Site Status

A.O. Città della Salute e della Scienza di Torino - Ospedale Regina Margherita

Torino, , Italy

Site Status

S.C. Ematologia U, A.O.U. Città della Salute e della Scienza di Torino

Torino, , Italy

Site Status

Vilnius University Hospital, Santariskiu Klinikos-Children's Hospital

Vilnius, , Lithuania

Site Status

Oslo University Hospital

Oslo, , Norway

Site Status

Centro Hospitalar Cova da Beira

Covilha, , Portugal

Site Status

National Haemophilia Center

Bratislava, , Slovakia

Site Status

Great Ormond Street Hospital (GOSH)

London, , United Kingdom

Site Status

St. Thomas' Hospital

London, , United Kingdom

Site Status

The Royal London Hospital

London, , United Kingdom

Site Status

Nottingham University Hospitals NHS Trust

Nottingham, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Argentina Belarus Czechia Ecuador France Guatemala Italy Lithuania Norway Portugal Slovakia United Kingdom

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

GENA-99

Identifier Type: -

Identifier Source: org_study_id