Trial record 1 of 1 for:    GENA-99
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Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02962765
Recruitment Status : Recruiting
First Posted : November 11, 2016
Last Update Posted : August 22, 2018
Information provided by (Responsible Party):

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.

Condition or disease
Hemophilia A

Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
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 Start Date : January 2015
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Primary Outcome Measures :
  1. Incidence of FVIII inhibitors [ Time Frame: 100 exposure days ]
    FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.

  2. Incidence of adverse drug reactions [ Time Frame: 100 exposure days ]
    Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.

Secondary Outcome Measures :
  1. Annualized rate of breakthrough bleeds to assess efficacy in prophylactic treatment [ Time Frame: 100 exposure days ]
    The occurrence of bleeding events during the study will documented in the treatment diary.

  2. Assessment of the efficacy of on-demand treatment [ Time Frame: 100 exposure days ]
    The treatment of bleeding episodes (BE) will be assessed by 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 efficacy scale: 'excellent', 'good', 'moderate', 'none'. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.

  3. Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians [ Time Frame: 100 exposure days ]
    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 haematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.

Biospecimen Retention:   Samples Without DNA
Citrate Plasma for measuring FVIII inhibitors is recommended throughout the study.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02962765

Contact: Sigurd Knaub, PhD 41 55 451 2141

United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Nicklaus Children's Hospital Recruiting
Miami, Florida, United States, 33155
United States, Louisiana
Tulane University Recruiting
New Orleans, Louisiana, United States, 70112
United States, Nevada
Hemophilia Treatment Center of Nevada Recruiting
Las Vegas, Nevada, United States, 89109
United States, Texas
Gulf States Hemophilia and Thrombophilia Recruiting
Houston, Texas, United States, 77030
Fundación de Hemofilia de Salta Recruiting
Salta, Argentina
Centro Mayo Recruiting
Santiago del Estero, Argentina
Belarusian Research Center for Pediatric Oncology, Hematology and Immunology Recruiting
Borovlyany, Belarus
L'Azienda Ospedaliero Universitaria Consorziale Policlinico, U.O. di Medicina Trasfusionale, Centro Emofilia e Trombosi Recruiting
Bari, Italy
U.O.C. Ematologia, Ospedale San Giacomo Apostolo Recruiting
Castelfranco Veneto, Italy
UOC Malattie emorragiche e della coagulazione, Azienda Ospedaliera Universitaria Careggi Recruiting
Firenze, Italy
Fondazione IRCCS Ca Granda Recruiting
Milan, Italy
AOU Federico II - Dipartimento di Medicina Clinica e Chirurgica Recruiting
Naples, Italy
Azienda Ospedaliera di Padova Recruiting
Padova, Italy
Ospedale ARNAS Civico Not yet recruiting
Palermo, Italy
Dipartimento Di Medicina dell'Universita degli Studi di Perugia Recruiting
Perugia, Italy
Dipartimento di Biotecnologie Cellulari ed Ematologia -"Sapienza" Università di Roma Recruiting
Rome, Italy
S.C. Ematologia U, A.O.U. Città della Salute e della Scienza di Torino Recruiting
Torino, Italy
Oslo University Hospital Recruiting
Oslo, Norway
United Kingdom
Great Ormond Street Hospital (GOSH) Recruiting
London, United Kingdom
St. Thomas' Hospital Recruiting
London, United Kingdom
The Royal London Hospital Recruiting
London, United Kingdom
Nottingham University Hospitals NHS Trust Recruiting
Nottingham, United Kingdom
Sponsors and Collaborators
Principal Investigator: Kate Khair, PhD Great Ormond Street Hospital

Responsible Party: Octapharma Identifier: NCT02962765     History of Changes
Other Study ID Numbers: GENA-99
First Posted: November 11, 2016    Key Record Dates
Last Update Posted: August 22, 2018
Last Verified: February 2018

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn