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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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2016 by Octapharma
Sponsor:
Information provided by (Responsible Party):
Octapharma
ClinicalTrials.gov Identifier:
NCT02962765
First received: November 8, 2016
Last updated: November 9, 2016
Last verified: November 2016
  Purpose
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
Hemophilia A

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Octapharma:

Primary Outcome Measures:
  • 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

  • Incidence of adverse drug reactions [ Time Frame: 100 exposure days ]

Secondary Outcome Measures:
  • Annualized rate of breakthrough bleeds to assess efficacy in prophylactic [ Time Frame: 100 exposure days ]
  • Efficacy assessment by the patient to assess efficacy in on-demand treatment [ Time Frame: 100 exposure days ]
    The treatment of BE (bleeding episodes) will be assessed by using a 4 point efficacy scale: excellent, good, moderate, none. Excellent and good ratings will be considered "successfully treated".

  • Overall assessment of the effectiveness of surgical prophylaxis by the treating physician [ Time Frame: 100 exposure days ]

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

Estimated Enrollment: 200
Study Start Date: January 2015
Estimated Study Completion Date: March 2020
Estimated Primary Completion Date: June 2019 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
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.
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02962765

Contacts
Contact: Cristina Solomon, MD 41 55 451 2189 Cristina.Solomon@octapharma.com

Locations
United States, Nevada
Hemophilia Treatment Center of Nevada Recruiting
Las Vegas, Nevada, United States, 89109
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
Sponsors and Collaborators
Octapharma
Investigators
Principal Investigator: Kate Khair, PhD Great Ormond Street Hospital
  More Information

Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02962765     History of Changes
Other Study ID Numbers: GENA-99
Study First Received: November 8, 2016
Last Updated: November 9, 2016

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

ClinicalTrials.gov processed this record on April 21, 2017