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Completed

NCT Number: NCT02962765

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

Male

Study type

Observational

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. 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)

    Total number of bleeding episodes under prophylaxis treatment divided by the duration of prophylactic phase (in years)

  2. 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)

    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.

  3. Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians

    Time frame: From start of surgery until end of post-operative period

    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'.

  4. 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)

    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.

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study 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

Important dates

Study start
2015
Primary completion
2020
Study completion
2020
First posted
Nov 11, 2016
Registry last updated
Oct 21, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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