Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
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| ClinicalTrials.gov Identifier: NCT02962765 |
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Recruitment Status :
Recruiting
First Posted : November 11, 2016
Last Update Posted : December 6, 2019
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Sponsor:
Octapharma
Information provided by (Responsible Party):
Octapharma
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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 |
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| 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 : | July 2020 |
| Estimated Study Completion Date : | July 2020 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Hemophilia
MedlinePlus related topics:
Hemophilia
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 ]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 :
- 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.
- 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'.
- 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.
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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.
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.
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 ClinicalTrials.gov identifier (NCT number): NCT02962765
Contacts
| Contact: Sigurd Knaub, PhD | 41 55 451 2141 | sigurd.knaub@octapharma.com |
Locations
Show 35 study locations
Sponsors and Collaborators
Octapharma
Investigators
| Principal Investigator: | Kate Khair, PhD | Great Ormond Street Hospital |
| Responsible Party: | Octapharma |
| ClinicalTrials.gov Identifier: | NCT02962765 |
| Other Study ID Numbers: |
GENA-99 |
| First Posted: | November 11, 2016 Key Record Dates |
| Last Update Posted: | December 6, 2019 |
| Last Verified: | December 2019 |
Additional relevant MeSH terms:
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Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |