An international investigator trial to capture different approaches in the treatment of Haemophilia A Patients With FVIII Inhibitors

Phase 1

• Conditions: Inhibitor-Positive patients with Haemophilia A; MedDRA version: 20.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2019-003427-38-SE
• Lead Sponsor: HZRM – Hämophilie-Zentrum Rhein Main GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-12-17
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 120

Inclusion Criteria

1. Participants can be of any age at the time of enrolment
2. Persons with HA, of any severity, who have a historical inhibitor titre = 0.6 BU/mL, including those who have failed previous ITI attempt(s)
3. Persons undergoing ITI with Nuwiq®, octanate® or wilate® and/or receiving prophylactic therapy with emicizumab, aPCC or rFVIIa
4. Male
5. Participants or participants’ parent(s)/legal guardian(s) must be capable of giving signed informed consent and able to understand the trial documents

Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

- Participants are excluded from the trial if any coagulation disorder other than HA is diagnosed
- Partly retrospective patients will be excluded if detailed documentation on treatment, all BEs, inhibitor titres and FVIII levels is not available for the retrospective period

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified