Genotype and Adeno-Associated Virus (AAV) Immune Status of Patients with Inherited Blood Diseases (Haemophilia A and B)

Not Applicable

• Conditions: Haemophilia A; Haemophilia B; Blood - Clotting disorders; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12617001099314
• Lead Sponsor: Department of Cell & Molecular Therapies, Royal Prince Alfred Hospital

Brief Summary

This study planned to recruit 30 participants. However, recruitment had to cease due to a halt in testing at the central laboratory. A total of 16 participants were recruited but one participant was excluded as their sample was not analysed by the central laboratory. Seven participants had haemophilia A and eight participants had haemophilia B. Seven of the study participants did not have AAV neutralising antibodies.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-07-28
• Study Completion: 2020-07-06
• Last Update Posted: 15 November 2021

Recruitment & Eligibility

• Status: Stopped early
• Sex: Male
• Target Recruitment: 15

Inclusion Criteria

1. Male of 18 years or older.
2. Hereditary blood disease, including Haemophilia A or B.
3. Able to provide informed consent according to the guidelines of the Sydney Local Health District Ethics Review Committee.

Exclusion Criteria

1. Subjects with a blood disease not due to a genetic mutation.
2. Subjects with less than 1-year life expectancy.
3. Investigator judgment that subject will be unable to comply with study endpoints.

Study & Design

• Study Type: Observational
• Study Design: Not specified