A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Phase 1

• Conditions: Haemophilia B; MedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-005080-40-GB
• Lead Sponsor: Freeline Therapeutics Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-02-19
• Last Update Posted: 4 May 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 50

Inclusion Criteria

1. Patients who have previously received FLT180a within a clinical study.
2. Able to give full informed consent and able to comply with all requirements of the study including long-term follow-up for the timeframe the study requires.
3. Willing to practice barrier contraception until at least 3 consecutive semen samples after vector administration are negative for vector sequence*.
*only applicable if vector genome shedding in semen sample persists at end of study in the preceding clinical study where FLT180a is administered.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 35
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion Criteria

None, if both inclusion criteria are met

Study & Design

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