A clinical study to learn about the effects of a virus that transfers the gene for human coagulation factor IX in adults with moderate/severe to severe inherited coagulations defects in the long term

Phase 1

• Conditions: Moderate/severe to severe hemophilia B; MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-003430-25-GB
• Lead Sponsor: ltragenyx Pharmaceutical, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-03-13
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 6

Inclusion Criteria

1. Willing and able to provide written informed consent

2. Completed the Cohort 1/Week 52 or Cohort 2/week 44 visit in Study 101HEMB01

3. Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable

4. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements
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 5
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1. Planned or current participation in another interventional clinical study that may confound the safety or efficacy evaluation of DTX101 during the duration of this study

2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study

Study & Design

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