Clinical trial NCT02971969: Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Official title: A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia B
Keywords: gene therapy
Hemophilia B
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2016-11-23
Enrollment: 6
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01 clinical study

Brief summary: A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.

Detailed description: Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation. Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to chronic arthropathy (joint damage), intracranial hemorrhage, and even death. In patients with moderate/severe to severe hemophilia B, the majority of bleeding episodes occur in the joints and, if not treated, lead to debilitating damage and a decreased quality of life.

Study 101HEMB02 is a long-term follow-up study to evaluate the safety, tolerability, and efficacy of AAVrh10-mediated gene therapy of human FIX in subjects with moderate/severe to severe hemophilia B. The primary objective of the study is to determine the long-term safety and efficacy of DTX101 following a single IV infusion (administered during Study 101HEMB01) in adults with moderate/severe to severe hemophilia B.

This study was previously posted by Dimension Therapeutics, which has been acquired by Ultragenyx.

Eligibility criteria: Inclusion Criteria:

1. Willing and able to provide written informed consent.
2. Completed the Week 52 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.

Exclusion Criteria:

1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety 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

Primary outcomes:
measure: Incidence of adverse events and serious adverse events by dosing group
timeFrame: 208 weeks
measure: Change from baseline in FIX activity level
timeFrame: 208 weeks
Secondary outcomes:
measure: Number of bleeding episodes requiring recombinant FIX infusion
timeFrame: 208 weeks
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Gainesville
country: United States
facility: UF CRC - Clinical Research Center
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan Hospital and Health Systems
state: Michigan
city: Basingstoke
country: United Kingdom
facility: Haemophilia, Haemostasis & Thrombosis Centre
state: Hampshire
city: Manchester
country: United Kingdom
facility: Manchester Haemophilia Comprehensive Care Center
Study officials:
affiliation: Ultragenyx Pharmaceutical Inc
name: Medical Director
role: STUDY_DIRECTOR
References: