NCT02971969

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2017

Longer than P75 for all trials

Geographic Reach
2 countries

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 21, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 23, 2016

Completed
1 month until next milestone

Study Start

First participant enrolled

January 1, 2017

Completed
4.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 6, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 6, 2021

Completed
Last Updated

January 6, 2022

Status Verified

January 1, 2022

Enrollment Period

4.8 years

First QC Date

November 21, 2016

Last Update Submit

January 4, 2022

Conditions

Hemophilia B

Keywords

gene therapyHemophilia B

Outcome Measures

Primary Outcomes (2)

  • Incidence of adverse events and serious adverse events by dosing group

    208 weeks

  • Change from baseline in FIX activity level

    208 weeks

Secondary Outcomes (1)

  • Number of bleeding episodes requiring recombinant FIX infusion

    208 weeks

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01 clinical study

You may qualify if:

  • Willing and able to provide written informed consent.
  • Completed the Week 52 visit in Study 101HEMB01.
  • Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable.
  • Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

UF CRC - Clinical Research Center

Gainesville, Florida, 32610, United States

Location

University of Michigan Hospital and Health Systems

Ann Arbor, Michigan, 48109-5872, United States

Location

Haemophilia, Haemostasis & Thrombosis Centre

Basingstoke, Hampshire, RG24 9NA, United Kingdom

Location

Manchester Haemophilia Comprehensive Care Center

Manchester, M13 9WL, United Kingdom

Location

Related Publications (1)

  • Pipe S, Poma A, Rajasekhar A, Everington T, Sankoh S, Allen J, Cataldo J, Crombez E. Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies. Blood Adv. 2025 Jun 24;9(12):2980-2987. doi: 10.1182/bloodadvances.2024015184.

    PMID: 40197980DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Blood

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • Medical Director

    Ultragenyx Pharmaceutical Inc

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 21, 2016

First Posted

November 23, 2016

Study Start

January 1, 2017

Primary Completion

November 6, 2021

Study Completion

November 6, 2021

Last Updated

January 6, 2022

Record last verified: 2022-01

Locations

GB(2)US(3)