Hemophilia BNCT02971969
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
An observational study of Hemophilia B, run by Ultragenyx Pharmaceutical Inc at 5 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B
- How many people
- 6 participants
- Where
- 5 sitesin 2 countries
- Study dates
- Jan 2017to 6 Nov 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01 clinical study
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
United States3 sites
University of Michigan Hospital and Health Systems
Ann Arbor, Michigan, 48109-5872
UF CRC - Clinical Research Center
Gainesville, Florida, 32610
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
United Kingdom2 sites
Haemophilia, Haemostasis & Thrombosis Centre
Basingstoke, Hampshire, RG24 9NA
Manchester Haemophilia Comprehensive Care Center
Manchester, M13 9WL
About this study
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
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.
Read the 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.
- Conditions
- Hemophilia B
- Keywords
- gene therapy, Hemophilia B
- Medical subject headings
- Hemophilia B
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events and serious adverse events by dosing group
Measured: 208 weeks
Change from baseline in FIX activity level
Measured: 208 weeks
Secondary outcomes (1)
Number of bleeding episodes requiring recombinant FIX infusion
Measured: 208 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2016 (estimated)
- Study startJan 2017
- Primary completion6 Nov 2021
- Study completion6 Nov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02971969
- Sponsor's protocol ID
- 101HEMB02
- Other IDs
- 2016-003430-25, 2016
- Status last verified
- Jan 2022
- First submitted
- 21 Nov 2016
- Last updated
- 6 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 40197980
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02971969
- Acronym / sponsor ID
- 101HEMB02
- Conditions
- Hemophilia B
- Keywords
- gene therapy, Hemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- Jan 2017
- Primary completion
- 6 Nov 2021 (actual)
- Study completion
- 6 Nov 2021 (actual)
- First posted
- 23 Nov 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01 clinical study
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of adverse events and serious adverse events by dosing group
Time frame: 208 weeks
Change from baseline in FIX activity level
Time frame: 208 weeks
1 secondary outcome
Number of bleeding episodes requiring recombinant FIX infusion
Time frame: 208 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2022.
- Derived from this study: PubMed 40197980Pipe 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Ultragenyx Pharmaceutical Inc
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B, run by Ultragenyx Pharmaceutical Inc at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events and serious adverse events by dosing group (time frame: 208 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02971969, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2022.