Ornithine Transcarbamylase DeficiencyNCT04909346
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
An observational study of Ornithine Transcarbamylase Deficiency and Wilson Disease, run by Ultragenyx Pharmaceutical Inc at 5 sites in 5 countries.
Trial summary
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
- How many people
- 51 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 23 Jun 2021to 17 Nov 2022
- 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
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Study population will include approximately equal proportions of subjects with OTC deficiency, GSDIa, and Wilson Disease
Who may be able to join
- Diagnosis of OTC deficiency, GSDIa, or Wilson Disease.
- Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
Who cannot join
- Prior exposure to an AAV-based gene therapy.
- Concurrent or previous participation in another Ultragenyx clinical study.
- Recipient of a liver transplant.
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
United States1 site
Synexus Clinical Research US (Virtual Trial)
Akron, Ohio, 44311
Brazil1 site
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90560-030
Spain1 site
Hospital Clínico Universitario de Santiago de Compostela
Santiago de Compostela, 15706
United Kingdom1 site
Synexus North East Clinical Research Centre
Hexham, NE46 1QJ
About this study
Official title: Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease
Read the detailed description
The study is primarily designed to follow a virtual model, in which Screening will take place over a telephone/video call. The study will comprise a single home health visit, either on the same day as Screening or within 30 days of enrollment. A safety follow-up may occur up to 5 days after Day 1 assessments are completed.
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease, Hepatolenticular Degeneration, Hepatorenal form of glycogen storage disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa
Measured: Up to 35 days
Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease
Measured: Up to 35 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2021
- Study start23 Jun 2021
- Primary completion17 Nov 2022
- Study completion17 Nov 2022
Why it stopped: Sponsor decision not related to safety concerns
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ornithine Transcarbamylase DeficiencyStudies 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
- 51 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
- NCT04909346
- Sponsor's protocol ID
- UX431-CL001
- Status last verified
- Jun 2023
- First submitted
- 26 May 2021
- Last updated
- 12 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease
- ClinicalTrials.gov ID
- NCT04909346
- Acronym / sponsor ID
- UX431-CL001
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor decision not related to safety concerns
- Enrolment
- 51 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 23 Jun 2021 (actual)
- Primary completion
- 17 Nov 2022 (actual)
- Study completion
- 17 Nov 2022 (actual)
- First posted
- 1 Jun 2021 (actual)
- 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.
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease
Detailed description
The study is primarily designed to follow a virtual model, in which Screening will take place over a telephone/video call. The study will comprise a single home health visit, either on the same day as Screening or within 30 days of enrollment. A safety follow-up may occur up to 5 days after Day 1 assessments are completed.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Study population will include approximately equal proportions of subjects with OTC deficiency, GSDIa, and Wilson Disease
Key inclusion criteria (2 listed)
- Diagnosis of OTC deficiency, GSDIa, or Wilson Disease.
- Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
Key exclusion criteria (4 listed)
- Prior exposure to an AAV-based gene therapy.
- Concurrent or previous participation in another Ultragenyx clinical study.
- Recipient of a liver transplant.
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
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
Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa
Time frame: Up to 35 days
Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease
Time frame: Up to 35 days
Results and safety
The study was stopped early (reason given: Sponsor decision not related to safety concerns). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2023.
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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 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Ornithine Transcarbamylase Deficiency and Wilson Disease, run by Ultragenyx Pharmaceutical Inc at 5 sites in 5 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 2 inclusion and 4 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: stopped early, as of the sponsor's last update on 12 Jun 2023. 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, Brazil, Canada, Spain, 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 was stopped early (reason given: Sponsor decision not related to safety concerns). Results are not available in the source record.
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?
Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa (time frame: Up to 35 days). 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?
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 12 Jun 2023.