Ornithine Transcarbamylase Deficiency DiseasePhase 1NCT00004498
Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
A Phase 1 study testing Adenoviral Vector-Mediated Gene Transfer in people with Ornithine Transcarbamylase Deficiency Disease, run by University of Pennsylvania.
Trial summary
OBJECTIVES: I. Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency. Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- Ornithine Transcarbamylase Deficiency Disease
- What is being tested?
- Adenoviral Vector-Mediated Gene Transfer
- How many people
- 21 participants
- Where
- No sites listed yet
- Study dates
- Jul 1998to Sep 2000
- Phase
- Phase 1
- 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 to 69 years
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Diagnosis of partial ornithine transcarbamylase deficiency Female heterozygote with abnormal allopurinol challenge or underlying defect in either N15 urea or N15 glutamine OR Male with childhood/adulthood onset OR Family history of 2 affected children
- Stable for at least 1 month prior to study
- Plasma ammonium levels less than 50 micromoles
--Prior/Concurrent Therapy--
- Concurrent alternate pathway therapy to control hyperammonemia allowed
--Patient Characteristics--
- Hepatic: No history of liver disease
- Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No high level of neutralizing antibodies to the adenovirus
About this study
OBJECTIVES:
- Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency.
Read the detailed description
PROTOCOL OUTLINE: This is a dose escalation study. Patients undergo a femoral arterial placement of a hepatic intraarterial catheter. Patients then receive adenoviral vector mediated gene transfer intravascularly over 30 minutes.
Cohorts of 3 patients each receive escalating doses of adenoviral vector until the maximum tolerated dose is determined.
Patients are followed at 3, 5, 7, 8, 15, and 29 days, at 2 months, and then every 3 months thereafter.
Completion date provided represents the completion date of the grant per OOPD records
- Being tested
- Adenoviral Vector-Mediated Gene Transfer
- Keywords
- genetic diseases and dysmorphic syndromes, inborn errors of metabolism, ornithine transcarbamylase deficiency, rare disease, urea cycle disorder
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease, Genetic Diseases, Inborn, Metabolism, Inborn Errors, Rare Diseases, Urea Cycle Disorders, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adenoviral Vector-Mediated Gene TransferGenetic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startJul 1998
- Study completionSep 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ornithine Transcarbamylase Deficiency DiseaseStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Enrolment
- 21 participants
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Investigators
- Steven E. Raper, Study chair, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004498
- Sponsor's protocol ID
- 199/14290
- Other IDs
- UPSM-FDR001529
- Status last verified
- Feb 2000
- First submitted
- 18 Oct 1999
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
- ClinicalTrials.gov ID
- NCT00004498
- Acronym / sponsor ID
- 199/14290
- Keywords
- genetic diseases and dysmorphic syndromes, inborn errors of metabolism, ornithine transcarbamylase deficiency, rare disease, urea cycle disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 21 participants
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Study start
- Jul 1998
- Study completion
- Sep 2000
- First posted
- 19 Oct 1999 (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.
OBJECTIVES:
- Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency.
Detailed description
PROTOCOL OUTLINE: This is a dose escalation study. Patients undergo a femoral arterial placement of a hepatic intraarterial catheter. Patients then receive adenoviral vector mediated gene transfer intravascularly over 30 minutes.
Cohorts of 3 patients each receive escalating doses of adenoviral vector until the maximum tolerated dose is determined.
Patients are followed at 3, 5, 7, 8, 15, and 29 days, at 2 months, and then every 3 months thereafter.
Completion date provided represents the completion date of the grant per OOPD records
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adenoviral Vector-Mediated Gene Transfer | Genetic | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
Related trials for Ornithine Transcarbamylase Deficiency Disease
RecruitingObservational study
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
RecruitingObservational study
Abbott Vascular Medical Device Registry
Acute Myocardial Infarction, Restenoses, Coronary
RecruitingPhase 1/2
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Active, not recruitingPhase 3
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Active, not recruitingPhase 3
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
OTC Deficiency
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.
Steven E. Raper study chair
University of Pennsylvania
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Adenoviral Vector-Mediated Gene Transfer in people with Ornithine Transcarbamylase Deficiency Disease, run by University of Pennsylvania.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. 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.
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 25 Mar 2015.