Tyrosinemia INCT00004443
Study of NTBC for Tyrosinemia I
A study testing NTBC in people with Tyrosinemia I, run by University of Washington at 3 sites in 2 countries.
Trial summary
OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I. II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in renal tubular damage, reduction in the need for liver transplantation, and reduction in the development of hepatocarcinoma in these patients. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Tyrosinemia I
- What is being tested?
- NTBC
- How many people
- 20 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Oct 1998to Sep 2006
- Phase
- Interventional
- 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 Up to 17 years
- All sexes
- No healthy volunteers
- Child
- Confirmed diagnosis of tyrosinemia Detected through newborn screening before the onset of symptoms OR Diagnosed on the basis of symptoms (liver disease, neurological crises, growth failure) and succinylacetone in urine or blood and/or fumarylacetoacetate dehydratase deficiency in cultured fibroblasts or liver biopsy
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Oregon Health Sciences University
Portland, Oregon, 97201-3098
University of Washington School of Medicine
Seattle, Washington, 98195
About this study
OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.
II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in renal tubular damage, reduction in the need for liver transplantation, and reduction in the development of hepatocarcinoma in these patients.
Read the detailed description
PROTOCOL OUTLINE: Patients are stratified according to age at onset of symptoms (0-2 months vs 2-6 months vs greater than 6 months).
Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable toxicity.
Patients are followed once a month for 6 months, then every 3 months thereafter.
Completion date provided represents the completion date of the grant per OOPD records
- Conditions
- Tyrosinemia I
- Being tested
- NTBC
- Keywords
- inborn errors of metabolism, rare disease, tyrosinemia I
- Medical subject headings
- Metabolism, Inborn Errors, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NTBCDrug
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 startOct 1998
- Study completionSep 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tyrosinemia IStudies of the same condition, open and under way.
- More trials run by University of WashingtonThe 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 N/A
- Primary purpose
- Treatment
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Washington
- Sponsor type
- University, hospital or other
- Investigators
- C. Ronald Scott, Study chair, University of Washington
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004443
- Sponsor's protocol ID
- 199/13443
- Other IDs
- UWASH-FDR001445
- Status last verified
- Apr 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
- Study of NTBC for Tyrosinemia I
- ClinicalTrials.gov ID
- NCT00004443
- Acronym / sponsor ID
- 199/13443
- Conditions
- Tyrosinemia I
- Keywords
- inborn errors of metabolism, rare disease, tyrosinemia I
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of WashingtonUniversity, hospital or other
- Study start
- Oct 1998
- Study completion
- Sep 2006
- 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: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.
II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in renal tubular damage, reduction in the need for liver transplantation, and reduction in the development of hepatocarcinoma in these patients.
Detailed description
PROTOCOL OUTLINE: Patients are stratified according to age at onset of symptoms (0-2 months vs 2-6 months vs greater than 6 months).
Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable toxicity.
Patients are followed once a month for 6 months, 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
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| NTBC | Drug | – |
Primary outcome
Not reported in the source record.
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 25 Mar 2015.
Related trials for Tyrosinemia I
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
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.
C. Ronald Scott study chair
University of Washington
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing NTBC in people with Tyrosinemia I, run by University of Washington at 3 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?
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 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Canada. 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.
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.