Byler DiseaseNCT01784718
Buphenyl Therapy for Byler's Disease
A study testing Buphenyl in people with Byler Disease, run by University of Pittsburgh.
Trial summary
This is a single patient compassionate use protocol to determine whether Buphenyl (4-phenylbutyrate) will improve the poor bile flow associated with Byler Disease. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Byler Disease
- What is being tested?
- Buphenyl
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 1 year and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Byler Disease
- Cholestasis unresponsive to pharmacologic therapy
Who cannot join
- Allergy/Hypersensitivity to Buphenyl
About this study
Official title: Compassionate Use of Buphenyl® in the Treatment of Byler's Disease
This is a single patient compassionate use protocol to determine whether Buphenyl (4-phenylbutyrate) will improve the poor bile flow associated with Byler Disease.
- Conditions
- Byler Disease
- Being tested
- Buphenyl
- Medical subject headings
- Cholestasis, progressive familial intrahepatic 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BuphenylDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Byler DiseaseStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01784718
- Sponsor's protocol ID
- PRO12070385
- Status last verified
- Feb 2013
- First submitted
- 4 Feb 2013
- Last updated
- 6 Feb 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of Buphenyl® in the Treatment of Byler's Disease
- ClinicalTrials.gov ID
- NCT01784718
- Acronym / sponsor ID
- PRO12070385
- Conditions
- Byler Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- First posted
- 6 Feb 2013 (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.
This is a single patient compassionate use protocol to determine whether Buphenyl (4-phenylbutyrate) will improve the poor bile flow associated with Byler Disease.
Study design
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Byler Disease
- Cholestasis unresponsive to pharmacologic therapy
Key exclusion criteria (1 listed)
- Allergy/Hypersensitivity to Buphenyl
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 |
|---|---|---|
| Buphenyl | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2013.
Related trials for Byler Disease
Active, not recruitingObservational study
Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
PFIC - Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea
Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
Pediatric Evaluation and Registry for Liver Cholestasis in Canada
PFIC - Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome (ALGS)
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Buphenyl in people with Byler Disease, run by University of Pittsburgh.
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 1 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: status not confirmed, as of the sponsor's last update on 6 Feb 2013. 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.
Are results available for this study?
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 6 Feb 2013.