Crigler-Najjar SyndromeLUSTRONCT03078881
Clinical Assessment Study in Crigler-Najjar Syndrome
An observational study of Crigler-Najjar Syndrome, run by Audentes Therapeutics at 4 sites in 3 countries.
Trial summary
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Crigler-Najjar Syndrome
- How many people
- 8 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 10 Mar 2017to 23 Jan 2019
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.
Who may be able to join
- Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene
- Subject is aged equal or greater than 1 year of age
- Subject receives daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time)
Who cannot join
- Subject is currently participating in an interventional study or has received gene or cell therapy
- Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the planned participation period of this study
- Subject has significant cholestatic disease, in the opinion of the investigator
- Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening
- Subject has any clinically significant underlying liver disease (other than Crigler-Najjar syndrome), in the opinion of the investigator
- Subject has a history of, or currently has, a clinically important condition other than Crigler-Najjar syndrome, in the opinion of the investigator
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
Clinic for Special Children
Strasburg, Pennsylvania, 17579
Children's Hospital at Montefiore
The Bronx, New York, 10467
About this study
Official title: LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.
- Conditions
- Crigler-Najjar Syndrome
- Medical subject headings
- Crigler-Najjar Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome
Measured: Up to 2 years
Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period
Measured: Up to 2 years
Assess phototherapy usage over the course of the study
Measured: Up to 2 years
Secondary outcomes (1)
Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2017
- Study start10 Mar 2017
- Primary completion23 Jan 2019
- Study completion23 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crigler-Najjar SyndromeStudies of the same condition, open and under way.
- 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
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Audentes Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM, Study director, Audentes Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03078881
- Sponsor's protocol ID
- AT342-01
- Acronym
- LUSTRO
- Status last verified
- Feb 2019
- First submitted
- 8 Mar 2017
- Last updated
- 6 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
- ClinicalTrials.gov ID
- NCT03078881
- Acronym / sponsor ID
- LUSTRO / AT342-01
- Conditions
- Crigler-Najjar Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Audentes TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 10 Mar 2017 (actual)
- Primary completion
- 23 Jan 2019 (actual)
- Study completion
- 23 Jan 2019 (actual)
- First posted
- 14 Mar 2017 (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.
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.
Key inclusion criteria (3 listed)
- Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene
- Subject is aged equal or greater than 1 year of age
- Subject receives daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time)
Key exclusion criteria (6 listed)
- Subject is currently participating in an interventional study or has received gene or cell therapy
- Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the planned participation period of this study
- Subject has significant cholestatic disease, in the opinion of the investigator
- Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening
- Subject has any clinically significant underlying liver disease (other than Crigler-Najjar syndrome), in the opinion of the investigator
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
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome
Time frame: Up to 2 years
Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period
Time frame: Up to 2 years
Assess phototherapy usage over the course of the study
Time frame: Up to 2 years
1 secondary outcome
Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL
Time frame: Up to 2 years
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 Feb 2019.
- Individual participant dataSharing planned: No
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RecruitingPhase 2
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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.
Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM study director
Audentes Therapeutics
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Crigler-Najjar Syndrome, run by Audentes Therapeutics at 4 sites in 3 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 3 inclusion and 6 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 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Israel, 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?
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome (time frame: Up to 2 years). The study lists 3 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 6 Feb 2019.