Crigler Najjar SyndromeNCT02302690
Immunity Against AAV in Crigler Najjar Patient
An observational study of Crigler Najjar Syndrome, run by Genethon at 1 site in France.
Trial summary
The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status. A first objective is to assess the presence of neutralizing AAV antibodies in the serum of CN patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Crigler Najjar Syndrome
- What is being tested?
- Blood sample for immunology
- How many people
- 49 participants
- Where
- 1 siteFrance
- Study dates
- Nov 2014to Jun 2017
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Crigler Najjar syndrome
Who may be able to join
- The patient has a Crigler-Najjar syndrome
- The molecular diagnosis has been confirmed by UGT1A1 gene DNA sequencing
Who cannot join
- The patient is liver transplanted
- The patient is suffering from severe associated pathologies precluding further enrolment in a future interventional clinical study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of the Prevalence of Pre-existing Immunity Against the Adeno-associated Virus (AAV) in Patients With Crigler-Najjar Syndrome
The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status. A first objective is to assess the presence of neutralizing AAV antibodies in the serum of CN patients.
- Conditions
- Crigler Najjar Syndrome
- Being tested
- Blood sample for immunology
- Medical subject headings
- Crigler-Najjar Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood sample for immunologyBiological
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the presence of neutralizing AAV antibodies in the serum of CN patients
Measured: 1 day visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2014 (estimated)
- Study startNov 2014
- Primary completionJun 2017
- Study completionJun 2017
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
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genethon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02302690
- Sponsor's protocol ID
- CRIG-COL-01
- Status last verified
- Dec 2017
- First submitted
- 25 Nov 2014
- Last updated
- 13 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of the Prevalence of Pre-existing Immunity Against the Adeno-associated Virus (AAV) in Patients With Crigler-Najjar Syndrome
- ClinicalTrials.gov ID
- NCT02302690
- Acronym / sponsor ID
- CRIG-COL-01
- Conditions
- Crigler Najjar Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- GenethonUniversity, hospital or other
- Study start
- Nov 2014
- Primary completion
- Jun 2017 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 27 Nov 2014 (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.
The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status. A first objective is to assess the presence of neutralizing AAV antibodies in the serum of CN patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Crigler Najjar syndrome
Key inclusion criteria (2 listed)
- The patient has a Crigler-Najjar syndrome
- The molecular diagnosis has been confirmed by UGT1A1 gene DNA sequencing
Key exclusion criteria (2 listed)
- The patient is liver transplanted
- The patient is suffering from severe associated pathologies precluding further enrolment in a future interventional clinical study
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 |
|---|---|---|
| Blood sample for immunology | Biological | – |
Primary outcome
To assess the presence of neutralizing AAV antibodies in the serum of CN patients
Time frame: 1 day visit
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 13 Dec 2017.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: 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 Genethon at 1 site in France.
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 2 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 13 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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?
To assess the presence of neutralizing AAV antibodies in the serum of CN patients (time frame: 1 day visit). 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 13 Dec 2017.