Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 5:54 PM
Ignite Modification Date:
2025-12-24 @ 5:54 PM
NCT ID:
NCT02829268
Eligibility Criteria:
Inclusion Criteria:
Patients must meet all of the following criteria to be eligible for enrolment:
1. The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
a. Documented functionally relevant recessive mutations on both alleles of the WFS1 gene or dominant mutation on one allele of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening.
2. The patient is at least 5 years of age (biological age) at the time of written informed consent.
3. The patient, patient's parent(s), or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians' consent and patient's assent, as relevant, must be obtained.
Exclusion Criteria:
Patients who meet any of the following criteria are not eligible for this study:
1. The patient has clinically significant non-Wolfram related CNS involvement which is judged by the investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
2. The patient has a known defect in oxidative phosphorylation (such as a confirmed mitochondrial myopathy)
3. The patient has abnormal liver function (defined as serum transaminases more than twice the upper limit of normal for the reference laboratory)
4. The patient has a significant medical or psychiatric co-morbidity that might affect study data or confound the integrity of study results.
5. The patient has received treatment with any investigational drug within the 30 days prior to study entry.
6. The patient has received blood product transfusions within 90 days prior to screening.
7. The patient is unable to comply with the protocol, (e.g. has a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioural instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
8. The patient has a known history of central apnea and/or ventilation requirements.
9. The patient has a known history of chronic obstructive pulmonary disease, pleural effusion, and/or myocardial disease.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
5 Years
Maximum Age:
60 Years
Study:
NCT02829268
Study Brief:
Protocol Section:
NCT02829268