Status:
COMPLETED
A Natural History Study of Infantile Neuroaxonal Dystrophy
Lead Sponsor:
Biojiva LLC
Conditions:
Neuroaxonal Dystrophy, Infantile
Eligibility:
All Genders
18-10 years
Brief Summary
This study is a longitudinal and prospective study of the natural history of infantile neuroaxonal dystrophy (INAD).
Detailed Description
After obtaining informed consent, the study participants' relevant medical records will be collected and reviewed for this study. Next, a clinic visit will be scheduled with the patient's family and a...
Eligibility Criteria
Inclusion
- Male or female 18 months to 10 years of age
- Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years)
- Homozygous or compound heterozygous PLA2G6 variants
- Signed informed consent form (ICF) prior to entry into the registry
Exclusion
- Diagnosis of atypical NAD (ANAD)
- Additional underlying diagnosis with features that overlap with INAD
- Unwilling or unable to allow medical record review
- Unwilling or unable to participate in serial assessments every 6 months (including deceased patients)
Key Trial Info
Start Date :
June 17 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 28 2022
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT04027816
Start Date
June 17 2019
End Date
February 28 2022
Last Update
April 8 2022
Active Locations (5)
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1
Peking University First Hospital
Beijing, China
2
National Research Centre
Cairo, Egypt
3
EN1 Neuro Services Pvt. Ltd
Mumbai, India
4
King Faisal Specialist Hospital and Research Center
Riyadh, Saudi Arabia