A Natural History Study of Infantile Neuroaxonal Dystrophy
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|ClinicalTrials.gov Identifier: NCT04027816|
Recruitment Status : Active, not recruiting
First Posted : July 22, 2019
Last Update Posted : February 21, 2021
|Condition or disease|
|Neuroaxonal Dystrophy, Infantile|
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 an observing MD/DO/MBBS from a sponsor site to confirm and clarify information in the medical records.
A baseline evaluation of clinical status will also be performed during this visit to serve as visit one in this longitudinal, prospective natural history study. Subsequent visits will take place every 6 months, for up to 24 months. Key components of this prospective study at each visit include a neurodevelopment exam tailored for INAD, application of the CHOP-INTEND neurodevelopment scale, the Hammersmith infant neurological examination and the modified Ashworth spasticity scale as well as collection of monthly parental severity scoring and monthly home videos of activities of daily living (ADLs).
Data from this study will be pooled and presented in aggregate, without identification of individual subjects.
|Study Type :||Observational|
|Estimated Enrollment :||45 participants|
|Official Title:||Protocol RT001-009: A Natural History Study of Infantile Neuroaxonal Dystrophy|
|Actual Study Start Date :||June 17, 2019|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
- INAD Mortality [ Time Frame: 1-2 years of follow-up is planned ]Overall analysis
- INAD Morbidity [ Time Frame: 1-2 years of follow-up is planned ]Videotaped, structured neurological assessment
- INAD Morbidity [ Time Frame: 1-2 years of follow-up is planned ]CHOP-INTEND Neuro-development Score
- INAD Morbidity [ Time Frame: 1-2 years of follow-up is planned ]Hammersmith infant neurological examination
- INAD morbidity [ Time Frame: 1-2 years of follow-up is planned ]Modified Ashworth spasticity scale
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04027816
|Peking University First Hospital|
|National Research Centre|
|EN1 Neuro Services Pvt. Ltd|
|King Faisal Specialist Hospital and Research Center|
|Riyadh, Saudi Arabia|
|National Institute of Neurology of Tunis|
|Study Director:||Peter Milner, MD||Retrotope, Inc.|