Natural History of Infantile Neuroaxonal Dystrophy | NCT03999814

Studies
Natural History of Infantile Neuroaxonal Dystrophy

NCT03999814

Sponsor

Biojiva LLC

Status

Completed

Last Update Posted

Jun 29, 2020Actual

Enrollment

13Actual

Phase

Not provided

Conditions

Infantile Neuroaxonal Dystrophy
INAD

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jun 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 30, 2018Actual

Primary Completion Date

Feb 27, 2020Actual

Completion Date

Feb 27, 2020Actual

First Submitted Date

Apr 25, 2019

First Submission Date that Met QC Criteria

Jun 24, 2019

First Posted Date

Jun 27, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 25, 2020

Last Update Posted Date

Jun 29, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This is a retrospective and cross-sectional review of the natural history of INAD.

Detailed Description

After obtaining informed consent, the study participants' relevant medical records will be collected and reviewed. If needed, a telephone or video conference will be scheduled with the patient's family to confirm and clarify information in the medical record. Deceased patients may be eligible for inclusion the retrospective registry if medical records are sufficient and indicate eligibility. Data will be pooled and presented in aggregate, without identification of individual subjects.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

13Actual

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To describe the natural history of infantile neuroaxonal dystrophy (INAD).	Overall analysis	Birth to time of enrollment.

Secondary Outcomes

Measure	Description	Time Frame
To look for trends in disease progression of INAD that may be helpful in planning future interventional trials in INAD.	Overall analysis	Birth to time of enrollment.
Evaluating and potentially validating a Assessment of Severity by Parent or Caregiver questionnaire.

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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 for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
Signed informed consent form (ICF) prior to entry into the registry

Exclusion Criteria:

Diagnosis of atypical NAD (ANAD)
Unwilling or unable to allow medical record review

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Months

Maximum Age

10 Years

Standard Ages

Child

Study Population

Living or deceased males or females with a diagnosis of classic INAD between the ages of 18 months and 10 years of age.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Peter Milner, MD	Biojiva LLC	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Sarah Endemann		Los Altos	California	94022	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D019150	Neuroaxonal Dystrophies

Ancestor Terms

ID	Term
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided