NCT03999814

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jul 2018

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 30, 2018

Completed
9 months until next milestone

First Submitted

Initial submission to the registry

April 25, 2019

Completed
2 months until next milestone

First Posted

Study publicly available on registry

June 27, 2019

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 27, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 27, 2020

Completed
Last Updated

June 29, 2020

Status Verified

June 1, 2019

Enrollment Period

1.6 years

First QC Date

April 25, 2019

Last Update Submit

June 25, 2020

Conditions

Infantile Neuroaxonal DystrophyINAD

Keywords

Infantile Neuroaxonal DystrophyINADPLA2G6

Outcome Measures

Primary Outcomes (1)

  • To describe the natural history of infantile neuroaxonal dystrophy (INAD).

    Overall analysis

    Birth to time of enrollment.

Secondary Outcomes (2)

  • To look for trends in disease progression of INAD that may be helpful in planning future interventional trials in INAD.

    Birth to time of enrollment.

  • Evaluating and potentially validating a Assessment of Severity by Parent or Caregiver questionnaire.

    At time of enrollment.

Eligibility Criteria

Age18 Months - 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
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.

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Sarah Endemann

Los Altos, California, 94022, United States

Location

MeSH Terms

Conditions

Neuroaxonal Dystrophies

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System Diseases

Study Officials

  • Peter Milner, MD

    Biojiva LLC

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 25, 2019

First Posted

June 27, 2019

Study Start

July 30, 2018

Primary Completion

February 27, 2020

Study Completion

February 27, 2020

Last Updated

June 29, 2020

Record last verified: 2019-06

Data Sharing

IPD Sharing
Will not share

Locations

US(1)