NCT04027816

Brief Summary

This study is a longitudinal and prospective study of the natural history of infantile neuroaxonal dystrophy (INAD).

Trial Health

93
On Track

Trial Health Score

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

Enrollment
43

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jun 2019

Typical duration for all trials

Geographic Reach
5 countries

5 active sites

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

June 17, 2019

Completed
29 days until next milestone

First Submitted

Initial submission to the registry

July 16, 2019

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 22, 2019

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 14, 2022

Completed
14 days until next milestone

Study Completion

Last participant's last visit for all outcomes

February 28, 2022

Completed
Last Updated

April 8, 2022

Status Verified

April 1, 2022

Enrollment Period

2.7 years

First QC Date

July 16, 2019

Last Update Submit

April 7, 2022

Conditions

Neuroaxonal Dystrophy, Infantile

Keywords

PLA2G6-related NBIAINADPLA2G6

Outcome Measures

Primary Outcomes (1)

  • INAD Mortality

    Overall analysis

    1-2 years of follow-up is planned

Secondary Outcomes (4)

  • INAD Morbidity

    1-2 years of follow-up is planned

  • INAD Morbidity

    1-2 years of follow-up is planned

  • INAD Morbidity

    1-2 years of follow-up is planned

  • INAD morbidity

    1-2 years of follow-up is planned

Eligibility Criteria

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

Living males or females with a diagnosis of classic INAD between 18 months to 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 or compound heterozygous PLA2G6 variants
  • Signed informed consent form (ICF) prior to entry into the registry

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Peking University First Hospital

Beijing, China

Location

National Research Centre

Cairo, Egypt

Location

EN1 Neuro Services Pvt. Ltd

Mumbai, India

Location

King Faisal Specialist Hospital and Research Center

Riyadh, Saudi Arabia

Location

National Institute of Neurology of Tunis

Tunis, Tunisia

Location

MeSH Terms

Conditions

Neuroaxonal Dystrophies

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System Diseases

Study Officials

  • Peter Milner, MD

    Biojiva LLC

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 16, 2019

First Posted

July 22, 2019

Study Start

June 17, 2019

Primary Completion

February 14, 2022

Study Completion

February 28, 2022

Last Updated

April 8, 2022

Record last verified: 2022-04

Data Sharing

IPD Sharing
Will not share

Locations

TU(1)EG(1)SA(1)CN(1)IN(1)