A Study to Assess Efficacy and Safety of RT001 in Subject... | NCT03570931

Studies
A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

NCT03570931

Sponsor

Biojiva LLC

Status

Active, not recruiting

Last Update Posted

Oct 14, 2021Actual

Enrollment

19Actual

Phase

Phase 2Phase 3

Conditions

Infantile Neuroaxonal Dystrophy

Interventions

RT001

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

Oct 2021

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 5, 2018Actual

Primary Completion Date

Aug 9, 2020Actual

Completion Date

Jun 30, 2022Estimated

First Submitted Date

Jun 11, 2018

First Submission Date that Met QC Criteria

Jun 25, 2018

First Posted Date

Jun 27, 2018Actual

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

Oct 6, 2021

Last Update Posted Date

Oct 14, 2021Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

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

The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).

Detailed Description

This is a single arm open-label study with a structured observation of INAD patients treated with RT001. Enrolled subjects will undergo observation and testing to determine the effect of RT001 treatment. Fifteen to twenty eligible subjects will be treated with RT001 for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

19Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
RT001	Experimental	RT001, oral, 3.84 g/day	Drug: RT001

Interventions

Name	Type	Description	Arm Group Labels	Other Names
RT001	Drug	RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.	RT001

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Modified Ashworth Spasticity Scale	Change from baseline in the Modified Ashworth spasticity scale.	12 months

Secondary Outcomes

Measure	Description	Time Frame
INAD Progression Composite	Change from baseline in an INAD composite score to assess the overall treatment effect on the most progressive aspects of the disease	12 months
Progression Free Survival Time

Other Outcomes

Measure	Description	Time Frame
Modified Infantile Neuroaxonal Dystrophy Rating Scale (mINAD-RS24)	Change in score from baseline derived from a structured pediatric neurological development exam tailored for INAD, involving elements of activities of daily living and vital functions.	12 months
Modified Parental Rating Scale (mPRS22)

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)
Must have impairment in at least 2 of the assessed categories at baseline
Signed informed consent form (ICF) prior to entry into the study
Able to provide the necessary blood samples

Exclusion Criteria:

Received treatment with other experimental therapies within the last 30 days prior to the first dose
Requiring mechanical ventilation, other than positive air pressure support primarily for mitigation of sleep apnea.
Have a life expectancy of less than one year
Diagnosis of atypical NAD (ANAD)
Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled

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

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Peter Milner, MD	Chief Medical Officer	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of California San Francisco, Benioff Children's Hospital		San Francisco	California	94158	United States

IPD Sharing Statement Module

Plan to Share IPD

Undecided

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Release Date	Unrelease Date	Unrelease Date Unknown	Reset Date	MCP Release Number
Apr 7, 2022			May 4, 2022	7
Jun 27, 2025

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