Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT03570931

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Infantile Neuroaxonal Dystrophy Brain Diseases Central Nervous System Diseases Nervous System Diseases Neuroaxonal Dystrophies

Age range

18 month–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

University of California San Francisco, Benioff Children's Hospital, San Francisco, California, United States

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

RT001

Drug

RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.

Other names: Deuterated linoleic acid

Primary outcomes

  1. Modified Ashworth Spasticity Scale

    Time frame: 12 months

    Change from baseline in the Modified Ashworth spasticity scale.

Secondary outcomes

  1. INAD Progression Composite

    Time frame: 12 months

    Change from baseline in an INAD composite score to assess the overall treatment effect on the most progressive aspects of the disease

  2. Progression Free Survival Time

    Time frame: All available data

    Progression free survival time (mortality or pneumonia)

Other outcomes

  1. Modified Infantile Neuroaxonal Dystrophy Rating Scale (mINAD-RS24)

    Time frame: 12 months

    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.

  2. Modified Parental Rating Scale (mPRS22)

    Time frame: 12 months

    Change in score from baseline from a parental rating scale tailored for INAD, involving elements of activities of daily living and vital functions

  3. Original Infantile Neuroaxonal Dystrophy Rating Scale (INAD-RS40)

    Time frame: 12 months

    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.

  4. Original Parental Rating Scale (mPRS33)

    Time frame: 12 months

    Change in score from baseline from a parental rating scale tailored for INAD, involving elements of activities of daily living and vital functions

  5. Incidence of Treatment-Emergent Adverse Events

    Time frame: 12 months

    The incidence of treatment-emergent adverse events will be presented by severity and relationship to study drug.

Sponsors and collaborators

Lead sponsor

Biojiva LLC

Industry

Registry information

Official study title

A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

Important dates

Study start
2018
Primary completion
2020
Study completion
2022
First posted
Jun 27, 2018
Registry last updated
Oct 14, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.