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

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ClinicalTrials.gov Identifier: NCT03570931
Recruitment Status : Active, not recruiting
First Posted : June 27, 2018
Last Update Posted : October 14, 2021
Sponsor:
Information provided by (Responsible Party):
Retrotope, Inc.

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

Condition or disease Intervention/treatment Phase
Infantile Neuroaxonal Dystrophy Drug: RT001 Phase 2 Phase 3

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Single arm open-label study
Masking: None (Open Label)
Masking Description: Open Label
Primary Purpose: Treatment
Official Title: A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
Actual Study Start Date : November 5, 2018
Actual Primary Completion Date : August 9, 2020
Estimated Study Completion Date : June 30, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: RT001
RT001, oral, 3.84 g/day
 Drug: RT001
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.
Other Name: Deuterated linoleic acid


Outcome Measures
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Primary Outcome Measures :
  1. Modified Ashworth Spasticity Scale [ Time Frame: 12 months ]
    Change from baseline in the Modified Ashworth spasticity scale.


Secondary Outcome Measures :
  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 Outcome Measures:
  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.


Eligibility Criteria
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Ages Eligible for Study:   18 Months to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female 18 months to 10 years of age
  2. Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years)
  3. Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
  4. Must have impairment in at least 2 of the assessed categories at baseline
  5. Signed informed consent form (ICF) prior to entry into the study
  6. Able to provide the necessary blood samples

Exclusion Criteria:

  1. Received treatment with other experimental therapies within the last 30 days prior to the first dose
  2. Requiring mechanical ventilation, other than positive air pressure support primarily for mitigation of sleep apnea.
  3. Have a life expectancy of less than one year
  4. Diagnosis of atypical NAD (ANAD)
  5. 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03570931


Locations
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United States, California
University of California San Francisco, Benioff Children's Hospital
San Francisco, California, United States, 94158
United States, New Jersey
Jacobs Levy Genomics and Research Program
Morristown, New Jersey, United States, 07960
Sponsors and Collaborators
Retrotope, Inc.
Investigators
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Study Director: Peter Milner, MD Chief Medical Officer
More Information
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Responsible Party: Retrotope, Inc.
ClinicalTrials.gov Identifier: NCT03570931    
Other Study ID Numbers: RT001-008
First Posted: June 27, 2018    Key Record Dates
Last Update Posted: October 14, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Retrotope, Inc.:
INAD
Infantile Neuroaxonal Dystrophy
Additional relevant MeSH terms:
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Neuroaxonal Dystrophies
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases