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

Infantile Neuroaxonal Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03570931
• Other study ID #: RT001-008
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: November 5, 2018
• Est. completion date: June 30, 2022

Study information

• Verified date: October 2021
• Source: Retrotope, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 19
• Est. completion date: June 30, 2022
• Est. primary completion date: August 9, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Months to 10 Years

Eligibility

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

Related Conditions & MeSH terms

• Infantile Neuroaxonal Dystrophy

Intervention

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

Locations

Country	Name	City	State
United States	Jacobs Levy Genomics and Research Program	Morristown	New Jersey
United States	University of California San Francisco, Benioff Children's Hospital	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Retrotope, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	Modified Parental Rating Scale (mPRS22)	Change in score from baseline from a parental rating scale tailored for INAD, involving elements of activities of daily living and vital functions	12 months
Other	Original Infantile Neuroaxonal Dystrophy Rating Scale (INAD-RS40)	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
Other	Original Parental Rating Scale (mPRS33)	Change in score from baseline from a parental rating scale tailored for INAD, involving elements of activities of daily living and vital functions	12 months
Other	Incidence of Treatment-Emergent Adverse Events	The incidence of treatment-emergent adverse events will be presented by severity and relationship to study drug.	12 months
Primary	Modified Ashworth Spasticity Scale	Change from baseline in the Modified Ashworth spasticity scale.	12 months
Secondary	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
Secondary	Progression Free Survival Time	Progression free survival time (mortality or pneumonia)	All available data