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Clinical Trials/NCT03570931
NCT03570931Active, not recruitingPhase 2

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

Retrotope, Inc.2 sites in 1 country19 target enrollmentStarted: November 5, 2018Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Active, not recruiting
Enrollment
19
Locations
2
Primary Endpoint
Modified Ashworth Spasticity Scale
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Masking Description

Open Label

Eligibility Criteria

Ages
18 Months to 10 Years (Child)
Sex
All
Accepts Healthy Volunteers
No

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

Arms & Interventions

RT001

Experimental

RT001, oral, 3.84 g/day

Intervention: RT001 (Drug)

Outcomes

Primary Outcomes

Modified Ashworth Spasticity Scale

Time Frame: 12 months

Change from baseline in the Modified Ashworth spasticity scale.

Secondary Outcomes

  • Progression Free Survival Time(All available data)
  • INAD Progression Composite(12 months)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (2)

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