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A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients (FRAMES)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03917225
Recruitment Status : Completed
First Posted : April 17, 2019
Last Update Posted : September 25, 2020
Sponsor:
Information provided by (Responsible Party):
Minoryx Therapeutics, S.L.

Tracking Information
First Submitted Date  ICMJE April 10, 2019
First Posted Date  ICMJE April 17, 2019
Last Update Posted Date September 25, 2020
Actual Study Start Date  ICMJE March 26, 2019
Actual Primary Completion Date August 13, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
Change from baseline in spinal cord area cervical segment C2-C3 [mm2] [ Time Frame: 48 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 15, 2019)
To evaluate the efect of treatment with MIN-102 on the progression of Friedreich's Ataxia (FRDA) by MRI measurements [ Time Frame: 48 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
Official Title  ICMJE A Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophisyiological, and Clinical Markers in Patients With Friedreich's Ataxia
Brief Summary Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Friedreich Ataxia
Intervention  ICMJE
  • Drug: MIN-102
    Once-daily dosing with a volume specified by the pharmacokineteic specialistto achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.
  • Drug: Placebo
    Once-daily dosing with a volume specified by the pharmacokinetic specialist. Oral Suspension.
Study Arms  ICMJE
  • Active Comparator: Active
    Intervention: Drug: MIN-102
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 15, 2019)
36
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 14, 2020
Actual Primary Completion Date August 13, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male and female subjects aged ≥12 and ≤60 years, inclusive, with a genetically confirmed diagnose of Friedreich's Ataxia.
  • Be able to walk > 10 meters with support (two special sticks, stroller or accompanying person).
  • Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) of < 25.

Exclusion Criteria:

  • Age of onset of disease ≥25 years.
  • Higher degree of cardiomyopathy assessed by echocardigram.
  • Diabetes
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 60 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   France,   Germany,   Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03917225
Other Study ID Numbers  ICMJE MT-2-03
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Minoryx Therapeutics, S.L.
Study Sponsor  ICMJE Minoryx Therapeutics, S.L.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Minoryx Therapeutics, S.L.
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP