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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 : Active, not recruiting
First Posted : April 17, 2019
Last Update Posted : November 22, 2019
Sponsor:
Information provided by (Responsible Party):
Minoryx Therapeutics, S.L.

Study Description
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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

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: MIN-102 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : March 26, 2019
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : September 2020

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Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Active 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.
Placebo Comparator: Placebo Drug: Placebo
Once-daily dosing with a volume specified by the pharmacokinetic specialist. Oral Suspension.


Outcome Measures
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Primary Outcome Measures :
  1. Change from baseline in spinal cord area cervical segment C2-C3 [mm2] [ Time Frame: 48 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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): NCT03917225


Locations
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Belgium
Hôpital Erasme-ULB
Brussels, Belgium, B-1070
France
ICM, Groupe Hospitalier Pitié Salpêtrière
Paris, France, 75646
Germany
Universitätsklinikum RWTH
Aachen, Germany, 52074
Spain
Hospital Sant Joan de Déu
Barcelona, Spain
Hospital Universitario La Paz
Madrid, Spain, 28046
Sponsors and Collaborators
Minoryx Therapeutics, S.L.
More Information
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Responsible Party: Minoryx Therapeutics, S.L.
ClinicalTrials.gov Identifier: NCT03917225    
Other Study ID Numbers: MT-2-03
First Posted: April 17, 2019    Key Record Dates
Last Update Posted: November 22, 2019
Last Verified: November 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
 Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases