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

NCT03917225 | Completed Phase 2

• 1 other identifier: MT-2-03
• Study Type: interventional
• Target: 39
• Locations: 4 countries
• Sites: 5

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

Conditions

Friedreich Ataxia

Keywords

Leriglitazone; Friedreich's ataxia

Outcome Measures

Primary Outcomes (1)

• Change from Baseline in spinal cord area cervical segment C2-C3 [mm²]

  Spinal cord area cervical segment C2-C3 area was assessed at Baseline, Week 24, and Week 48 by morphometric magnetic resonance imaging (MRI) measurements. Morphometric changes in MRI parameters are associated with measures of clinical decline. In normal disease etiology, the spinal cord exhibits an initial decrease in area, and then reaches a plateau after 7-8 years from disease onset.

  Baseline to 48 weeks

Secondary Outcomes (24)

• Change from Baseline in SARA total score at Week 48

  Baseline to 48 weeks

• Change from Baseline in cervical spinal cord (C2-C7) in fractional anisotropy at Week 48

  Baseline to 48 weeks

• Change from Baseline in cervical spinal cord (C2-C7) in mean, axial, and radial diffusivity (10-³ mm²/s) at Week 48

  Baseline to 48 weeks

• Change from Baseline in spinal cord total N-acetylaspartate concentration/myo-inositol (tNAA/mIns) ratio as assessed by magnetic resonance spectroscopy (MRS) at Week 48

  Baseline to 48 weeks

• Change from Baseline in quantitative susceptibility mapping (QSM) for iron concentration (ppb) at Week 48

  Baseline to 48 weeks

Study Arms (2)

MIN-102

EXPERIMENTAL

MIN-102 (5-\[\[4-\[2-\[5-(1-Hydroxyethyl)-2-pyridinyl\]ethoxy\]phenyl\]methyl\]-2,4-thiazolidinedione hydrochloride (1:1)). Dosing is once daily at approximately the same time each morning throughout the entire treatment phase (48 weeks).

Drug: MIN-102

Placebo

PLACEBO COMPARATOR

Matches MIN-102 visually and by taste. Dosing is once daily at approximately the same time each morning throughout the entire treatment phase (48 weeks).

Drug: Placebo

Interventions

MIN-102 DRUG

Once-daily dosing with a volume specified by the pharmacokinetic specialist to achieve the desired plasma exposure. MIN-102 oral suspension, strength 15 mg/ml.

MIN-102

Placebo DRUG

Once-daily dosing with a volume specified by the pharmacokinetic specialist. Oral suspension.

Placebo

Eligibility Criteria

• Age: 12 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male and female subjects aged ≥12 and ≤60 years, inclusive, with a genetically confirmed diagnosis 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.

You may not qualify if:

• Age of onset of disease ≥25 years.
• Higher degree of cardiomyopathy assessed by echocardiogram.
• Diabetes.

Sponsors & Collaborators

• Minoryx Therapeutics, S.L.: lead

Study Sites (5)

Hôpital Erasme-ULB

Brussels, B-1070, Belgium

Location

ICM, Groupe Hospitalier Pitié Salpêtrière

Paris, 75646, France

Location

Universitätsklinikum RWTH

Aachen, 52074, Germany

Location

Hospital Sant Joan de Déu

Barcelona, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Related Publications (1)

• Pandolfo M, Reetz K, Darling A, Rodriguez de Rivera FJ, Henry PG, Joers J, Lenglet C, Adanyeguh I, Deelchand D, Mochel F, Pousset F, Pascual S, Van den Eede D, Martin-Ugarte I, Vila-Brau A, Mantilla A, Pascual M, Martinell M, Meya U, Durr A. Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES). Neurol Genet. 2022 Nov 1;8(6):e200034. doi: 10.1212/NXG.0000000000200034. eCollection 2022 Dec.

  PMID: 36524101 DERIVED

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Alexandra Durr

  ICM, Groupe Hospitalier Pitié Salpêtrière, Paris, France, 75646

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2019
• First Posted: April 17, 2019
• Study Start: March 26, 2019
• Primary Completion: August 13, 2020
• Study Completion: September 14, 2020
• Last Updated: October 13, 2022

Record last verified: 2022-10

Locations

BE (1); DE (1); ES (2); FR (1)