Friedreich AtaxiaPhase 2FRAMESNCT03917225
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
A Phase 2 study testing MIN-102 in people with Friedreich Ataxia, run by Minoryx Therapeutics, S.L. at 5 sites in 4 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- MIN-102
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 26 Mar 2019to 14 Sep 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- Age of onset of disease ≥25 years.
- Higher degree of cardiomyopathy assessed by echocardiogram.
- Diabetes.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
Spain2 sites
Hospital Sant Joan de Déu
Barcelona
Hospital Universitario La Paz
Madrid, 28046
About this study
Official title: A Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients With Friedreich's Ataxia
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
Read the detailed description
This study investigated whether treatment with MIN-102 was able to influence clinical, neurophysiological, and imaging parameters, as well as various peripheral and central nervous system biochemical markers related to mitochondrial dysfunction, in patients with Friedreich's ataxia (FRDA), over a period of 48 weeks in a double-blind, placebo-controlled design.
Patients were screened following written consent (and assent if the patient was a minor) at the Screening visit (V-1). Eligible patients attended a Baseline visit (V0) within a maximum of 28 days after V-1 and were randomized in a 2:1 ratio to receive an individualized starting dose of MIN-102 or placebo, which they took daily for 48 weeks. Patients received individualized starting doses based on gender and age, which were subsequently modified based on pharmacokinetic (PK) parameters obtained from blood samples at V1 to achieve a target MIN-102 exposure of 170 μg.hr/mL.
In addition to the Screening visit (V-1) and Baseline visit (V0), patients were evaluated at 2 interim safety visits (ISV) occurring 2 and 8 weeks after V0 (ISV1 and ISV2; permitted to be home visits performed by a Good Clinical Practice [GCP]-certified nurse), and at 4 weeks (V1), 12 weeks (V2), 24 weeks (V3), 36 weeks (V4), and 48 weeks (V5) after V0.
Results of all scheduled assessments were made available to the investigator as soon as possible. Patients received regularly scheduled phone calls at 6, 10, 16, 20, 28, 32, 40, and 44 weeks after V0 to review changes in concomitant medications and adverse events (AEs), particularly for symptoms possibly indicative of cardiac failure. A Final Follow-up Visit (FUV) took place 4 weeks after the last dose of study drug.
Evaluations consisted of imaging evaluations at V0, V3, and V5, evaluations of clinical status using the Scale for the Assessment and Rating of Ataxia (SARA), cerebellar composite functional scale (CCFS), global clinical rating scales, and patient questionnaires at V0, V3, and V5, assessment of biochemical markers in plasma at V0, V2, V3, and V5, and blood sampling for plasma levels of MIN-102 and its main metabolite (M3) at all scheduled on-site visits (except for ISV1 and ISV2). Assessments for safety and tolerability included collection of AEs, as well as electrocardiograms (ECGs), echocardiograms, and laboratory tests. Palatability was assessed at V0, V1, V2, V3, and V4. Measurements of motor evoked potentials (MEPs) and assessment of biochemical markers in cerebrospinal fluid (CSF) were optional evaluations at V0, V3, and V5. All assessments for safety, tolerability, and biochemical markers in plasma were also performed at premature discontinuation.
- Conditions
- Friedreich Ataxia
- Being tested
- MIN-102, Placebo
- Keywords
- Leriglitazone, Friedreich's ataxia
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MIN-102Drug
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.
PlaceboDrug
Once-daily dosing with a volume specified by the pharmacokinetic specialist. Oral suspension.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to 48 weeks
Secondary outcomes (24)
Change from Baseline in SARA total score at Week 48
The SARA is an 8-item clinical rating scale. Five of the items are scored with points between 0 and 4, gait is scored with points between 0 and 8, and stance and speech disturbance are scored with points between 0 and 6. Total maximum score is 40 indicating maximum disability. SARA was determined at Screening, Baseline, Week 24, and Week 48.
Measured: Baseline to 48 weeks
Change from Baseline in cervical spinal cord (C2-C7) in fractional anisotropy at Week 48
MRI diffusion tensor imaging (DTI) was used for assessments at Baseline, Week 24, and Week 48 to measure rate of disease progression.
Measured: 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
MRI DTI was used for assessments at Baseline, Week 24, and Week 48 to measure rate of disease progression.
Measured: 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
This metabolite ratio was used to measure rate of disease progression at Baseline, Week 24, and Week 48.
Measured: Baseline to 48 weeks
Change from Baseline in quantitative susceptibility mapping (QSM) for iron concentration (ppb) at Week 48
QSM was acquired in the cerebellum, using a gradient echo MRI sequence. It was used to quantify local tissue properties of the dentate nucleus, which reflect iron concentration. Measurements were taken at Baseline, Week 24, and Week 48.
Measured: Baseline to 48 weeks
Change from Baseline in dentate nuclei volume at Week 48
QSM was acquired in the cerebellum, using a gradient echo MRI sequence. It was used to quantify local tissue properties of the dentate nucleus, including estimating the volume of the dentate nucleus. Reported values were normalized by total intracranial volume (TICV) and were dimensionless. Measurements were taken at Baseline, Week 24, and Week 48.
Measured: Baseline to 48 weeks
Change from Baseline in fixel-based analysis (FBA) of the brain at Week 48: fiber density (FD)
Diffusion metrics related to the integrity of the corticospinal tract (CST), selected brain regions, and spinal cord fibers were extracted from diffusion-weighted images. Fixel-based analyses of the CST, the superior and inferior cerebellar peduncles (SCP and ICP), the posterior limb of the internal capsule (PLIC), the superior corna radiata (SCR), and the medial lemniscus (mLEM) were summarized to determine changes to the fibers. Fiber density measured number of axons in these structures.
Measured: Baseline to 48 weeks
Change from Baseline in fixel-based analysis (FBA) of the brain at Week 48: fiber cross-section (FC)
Diffusion metrics related to the integrity of the CST, selected brain regions, and spinal cord fibers were extracted from diffusion-weighted images. Fixel-based analyses of the CST, the SCP and ICP, the PLIC, the SCR, and the mLEM were summarized to determine changes to the fibers. Fiber cross-section measured the area of the fiber cross-section in these structures.
Measured: Baseline to 48 weeks
Change from Baseline in fixel-based analysis (FBA) of the brain at Week 48: fiber density and cross-section (FDC)
Diffusion metrics related to the integrity of the CST, selected brain regions, and spinal cord fibers were extracted from diffusion-weighted images. Fixel-based analyses of the CST, the SCP and ICP, the PLIC, the SCR, and the mLEM were summarized to determine changes to the fibers. Fiber density and cross-section was a combination of both FD and FC in these structures.
Measured: Baseline to 48 weeks
Change from Baseline in Cerebellar Composite Functional Scale (CCFS) at Week 48
The CCFS is a derived score based on results of two tests which are carried out with the dominant hand: the 9-hole peg test and clicking. The 9-hole peg test measures the time it takes for the patient to place 9 pegs in holes. In the clicking test, the subject has to press 2 buttons with the index finger of their dominant hand. The buttons are mounted on a board and have to be pressed in an alternating fashion for 10 times. As an outcome, Z-scores were calculated by subtracting the expected time that was obtained from healthy controls from the time measured in the patient. CCFS was calculated as log10 (7+Z pegboard dominant hand/10 + 4*Z click dominant hand/10). The CCFS assesses the severity of cerebellar ataxia; higher scores indicate greater severity of disease.
Measured: Baseline to 48 weeks
Change from Baseline in quality of life as measured by European Quality of Life 5 Dimensions (EQ-5D-5L) at Week 48
The EQ-5D-5L is a patient-rated scale with 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) where patients are asked to rate themselves according to presented responses at 5 levels of severity for each dimension (level 1 indicates no problems; level 5 indicates disability). They also rate their overall health on the particular study day on a scale from 0 to 100 points, with 0 indicating the worst possible health. The outcomes are the sum of the scores of the responses on the 5 dimensions, the overall rating of health in terms of points, and an index score based on both scores. Index scores range between 0 and 1 (with higher scores indicating better quality of life).
Measured: Baseline to 48 weeks
Change from Baseline in fatigue severity scale (FSS) at Week 48
Patients rate their level of fatigue on the FSS by responding to 9 presented statements where a score of 1 means maximum disagreement and 7 means strongest agreement. The outcome is the sum of scores over the 9 items. Higher scores indicate greater severity of fatigue.
Measured: Baseline to 48 weeks
Change from Baseline in Activities of Daily Living subscale of Friedreich's Ataxia Rating Scale (FARS) at Week 48
Activities of daily living were assessed by patients using section II of the FARS. Section II contained 9 items where subjects were rated on a scale between 0 (normal function) and 4 (most severely disturbed function). The scores for the individual 9 items are added to yield the total score. A higher score indicates a greater level of disability.
Measured: Baseline to 48 weeks
Change from Baseline in Clinician Global Impression of Severity (CGI-S) at Week 48
The CGI-S rates the severity of illness based on a 1-7 point scale completed by the investigator, where 1 = "normal, not at all ill" and 7 = "among the most extremely-ill patients".
Measured: Baseline to 48 weeks
Assessment from Baseline in Clinician Global Impression of Improvement (CGI-I) at Week 48
The CGI-I is a tool for assessing total overall improvement as judged by the investigator that considers whether or not the improvement is entirely due to the study medication treatment. The CGI-I rates the improvement based on a 1-7 point scale, where 1 = "very much improved" and 7 = "very much worse".
Measured: 24 weeks and 48 weeks
Assessment from Baseline in Patient Global Impression of Improvement (PGI-I) at Week 48
The PGI-I is a tool for assessing total overall improvement as judged by the patient during study medication treatment. The PGI-I rates the improvement based on a 1-7 point scale, where 1 = "very much improved" and 7 = "very much worse".
Measured: 24 weeks and 48 weeks
Percentage of patients responding to palatability of study drug questions
Palatability of the study drug was assessed by the patient at Baseline, Week 4, Week 12, Week 24, and Week 36 immediately after swallowing and 10 minutes after swallowing. Patients rated the taste as "Super-good", "Good", "Neutral", "Bad", and "Super-bad". Patients were also asked if they would take the study drug every day (possible responses were "Yes", "Not sure", and "No". The percentage of patients in each category was presented.
Measured: Baseline, Week 4, Week 12, Week 24, and Week 36
Frequency and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Adverse events were monitored from Baseline until the Final Follow-up Visit (28 days after the last dose of study drug). TEAEs were defined as AEs occurring on or after the first dose of study drug. Number of patients with AEs and SAEs graded between Grade 1 (mild) and Grade 5 (death) was presented.
Measured: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: percentage change from Baseline in body weight (kg)
Weight measured at Baseline and all subsequent in-person visits.
Measured: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: change from Baseline in blood pressure (mmHg)
Systolic and diastolic blood pressure measured at Baseline and all subsequent in-person visits.
Measured: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: change from Baseline in pulse rate (beats/min)
Measured at Baseline and all subsequent in-person visits.
Measured: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: change from Baseline in body temperature (°C)
Measured at baseline and all subsequent in-person visits.
Measured: Baseline to Final Follow-up Visit (approximately 52 weeks)
Number of patients with abnormal, clinically significant electrocardiogram (ECG) and echocardiogram interpretations
Echocardiograms were performed at Screening, Week 4, Week 12, Week 24, Week 24, Week 48, and the Final Follow-up Visit; 12-lead ECGs were performed at Screening, Baseline (pre-dose and 3 hours post-dose), 3 hours post-dose at Week 4, Week 12, Week 24, Week 36, and at Week 48 and the Final Follow-up Visit. The number of patients with normal, abnormal not clinically significant, abnormal clinically significant, and not assessable was tabulated.
Measured: Baseline to Final Follow-up Visit (approximately 52 weeks)
Number of patients with abnormal, clinically significant physical examination findings
Body systems examined included abdomen, extremities, heart, lungs, and skin.
Measured: Screening to Final Follow-up Visit (approximately 56 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2019
- Study start26 Mar 2019
- Primary completion13 Aug 2020
- Study completion14 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alexandra Durr, Principal investigator, ICM, Groupe Hospitalier Pitié Salpêtrière, Paris, France, 75646
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03917225
- Sponsor's protocol ID
- MT-2-03
- Acronym
- FRAMES
- Status last verified
- Oct 2022
- First submitted
- 10 Apr 2019
- Last updated
- 13 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 36524101
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT03917225
- Acronym / sponsor ID
- FRAMES / MT-2-03
- Conditions
- Friedreich Ataxia
- Keywords
- Leriglitazone, Friedreich's ataxia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Minoryx Therapeutics, S.L.Industry (pharma, biotech, devices)
- Study start
- 26 Mar 2019 (actual)
- Primary completion
- 13 Aug 2020 (actual)
- Study completion
- 14 Sep 2020 (actual)
- First posted
- 17 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
This study investigated whether treatment with MIN-102 was able to influence clinical, neurophysiological, and imaging parameters, as well as various peripheral and central nervous system biochemical markers related to mitochondrial dysfunction, in patients with Friedreich's ataxia (FRDA), over a period of 48 weeks in a double-blind, placebo-controlled design.
Patients were screened following written consent (and assent if the patient was a minor) at the Screening visit (V-1). Eligible patients attended a Baseline visit (V0) within a maximum of 28 days after V-1 and were randomized in a 2:1 ratio to receive an individualized starting dose of MIN-102 or placebo, which they took daily for 48 weeks. Patients received individualized starting doses based on gender and age, which were subsequently modified based on pharmacokinetic (PK) parameters obtained from blood samples at V1 to achieve a target MIN-102 exposure of 170 μg.hr/mL.
In addition to the Screening visit (V-1) and Baseline visit (V0), patients were evaluated at 2 interim safety visits (ISV) occurring 2 and 8 weeks after V0 (ISV1 and ISV2; permitted to be home visits performed by a Good Clinical Practice [GCP]-certified nurse), and at 4 weeks (V1), 12 weeks (V2), 24 weeks (V3), 36 weeks (V4), and 48 weeks (V5) after V0.
Results of all scheduled assessments were made available to the investigator as soon as possible. Patients received regularly scheduled phone calls at 6, 10, 16, 20, 28, 32, 40, and 44 weeks after V0 to review changes in concomitant medications and adverse events (AEs), particularly for symptoms possibly indicative of cardiac failure. A Final Follow-up Visit (FUV) took place 4 weeks after the last dose of study drug.
Evaluations consisted of imaging evaluations at V0, V3, and V5, evaluations of clinical status using the Scale for the Assessment and Rating of Ataxia (SARA), cerebellar composite functional scale (CCFS), global clinical rating scales, and patient questionnaires at V0, V3, and V5, assessment of biochemical markers in plasma at V0, V2, V3, and V5, and blood sampling for plasma levels of MIN-102 and its main metabolite (M3) at all scheduled on-site visits (except for ISV1 and ISV2). Assessments for safety and tolerability included collection of AEs, as well as electrocardiograms (ECGs), echocardiograms, and laboratory tests. Palatability was assessed at V0, V1, V2, V3, and V4. Measurements of motor evoked potentials (MEPs) and assessment of biochemical markers in cerebrospinal fluid (CSF) were optional evaluations at V0, V3, and V5. All assessments for safety, tolerability, and biochemical markers in plasma were also performed at premature discontinuation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MIN-102 | Experimental | Drug: MIN-102MIN-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). |
| Placebo | Placebo comparator | Drug: PlaceboMatches MIN-102 visually and by taste. Dosing is once daily at approximately the same time each morning throughout the entire treatment phase (48 weeks). |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- Age of onset of disease ≥25 years.
- Higher degree of cardiomyopathy assessed by echocardiogram.
- Diabetes.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MIN-102Once-daily dosing with a volume specified by the pharmacokinetic specialist to achieve the desired plasma exposure. MIN-102 oral suspension, strength 15 mg/ml. | Drug | MIN-102 |
| PlaceboOnce-daily dosing with a volume specified by the pharmacokinetic specialist. Oral suspension. | Drug | Placebo |
Primary outcome
Change from Baseline in spinal cord area cervical segment C2-C3 [mm²]
Time frame: Baseline to 48 weeks
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.
24 secondary outcomes
Change from Baseline in SARA total score at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in cervical spinal cord (C2-C7) in fractional anisotropy at Week 48
Time frame: 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
Time frame: 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
Time frame: Baseline to 48 weeks
Change from Baseline in quantitative susceptibility mapping (QSM) for iron concentration (ppb) at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in dentate nuclei volume at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in fixel-based analysis (FBA) of the brain at Week 48: fiber density (FD)
Time frame: Baseline to 48 weeks
Change from Baseline in fixel-based analysis (FBA) of the brain at Week 48: fiber cross-section (FC)
Time frame: Baseline to 48 weeks
Change from Baseline in fixel-based analysis (FBA) of the brain at Week 48: fiber density and cross-section (FDC)
Time frame: Baseline to 48 weeks
Change from Baseline in Cerebellar Composite Functional Scale (CCFS) at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in quality of life as measured by European Quality of Life 5 Dimensions (EQ-5D-5L) at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in fatigue severity scale (FSS) at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in Activities of Daily Living subscale of Friedreich's Ataxia Rating Scale (FARS) at Week 48
Time frame: Baseline to 48 weeks
Change from Baseline in Clinician Global Impression of Severity (CGI-S) at Week 48
Time frame: Baseline to 48 weeks
Assessment from Baseline in Clinician Global Impression of Improvement (CGI-I) at Week 48
Time frame: 24 weeks and 48 weeks
Assessment from Baseline in Patient Global Impression of Improvement (PGI-I) at Week 48
Time frame: 24 weeks and 48 weeks
Percentage of patients responding to palatability of study drug questions
Time frame: Baseline, Week 4, Week 12, Week 24, and Week 36
Frequency and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: percentage change from Baseline in body weight (kg)
Time frame: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: change from Baseline in blood pressure (mmHg)
Time frame: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: change from Baseline in pulse rate (beats/min)
Time frame: Baseline to Final Follow-up Visit (approximately 52 weeks)
Vital signs: change from Baseline in body temperature (°C)
Time frame: Baseline to Final Follow-up Visit (approximately 52 weeks)
Number of patients with abnormal, clinically significant electrocardiogram (ECG) and echocardiogram interpretations
Time frame: Baseline to Final Follow-up Visit (approximately 52 weeks)
Number of patients with abnormal, clinically significant physical examination findings
Time frame: Screening to Final Follow-up Visit (approximately 56 weeks)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2022.
- Derived from this study: PubMed 36524101Pandolfo 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.
Related trials for Friedreich Ataxia
RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich Ataxia
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexandra Durr principal investigator
ICM, Groupe Hospitalier Pitié Salpêtrière, Paris, France, 75646
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MIN-102 in people with Friedreich Ataxia, run by Minoryx Therapeutics, S.L. at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Spain, Belgium, France, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in spinal cord area cervical segment C2-C3 [mm²] (time frame: Baseline to 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03917225, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2022.