Study comparing the effect of MIN-102 and an inactive substance to learn more about the effects of the MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients with Friedreich’s Ataxia, in which neither investigators nor subjects know what treatment the subject is receiving.

Phase 1

• Conditions: Friedreich’s Ataxia; MedDRA version: 20.0Level: PTClassification code 10017374Term: Friedreich's ataxiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2018-004405-64-DE
• Lead Sponsor: Minoryx Therapeutics BE, SA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-12-19
• Last Update Posted: 26 April 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

1. Signed Informed Consent by the subject or parent/legal guardian, and/or consent or assent for minors as required by national laws.
2. Male and female subjects aged =12 and =60 years, inclusive, with Friedreich’s Ataxia confirmed by genetic testing.
3. Total score on SARA of <25.
4. Score on SARA item 1 (gait) =2 and =6.
5. Male with a potentially fertile partner must be willing to use an acceptable method of contraception for the duration of the study and for 3 months after study drug discontinuation (acceptable methods are: use of a condom with spermicide or use of oral, implantable or injectable contraceptives, or intrauterine devices, or a diaphragm with spermicide or diaphragm with condom) or have had a vasectomy.
6. Female with childbearing potential must be willing to use highly effective contraceptive methods during screening, during the period of drug administration and for 30 days after stopping study drug administration. Highly effective contraception methods include the following:
? Total abstinence (defined as refraining from heterosexual intercourse during the entire period outlined above).
? Male or female sterilization. (bilateral tubal occlusion or vasectomized partner)
? Use of at least one of the following:
a. Use of oral, injectable, transdermal, intravaginal, or implantable hormonal methods of contraception
? combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: Oral, intravaginal or transdermal.
? progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable or implantable
b. Placement of an intrauterine device (IUD) or intrauterine system (IUS)
7. Female patients of childbearing potential must have a negative serum pregnancy test result within 7 days before first administration of study drug.
Are the trial subjects under 18? yes
Number of subjects for this age range: 18
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Age of onset of disease =25 years
2. Left ventricular ejection fraction (LVEF) <55%, on echocardiogram.
3. Subjects with a history of heart failure, ventricular arrhythmia, supraventricular tachycardia, or having a QTcF time of >480 msec in 3 consecutive ECG recordings taken at least 5 minutes apart.
4. Known intolerance to pioglitazone or other thiazolidinediones.
5. Subjects who are taking or have taken pioglitazone, or other thiazolidinediones, within the past 6 months prior to Screening.
6. Currently participating in or having participated in another interventional clinical study within 2 months prior to Screening.
7. Requiring other prohibited concomitant medication (see Table 4). Note: use of idebenone, coenzyme Q10, antioxidants, riboflavin, thiamine, vitamins C and E is permitted provided the dose has been constant for =3 months before Screening and is kept constant during the entire study.
8. Previous or current history of vesical polyps, bladder cell hyperplasia, or bladder cancer.
9. Previous or current history of other cancer, unless surgically resected and without evidence of recurrence for a minimum of 5 years.
10. Subjects with clinically significant anemia (i.e. hemoglobin below 110 g/L).
11. Glycated hemoglobin (HbA1c) levels >6.4% and fasting blood glucose levels = 0.9 times the lower limit of normal and = 1.1 times the upper limit of normal.
12. NT-proB-type natriuretic peptide level (NT pro BNP) >125 pg/mL
13. Abnormal liver enzyme tests for aspartate aminotransferase or alanine aminotransferase of >2 times the upper limit of normal or total bilirubin >1.5 times the upper limit of normal (unless
due to Gilbert’s syndrome).
14. A positive result on laboratory tests for hepatitis B surface antigen, hepatitis C antibody and human immunodeficiency virus antibody at screening.
15. Moderate or severe hepatic impairment (groups B and C according to the revised Child-Pugh classification; (Pugh, Murray-Lyon et al. 1973).
16. Chronic kidney disease (CKD) stages 3a or higher (according to CKD staging by Renal Association with an estimated glomerular filtration rate of <60 ml/min/1.73m2).
17. Contraindications for MRS/MRI procedure such as subjects with ferromagnetic materials in the body, such as dental braces, spinal rods, aneurysm clips, pacemakers, intraocular metal or cochlear implants.
18. Drug or alcohol abuse in the past 2 years by subject history and/or investigator assessment.
19. Conditions which could modify the absorption of the study drug, such as inflammatory bowel diseases, or stomach or intestinal resection.
20. Inability or unwillingness of the subject or subjects’ parents/caregivers to comply with the study procedures.
21. Other medical, neurological, psychiatric, or social conditions that in the investigator’s opinion are likely to confound the assessment of safety or efficacy, interfere with study conduct, or unfavorably alter the risk-benefit of subject participation.
22. Pregnant women as confirmed by a positive blood pregnancy test.
23. Female patients intending to breast feed a child while taking study drug or have taken study drug within 30 days after administration of the last dose.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified