Friedreich's Ataxia
Conditions
Interventions
Trade Name: Victoza
Product Name: Victoza
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: LIRAGLUTIDE
CAS Number: 204656-20-2
Concentration unit: mg/ml milligram
Sponsors
CUB - Hôpital Erasme
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male/female patients with FRDA diagnosed by confirmed genetic testing 2. Age 18 years to 70 years 3. Women who are not pregnant or breast feeding, and who do not intend to become pregnant. Women of childbearing potential who must use a reliable contraceptive method and must provide a negative urine pregnancy test at entry into the study 4. Stable doses of all medications for 30 days prior to study entry and for the duration of the study. 5. Patient’s written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Current or history of acute or chronic pancreatitis 2. History of gastric or other major bleeding, thyroid neoplasia, hypothyroidism or hyperthyroidism 3. Subjects who have experienced side-effects previously from GLP-1 analogs 4. Use of another investigational product in the past 28 days 5. Current insulin treatment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: In this pilot sudy our aim is to assess the frataxin-inducing effects of exenatide and liraglutide in FRDA patients. Safety and tolerability of 5 weeks treatment with exenatide and liraglutide in patients with FRDA will be evaluated.;Secondary Objective: To evaluate the change in clinical rating scales of Friedreich's Ataxia on liraglutide and exenatide treatment.;Primary end point(s): The change in frataxin expression measured in PBMCs, platelets and buccal swabs on a 5-week liraglutide and exenatide treatment. The safety and tolerability of 5 weeks of treatment.;Timepoint(s) of evaluation of this end point: Once weekly during the treatment and after 2 and 4 weeks of wash-out from the IMP. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change in neurological clinical rating scales for Friedreich's Ataxia: SARA (Scale for the Assessment and Rating of Ataxia) and INAS (Inventory of Non-Ataxic Symptoms) count; ADL assessments (Activities of Daily Living).;Timepoint(s) of evaluation of this end point: Once weekly during the treatment and after 2 and 4 weeks of wash-out from the IMP. | — |
Countries
Belgium
Contacts
Public ContactMiriam Cnop
ULB Center for Diabetes Research, Université Libre de Bruxelles
Outcome results
None listed