Studio randomizzato, in doppio cieco, controllato verso placebo, per valutare la sicurezza e la tollerabilita` e per esplorare l efficacia di Lu AA24493, in pazienti con Atassia di Friedreich - ND
- Conditions
- Friedreich`s Ataxia (FRDA)MedDRA version: 9.1Level: LLTClassification code 10017374
- Registration Number
- EUCTR2008-003662-25-IT
- Lead Sponsor
- H.Lundbeck A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 36
1.The patient is able to read and understand the Patient Information Sheet. 2.The patient or their representative has signed the Informed Consent Form 3.The patient has FRDA, diagnosed according to ICD-10, and with genetic test demonstrating >400 GAA nucleotide triplet repeats on the shorter of the two frataxin alleles 4.The patient has a SARA (Stance) sub-score of <=6 5.The patient has a SARA (Gait) sub-score of <=6 6.The patient is a man or woman, aged 18 years or over 7.The patient, if female, must: -agree not to try to become pregnant during the study, AND -use adequate contraception (adequate contraception is defined as oral/systemic contraception, intrauterine device, diaphragm in combination with spermicide, or condom for male partner in combination with spermicide), OR -have had her last natural menstruation at least 24 months prior to screening, OR -have been surgically sterilised prior to screening, OR -have had a hysterectomy prior to screening, OR -not be sexually active
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Co-morbidity: 1.The patient has a clinically significant unstable illness, for example, hepatic or renal insufficiency, or a cardiovascular, pulmonary, gastrointestinal, endocrine, neurological (unrelated to the clinical manifestations of FRDA), infectious, neoplastic, or metabolic disturbance. Cardiac manifestations of FRDA (such as hypertrophic cardiomyopathy) are acceptable provided that the cardiac illness is considered stable and unlikely to compromise the patient`s safety during the study. 2.For patients previously exposed to EPO the patient has antibodies to EPO (established from assessment at screening, Visit 1) 3.The patient has or has had polycythemia, venous thromboembolism or other recent vascular event. 4.The patient has anaemia as determined by a haemoglobin concentration lower than 0.6mM (1g/dl) below local, gender adjusted, normal range. 5.The patient has a disease or takes medication that, in the opinion of the investigator, could interfere with the assessments of safety, tolerability, or efficacy. Medication and Treatment: 6.The patient used/uses disallowed recent or concomitant medication or it is anticipated that the patient will require treatment with at least one of the disallowed concomitant medications during the study. 7.The patient has been treated with idebenone within 6 weeks prior to screening. 8.The patient has been treated with erythropoietin or erythropoietin analogues within 16 weeks prior to screening. 9.The patient has been treated with any investigational medicinal product within 30 days or 5 half lives (whichever is longer) prior to screening. Patient characteristics: 10.The patient has a clinically significant abnormal ECG. Changes in ECG associated with cardiac manifestations of FRDA (such as hypertrophic cardiomyopathy) are acceptable provided that the cardiac illness is considered stable and unlikely to compromise the patient`s safety during the study. 11.The patient has clinically significant abnormal vital signs not relating to FRDA symptoms. 12.The patient has one or more laboratory values outside the normal range, based on the blood or urine samples taken at the Screening Visit or Baseline Visit, that are considered by the investigator to be clinically significant. 13.The patient has/has had a disorder related to alcohol or drug abuse, as defined in DSM IV TR, within 6 months prior to screening. 14.The patient has a history of severe drug allergy or hypersensitivity, or known hypersensitivity to EPO. Patient status: 15.The patient is pregnant or breast-feeding. 16.The patient, in the opinion of the investigator, is unlikely to comply with the clinical study protocol or is unsuitable for any reason. 17.The patient is a member of the site personnel or their immediate families. 18.The patient has previously participated in this study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with FRDA;Secondary Objective: - To explore biomarkers of efficacy (frataxin, 8-OHdG, peroxides, malondialdehyde) - To explore efficacy by neurological assessment (Scale for the Assessment and Rating of Ataxia (SARA), Friedreich s Ataxia Rating Scale (FARS)) - To explore efficacy by the Clinical Global Impression scales(CGI-I/S) - To explore population pharmacokinetic parameters of Lu AA24493 - To evaluate the immunogenicity of Lu AA24493;Primary end point(s): As the efficacy analyses will be exploratory, no primary endpoint will be defined.
- Secondary Outcome Measures
Name Time Method