Fibrodysplasia Ossificans Progressive (FOP) is a rare genetically based disability, characterized by progressive heterotopic ossification, usually associated with congenital malformation of the toes. MedDRA version: 14.1 Level: HLGT Classification code 10028393 Term: Musculoskeletal and connective tissue disorders NEC System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
Conditions
Interventions
Product Name: Rosiglitazone
Pharmaceutical Form: Coated tablet
INN or Proposed INN: ROSIGLITAZONE MALEATE
CAS Number: 155141-29-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentr
Sponsors
A.O. UNIVERSITARIA INTEGRATA DI VERONA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ·patients of both sexes, older than 18 ·patients affected by FOP genetically confirmed ·patients with a joint mobility limitation > grade 2 (on a scale of 4 points) on at least one joint ·written informed consent signed by the patient or by a legally acceptable representative Are the trial subjects under 18? no Number of subjects for this age range: 0 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
Exclusion criteria: ·Patients affected by Bone Fibrodysplasia other than FOP ·Patients affected by heart failure, ischemic heart disease or with impaired liver function (ALT> 2 times the upper limit of normal) ·Patients under 18 ·Known hypersensitivity to the drug or the excipients 3.Pregnant or lactating women 4.alcoholism 5.drug dependence
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To verify the safety and efficacy of therapy with Rosiglitazone in patients with FOP evaluating the range variation of the joint mobility of wrists, elbows, shoulders, hips, knees and ankles using both a scale from 1(free joint) to 4 (locked joint) and a measure with a protractor; To evaluate a path-test of 10 metres;Secondary Objective: To evaluate changes in quality of life through the SF36 questionnaire; to evaluate changes of the bone turnover biomarkers concentration during the therapy; to evaluate changes of occiput-wall, finger-floor and bimalleolar distances; to monitor the ectopic ossifications (subgroup) size using an echographic evaluation.;Primary end point(s): 1)To evaluate the range of motion of wrists, elbows, shoulders, hips, knees, ankles before beginning treatment and after the end of treatment (follow-up) using a scale from 1 (free joint) to 4 (locked joint); 2)To evaluate the range of motion of wrists, elbows, shoulders, hips, knees, ankles before beginning treatment and after the end of treatment (follow-up) using a protractor (measured in degrees); 3)To record the time the patient spent (seconds) to perform the 10 metres-path test at the end of treatment (last visit).;Timepoint(s) of evaluation of this end point: nd | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1)to evaluate the quality of life using the SF-36 questionnaire before beginning treatment and at the end of treatment (follow-up); 2)To assess the bone turnover biomarkers (ALP(U/I), bone ALP (U/I), sCTX (mcg/l), PHT (pg/ml), DKK1 (pmol/l), osteocalcin (nmol/l), sclerostin (pmol/l), P1NP (ng/ml), Vit D (nmol/l)) concentration before beginning treatment and at the end of treatment (follow-up); 3)To record the occiput-wall distance (cm) before beginning treatment and at the end of treatment (follow-up); 4)To record the finger-floor distance (cm) before beginning treatment and at the end of treatment (follow-up); 5)To record the bimalleolar distance (cm) before beginning treatment and at the end of treatment (follow-up); 6)To evaluate the ectopic ossifications size (diameter in mm) using an echographic evaluation before beginning treatment and at the end of treatment (follow-up) (only in patients whose disease has not affected the traditional echographic windows);Timepoint(s) of evaluation of this end point: nd | — |
Countries
Italy
Contacts
Public ContactSSO di Reumatologia OCM
Azienda Ospedaliera Universitaria Integrata Verona
Outcome results
None listed