ADPKD type I MedDRA version: 9.1 Level: SOC Classification code 10038359 Term: Renal and urinary disorders
Conditions
Interventions
Trade Name: RAPAMUNE*100CPR RIV 1MG
Pharmaceutical Form: Tablet
INN or Proposed INN: Sirolimus
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1-
Product Name: Ram
Sponsors
AZIENDA OSPEDALIERA OSPEDALE POLICLINICO CONSORZIALE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age: > 18 and 40 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Evidence of active systemic or localized major infection at the time of initial rapamycin administration. 2. Evidence of infiltrate, cavitation, or consolidation on chest X-ray obtained during pre-study screening. 3. Use of any investigational drug or treatment up to 4 weeks prior to enrolling in the study and during the 6-month treatment phase. 4. Known hypersensitivity to rapamycin and its derivatives. 5. Treatment with terfenadine, cisapride, astemizole, pimozide, or ketoconazole (that is not discontinued prior to rapamycin administration). 6. Patients with a screening/baseline total white blood cell count 300 mg/dL ; fasting total cholesterol > 350 mg/dL ; urinary protein excretion rate > 1 g/24hours. 7. Other concomitant glomerular disease 8. Psychiatric disorders and any condition that might prevent full comprehension of the purposes and risks of the study 9. None or suspected malignancies within three years before enrolment in the study, with the exception of adequately treatment basal cell and squamous cell carcinomas of the skin 10. HIV positive patients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Thus the main objective of the present study is to define whether pharmacological inhibition of this serine-theronine kinase may reduce or even revert the progressive growth of renal cyst in population of APKD patients with moderate renal failure over a period of 24 months. We will also evaluate whether 24 months of treatment with rapamycin may also influence the progression of renal failure towards end stage renal disease.;Secondary Objective: Kidney volume estimated by MRI, EGF urinary levels, activity of p70S6 kinase, side-effects of the drug.;Primary end point(s): The primary end-points of the study is a combination of ESRD, doubling of serum creatinine (sCr), increase of 50% of the baseline sCr. | — |
Countries
Italy
Outcome results
None listed