RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY

• Register: EUCTR
• Last refreshed on: 10 July 2012
• Main ID: EUCTR2007-006557-25-IT
• Date of registration: 18/12/2007
• Prospective Registration: Yes
• Primary sponsor: AZIENDA OSPEDALIERA OSPEDALE POLICLINICO CONSORZIALE
• Public title: RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY
• Scientific title: RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY
• Date of first enrolment: 20/12/2007
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-006557-25
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: no Other: no

Countries of recruitment

Italy

Key inclusion & exclusion criteria

Inclusion criteria:
1. Age: > 18 and < 65 years. 2. Clinical, genetic and ultrasound diagnosis of ADPKD type I 3. Calculated creatinine clearance (MDRD formula) > 40 < 80 ml/min 4. Women who are of childbearing potential must have a negative serum pregnancy test before rapamycin administration and agree to use a medically acceptable method of contraception throughout the treatment period and for 3 months following discontinuation of rapamycin. Any woman becoming pregnant during the treatment period must discontinue the use of rapamycin
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) no
F.1.3.1 Number of subjects for this age range

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 < 3000/mm3; platelet count < 100,000/mm3; fasting triglycerides > 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

ADPKD type I
MedDRA version: 9.1 Level: SOC Classification code 10038359 Term: Renal and urinary disorders

Intervention(s)

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: Ramipril
Pharmaceutical Form: Tablet
INN or Proposed INN: Ramipril
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)

Secondary Objective: Kidney volume estimated by MRI, EGF urinary levels, activity of p70S6 kinase, side-effects of the drug.

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.

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.

Secondary ID(s)

RAPYD-STUDY