Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients With Autosomal Dominant Polycystic Kidney Disease

Novartis

Status and phase: Completed; Phase 4

Conditions: Autosomal Dominant Polycystic Kidney Disease

Treatments: Drug: Everolimus; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00414440

2006-001485-16

CRAD001ADE12

Details and patient eligibility

About

This study will assess whether everolimus (RAD001) is effective in preventing cyst and kidney expansion as well as worsening of renal function in patients with ADPKD and whether the application of 5 mg/day everolimus as monotherapy is safe and well tolerated.

Enrollment: 431 patients

Sex: All

Ages: 18 to 50 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

1. Clinical diagnosis of autosomal dominant polycystic kidney disease ADPKD
2. Chronic kidney disease (CKD) stage II / III
3. Females capable of becoming pregnant must have a negative serum pregnancy test within 7 days prior to or at baseline, and are required to practice an approved method of birth control for the duration of the study and for a period of 6 weeks following discontinuation of study medication, even where there has been a history of infertility

Exclusion Criteria

1. ADPKD patients with normal renal function
2. ADPKD patients with CKD stage IV
3. Patients with a history of subarachnoid bleeding
4. Patients with a history of severe infections
5. Patients with life-threatening urinary tract or cyst infection in the past
6. Patients who have received any investigational drug within four weeks prior to baseline
7. Patients who have been treated with any non-protocol immunosuppressive drug or treatment within one month prior to baseline

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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431 participants in 2 patient groups, including a placebo group

Everolimus

Experimental group

Description:

Patients in the everolimus group initially received 5 mg/day everolimus divided in 2 equal doses (i.e. 2.5 mg b.i.d.). Dose adjustments were performed to achieve a blood trough level of 3-8 ng/mL (maximum daily dose: 10 mg/day \[5 mg b.i.d.\]).

Treatment:

Drug: Everolimus

Placebo

Placebo Comparator group

Description:

Placebo tablets equivalent to the dosage of everolimus 5 mg/day, divided in 2 equal doses.

Treatment:

Drug: Placebo