Study of Lanreotide to Treat Polycystic Kidney Disease

• Status: Unknown status
• Trial end date: 2017-12-01
• Target enrollment: 0
• Participant gender: All

Summary

Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive cyst formation in both kidneys, in most patients leading to end stage renal disease. It is the most common hereditary renal disease with a prevalence of approximately 1 in 1,000 persons. The majority of patients also have progressive cyst formation in the liver, leading to pain, gastrointestinal discomfort and sometimes the need for liver transplantation. At present there is no proven therapeutic intervention to slow the rate of disease progression in human ADPKD. The development of renoprotective treatments that are well tolerated, is therefore of major importance.

In this respect, somatostatin analogues are promising for especially polycystic liver disease, but also for the renal phenotype. However, the studies that have been performed thus far with these agents, were underpowered and of too short duration to reach a definitive conclusion on the potential reno- and hepatoprotective efficacy of somatostatin analogues. Therefore, the present study is designed as a randomised clinical trial with sufficient duration of follow-up to investigate whether the somatostatin analogue Lanreotide slows progression of polycystic kidney and liver disease in ADPKD-patients.

To this end, 300 ADPKD patients, aged 18-60years, with an eGFR 30-60 ml/min/1.73 m2) will be randomized 1:1 to standard care or monthly subcutaneous lanreotide injections on top off standard care. These 300 subjects will go through 15 study visits in 3 years and 1 follow up visit. During these visits, questionnaires will be filled in, physical examinations will be performed, blood will be drawn and urine collected. After study completion, rate of renal function decline in lanreotide treated subjects will be compared to that of subject who received standard care.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Medical Center Groningen
• Collaborators: Erasmus Medical Center; Leiden University Medical Center; Radboud University
• Treatments: Angiopeptin; Lanreotide; Somatostatin

Criteria

Inclusion Criteria:

1. Diagnosis of ADPKD, based upon the modified Ravine criteria

2. Age between 18 and 60 years.

3. eGFR (MDRD) between 30 and 60 ml/min/1.73 m2.

4. Providing informed consent.

Exclusion Criteria:

1. Patients who, in the opinion of the study investigator may present a safety risk.

2. Patients who are unlikely to adequately comply with the trial's procedures [due for instance to medical conditions likely to require an extended interruption or discontinuation, history of substance abuse or noncompliance).

3. a. Patients taking medications or having concomitant illnesses likely to confound endpoint assessments (e.g. nephrotoxic medications such as chronic NSAID, cyclosporine, lithium immunosuppressant use) b. Patients having concomitant illnesses likely to confound endpoint assessments (e.g. diabetes mellitus for which medication is needed and patients with proteinuria > 1 g /24hr).

4. Patients who underwent surgical or drainage interventions for cystic kidney disease the year before study-entry or are likely candidates for these procedures within 2 years of start of the study.

5. Patients taking other experimental (i.e.,non approved by FDA/EMA or indication of ADPKD) therapies.

6. Patients having used Lanreotide (or another somatostatin analogue) in the 3 months before study start.

7. Patients with known intolerance for Lanreotide (or another somatostatin analogue).

8. Unwillingness to comply with reproductive precautions. Women who are capable of becoming pregnant must be willing to comply with approved birth control from two-weeks prior to, and for 60 days after taking investigational product.

9. Women, who are pregnant or breastfeeding.

10. Patients, who suffer from cardiac arrhythmias, that are thought to be dangerous in combination with lanreotide administration.

11. Patients, who ever suffered from symptomatic gallstones and did not undergo cholecystectomy.

12. Patients, who have a medical history of pancreatitis.

13. Patients, who have a medical history of infected liver cysts.

In addition:

• Patients, who underwent liver cyst drainage or surgery in the year before, can enter the study, but will not be assessed for change in liver volume.

• Patients having contraindications to, or interference with MRI assessments, as dictated by local regulation, will not be allowed to undergo MR imaging. However, these patients can enter the study, but will not be assessed for change in kidney and/or liver volume.