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Autosomal Dominant Polycystic Kidney Disease (ADPKD)Phase 3DIPAK1NCT01616927

Study of Lanreotide to Treat Polycystic Kidney Disease

A Phase 3 study testing Lanreotide in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), run by University Medical Center Groningen at 4 sites in Netherlands.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 60 years
What is being tested?
Lanreotide
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
300 participants
Where
4 sitesNetherlands
Study dates
Jun 2012to Dec 2017
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 60 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Diagnosis of ADPKD, based upon the modified Ravine criteria
  • Age between 18 and 60 years.
  • eGFR (MDRD) between 30 and 60 ml/min/1.73 m2.
  • Providing informed consent.

Who cannot join

  • Patients who, in the opinion of the study investigator may present a safety risk.
  • 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).
  • 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).
  • 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.
  • Patients taking other experimental (i.e.,non approved by FDA/EMA or indication of ADPKD) therapies.
  • Patients having used Lanreotide (or another somatostatin analogue) in the 3 months before study start.
Show all 16Show fewer
  • Patients with known intolerance for Lanreotide (or another somatostatin analogue).
  • 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.
  • Women, who are pregnant or breastfeeding.
  • Patients, who suffer from cardiac arrhythmias, that are thought to be dangerous in combination with lanreotide administration.
  • Patients, who ever suffered from symptomatic gallstones and did not undergo cholecystectomy.
  • Patients, who have a medical history of pancreatitis.
  • 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.

Trial locations and contacts

4 sites in 1 country.

Netherlands4 sites
  • University Medical Center Groningen

    Groningen

  • Leiden University Medical Center

    Leiden

  • Radboud University Medical Center

    Nijmegen

  • Erasmus Medisch Centrum

    Rotterdam

Other trials in Netherlands

About this study

Official title: The DIPAK 1 Study: A Randomised, Controlled Clinical Trial Assessing the Efficacy of Lanreotide to Halt Disease Progression in ADPKD

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.

Read the detailed description

Aims:

First, to determine whether Lanreotide attenuates progression of the renal phenotype in ADPKD patients as measured by change in rate of renal function decline and change in renal volume growth.

Second, to determine whether Lanreotide modifies progression of the liver phenotype in the subset of ADPKD patients with moderate to severe polycystic liver disease as measured by change in liver volume.

Methods:

Investigator driven, randomized, multi-center, controlled clinical trial.

Study population:

300 subjects, diagnosed with ADPKD, based on the revised Ravine criteria, with advanced disease and high likelihood of rapid disease progression (eGFR between 30 and 60 ml/min/1.73 m2 and age between 18 and 60 years).

Intervention:

Patients will be randomized (1:1) into two groups. One group will receive a dose of Lanreotide 120 mg sc every 28 days for 30 months. The dose of Lanreotide will be eGFR (BSA unadjusted) dependent. Subjects that reach an eGFR <30ml/min during the study will receive Lanreotide 90 mg sc every 28 days. Down-titration will also occur in case of dose related side effects. The other group of patients will receive standard care.

Main study endpoint:

Change in renal function in Lanreotide versus not treated patients, as assessed as slope through serial eGFR measurements over time during the treatment phase of the trial, with the value obtained at month 3 as first eGFR value for slope analysis.

Main secondary outcome variables:

  • to determine whether Lanreotide modifies ADPKD progression as measured by change in renal volume in the overall study population,
  • to determine whether Lanreotide modifies ADPKD progression as measured by change in liver volume in the subset of ADPKD patients with moderate to severe polycystic liver disease
  • to determine whether Lanreotide changes the quality of life
  • to determine whether Lanreotide is well tolerated
Being tested
Lanreotide
Keywords
ADPKD, lanreotide, polycystic kidney, polycystic liver
Medical subject headings
Polycystic Kidney, Autosomal Dominant, Polycystic Kidney Diseases, Polycystic liver disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LanreotideDrug

    Also called somatuline, lanreotide autogel

    Lanreotide will be administered once every 4 weeks as a subcutaneous injection

Study groups in NCT01616927
Study groupRoleWhat they receive
standard careNo interventionSubjects in this arm will receive standard care
LanreotideExperimentalNot described
Receives: Drug: Lanreotide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in renal function

    Change in renal function in Lanreotide versus not treated patients, as assessed as slope through all eGFR measurements taken at study visits during the treatment phase of the trial (n=10), with the value obtained at month 3 as first eGFR value for slope analysis.

    Measured: serial eGFR measurements from month 3 until end of treatment visit (month 30)

Secondary outcomes (6)
  1. change in renal volume

    to determine whether Lanreotide modifies ADPKD progression as measured by change in renal volume in the overall study population. Renal volume is measured at baseline, after 30 months of treatment and 3 months afterwards (follow-up visit).

    Measured: baseline and 3 months after end of treatment (follow-up; month 33)

  2. change in liver volume

    to determine whether Lanreotide modifies ADPKD progression as measured by change in liver volume in the subset of ADPKD patients with moderate to severe polycystic liver disease, defined as a liver volume of >2000 ml. Liver volume is measured at baseline, at month 30 and 3 months afterwards (follow-up visit))

    Measured: Baseline and end of treatment (month 30)

  3. change in quality of life

    to determine whether Lanreotide changes the quality of life (using specific questionnaires). These questionnaires will be filled in at baseline, after 3 months of treatment, after 1 year, after 2 years, at end of treatment (30 months) and at follow-up (3 months after end of treatment)

    Measured: baseline-end of treatment (month 30)

  4. tolerance

    to determine whether lanreotide is safe and well tolerated. This is assessed by investigating (severe)adverse events, vital signs, performing physical examination and clinical laboratory tests.

    Measured: baseline-end of treatment(month 30)

  5. change in renal function

    change in renal function in Lanreotide versus not treated patients, as assessed as change in eGFR from baseline versus eGFR obtained 3 months after cessation of treatment.

    Measured: baseline and 3 months after end of treatment (follow-up; month 33)

  6. Incidence of worsening renal function

    incidence of worsening renal function defined as a 30% decrease in eGFR and/or need for renal replacement therapy computed from pre-treatment eGFR

    Measured: from baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jun 2012 (estimated)
  2. Study startJun 2012
  3. Primary completionAug 2017 (estimated)
  4. Study completionDec 2017 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
300 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01616927
Sponsor's protocol ID
DIPAK1
Other IDs
2011-005017-37
Acronym
DIPAK1
Status last verified
May 2017
First submitted
6 Jun 2012
Last updated
23 May 2017
Results posted
No
Data monitoring committee
Yes

Publications

  • Meijer E, Drenth JP, d'Agnolo H, Casteleijn NF, de Fijter JW, Gevers TJ, Kappert P, Peters DJ, Salih M, Soonawala D, Spithoven EM, Torres VE, Visser FW, Wetzels JF, Zietse R, Gansevoort RT; DIPAK Consortium. Rationale and design of the DIPAK 1 study: a randomized controlled clinical trial assessing the efficacy of lanreotide to Halt disease progression in autosomal dominant polycystic kidney disease. Am J Kidney Dis. 2014 Mar;63(3):446-55. doi: 10.1053/j.ajkd.2013.10.011. Epub 2013 Dec 15.

    PubMed 24342522
  • Lantinga MA, D'Agnolo HM, Casteleijn NF, de Fijter JW, Meijer E, Messchendorp AL, Peters DJ, Salih M, Spithoven EM, Soonawala D, Visser FW, Wetzels JF, Zietse R, Drenth JP, Gansevoort RT; DIPAK Consortium. Hepatic Cyst Infection During Use of the Somatostatin Analog Lanreotide in Autosomal Dominant Polycystic Kidney Disease: An Interim Analysis of the Randomized Open-Label Multicenter DIPAK-1 Study. Drug Saf. 2017 Feb;40(2):153-167. doi: 10.1007/s40264-016-0486-x.

    PubMed 27995519
  • St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

    PubMed 39356039
  • Aapkes SE, de Haas RJ, Bernts LHP, Blijdorp CJ, Dekker SEI, van Gastel MDA, Meijer E, Veldman A, Drenth JPH, Gansevoort RT; DIPAK consortium. Incident Gallstones During Somatostatin Analog Treatment are Associated with Acute Biliary Complications Especially After Discontinuation. Drugs R D. 2021 Jun;21(2):179-188. doi: 10.1007/s40268-021-00342-7. Epub 2021 Mar 29.

    PubMed 33779943
  • van Aerts RMM, Kievit W, D'Agnolo HMA, Blijdorp CJ, Casteleijn NF, Dekker SEI, de Fijter JW, van Gastel M, Gevers TJ, van de Laarschot LFM, Lantinga MA, Losekoot M, Meijer E, Messchendorp AL, Neijenhuis MK, Pena MJ, Peters DJM, Salih M, Soonawala D, Spithoven EM, Visser FW, Wetzels JF, Zietse R, Gansevoort RT, Drenth JPH; DIPAK-1 Investigators. Lanreotide Reduces Liver Growth In Patients With Autosomal Dominant Polycystic Liver and Kidney Disease. Gastroenterology. 2019 Aug;157(2):481-491.e7. doi: 10.1053/j.gastro.2019.04.018. Epub 2019 Apr 22.

    PubMed 31022403
  • Meijer E, Visser FW, van Aerts RMM, Blijdorp CJ, Casteleijn NF, D'Agnolo HMA, Dekker SEI, Drenth JPH, de Fijter JW, van Gastel MDA, Gevers TJ, Lantinga MA, Losekoot M, Messchendorp AL, Neijenhuis MK, Pena MJ, Peters DJM, Salih M, Soonawala D, Spithoven EM, Wetzels JF, Zietse R, Gansevoort RT; DIPAK-1 Investigators. Effect of Lanreotide on Kidney Function in Patients With Autosomal Dominant Polycystic Kidney Disease: The DIPAK 1 Randomized Clinical Trial. JAMA. 2018 Nov 20;320(19):2010-2019. doi: 10.1001/jama.2018.15870.

    PubMed 30422235

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Lanreotide in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), run by University Medical Center Groningen at 4 sites in Netherlands.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 23 May 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in renal function (time frame: serial eGFR measurements from month 3 until end of treatment visit (month 30)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01616927, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2017.

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