NCT01680250

Brief Summary

The purpose of this study is to evaluate the effectiveness and safety of Sirolimus in reducing liver volume in autosomal dominant polycystic kidney disease.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
44

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2011

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2011

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

August 30, 2012

Completed
8 days until next milestone

First Posted

Study publicly available on registry

September 7, 2012

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2014

Completed
11 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2015

Completed
Last Updated

September 7, 2012

Status Verified

September 1, 2012

Enrollment Period

3 years

First QC Date

August 30, 2012

Last Update Submit

September 3, 2012

Conditions

Polycystic Kidney Diseases

Keywords

Total liver volumeLiver cyst

Outcome Measures

Primary Outcomes (1)

  • Total liver volume

    Change in total liver volume

    12 months

Secondary Outcomes (7)

  • Total liver volume

    24 months

  • Total kidney volume

    12 month

  • Estimated glomerular filtration rate

    12 month

  • Urinary biomarker

    12 month

  • Total kidney volume

    24 month

  • +2 more secondary outcomes

Other Outcomes (5)

  • Abdominal pain

    12month

  • Abdominal pain

    24 month

  • Infection

    24 month

  • +2 more other outcomes

Study Arms (1)

Sirolimus

EXPERIMENTAL

Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL

Drug: Sirolimus

Interventions

SirolimusDRUG

Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months

Also known as: Rapamune
Sirolimus

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age 18 - 65
  • Patients diagnosed as ADPKD based upon the unified criteria for ultrasonographic diagnosis of ADPKD
  • Polycystic liver with total liver volume \> 2500 mL or symptomatic polycystic liver
  • Estimated glomerular filtration rate (IDMS-traceable MDRD equation) \>= 30 mL/min/1.73m2

You may not qualify if:

  • Concomitant systemic renal parenchymal or urinary tract disease (random urine albumin-to-creatinine ratio \> 500 mg/g)
  • WBC \< 4,000/uL, platelet \< 100,000/uL, or hemoglobin \< 10.0 g/dL
  • Diabetes mellitus, cancer, or psychiatric disorder
  • Increased liver enzymes (2-fold above normal value)
  • Hypercholesterolemia (fasting cholesterol \> 200mg/dL) or hypertriglyceridemia (\>150 mg/dL) not controlled by lipid lowering therapy
  • Infection with hepatitis B, C, HIV
  • Any condition that could prevent full comprehension of the purpose and risks of the study
  • Pregnant or lactating women or fertile women without effective contraception
  • History of intervention, such as cyst aspiration or embolization in past 1 year

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Seoul National University Hospital

Seoul, 110-744, South Korea

RECRUITING

MeSH Terms

Conditions

Polycystic Kidney Diseases

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Kidney Diseases, CysticKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCiliopathiesGenetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Curie Ahn, MD, PhD

    Seoul National University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Curie Ahn, MD, PhD

CONTACT

82-2-2072-2222curie@snu.ac.kr

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 30, 2012

First Posted

September 7, 2012

Study Start

September 1, 2011

Primary Completion

September 1, 2014

Study Completion

August 1, 2015

Last Updated

September 7, 2012

Record last verified: 2012-09

Locations

KR(1)