Sirolimus for Massive Polycystic Liver | NCT01680250

Studies
Sirolimus for Massive Polycystic Liver

NCT01680250

Sponsor

Seoul National University Hospital

Status

Unknown status

Last Update Posted

Sep 7, 2012Estimated

Enrollment

44Estimated

Phase

Phase 2Phase 3

Conditions

Polycystic Kidney Diseases

Interventions

Sirolimus

Countries

South Korea

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2012

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 2011

Primary Completion Date

Sep 2014Estimated

Completion Date

Aug 2015Estimated

First Submitted Date

Aug 30, 2012

First Submission Date that Met QC Criteria

Sep 3, 2012

First Posted Date

Sep 7, 2012Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 3, 2012

Last Update Posted Date

Sep 7, 2012Estimated

Name	Class
Wyeth is now a wholly owned subsidiary of Pfizer	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common causes of end stage renal disease (ESRD), affecting an estimated 0.2% of population. Of ADPKD patients, 58% in 15-24 year, 85% in 25-34 year, and 94% in 35-46 year olds suffer from polycystic liver in addition to polycystic kidneys. Several anti-proliferative drugs have been used in clinical trials to stop cyst growth both in liver and kidneys. Among them, octreotide and sirolimus have been shown to be one of the most promising drugs to reduce cyst volume. Sirolimus already has been used as one of the most potential oral immunosuppressants. Moreover, the serum trough level is quite easy to measure. Sirolimus is the mTOR inhibitor that has been proven to be effective in reducing cyst growth both in animal models. However, its efficacy and safety is not well proven in previous studies. This is a open-label, prospective study to evaluate the effectiveness and safety of Sirolimus to reduce cyst growth in ADPKD patients with massive polycystic liver.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

44Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Sirolimus	Experimental	Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL	Drug: Sirolimus

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Sirolimus	Drug	Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months	Sirolimus

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Total liver volume	Change in total liver volume	12 months

Secondary Outcomes

Measure	Description	Time Frame
Total liver volume	Change in total liver volume	24 months
Total kidney volume	Change in total kidney volume	12 month

Other Outcomes

Measure	Description	Time Frame
Abdominal pain	Abdominal pain quantified by Visual Analog Scale	12month
Abdominal pain	Abdominal pain quantified by Visual Analog Scale	24 month

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Curie Ahn, MD, PhD	Contact	82-2-2072-2222		curie@snu.ac.kr

Overall Officials

Name	Affiliation	Role
Curie Ahn, MD, PhD	Seoul National University Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Seoul National University Hospital	Recruiting	Seoul		110-744	South Korea

Condition Browse Module

MeSH Terms

ID	Term
D007690	Polycystic Kidney Diseases

Ancestor Terms

ID	Term
D052177	Kidney Diseases, Cystic
D007674	Kidney Diseases
D014570	Urologic Diseases
D052776	Female Urogenital Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

ID	Term
D018942	Macrolides
D007783	Lactones
D009930	Organic Chemicals