Polycystic Kidney DiseasesPhase 2/3SILVERNCT01680250
Sirolimus for Massive Polycystic Liver
A Phase 2/3 study testing Sirolimus in people with Polycystic Kidney Diseases, run by Seoul National University Hospital at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Polycystic Kidney Diseases
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteSouth Korea
- Study dates
- Sep 2011to Aug 2015
- Phase
- Phase 2/3
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Seoul National University Hospital
Seoul, 110-744
Curie Ahn, MD, PhD, 82-2-2072-2222, curie@snu.ac.kr
About this study
Official title: An Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic Liver
The purpose of this study is to evaluate the effectiveness and safety of Sirolimus in reducing liver volume in autosomal dominant polycystic kidney disease.
Read the 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.
- Conditions
- Polycystic Kidney Diseases
- Being tested
- Sirolimus
- Keywords
- Total liver volume, Liver cyst
- Medical subject headings
- Polycystic Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune
Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Experimental | Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total liver volume
Change in total liver volume
Measured: 12 months
Secondary outcomes (7)
Total liver volume
Change in total liver volume
Measured: 24 months
Total kidney volume
Change in total kidney volume
Measured: 12 month
Estimated glomerular filtration rate
Change in estimated glomerular filtration rate
Measured: 12 month
Urinary biomarker
Urinary biomarker level
Measured: 12 month
Total kidney volume
Change in total kidney volume
Measured: 24 month
Estimated glomerular filtration rate
Change in estimated glomerular filtration rate
Measured: 24 month
Urinary biomarker
Urinary biomarker level
Measured: 24 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2012 (estimated)
- Study startSep 2011
- Primary completionSep 2014 (estimated)
- Study completionAug 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycystic Kidney DiseasesStudies of the same condition, open and under way.
- More trials run by Seoul National University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Seoul National University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Wyeth is now a wholly owned subsidiary of Pfizer
- Responsible party
- Sponsor
- Investigators
- Curie Ahn, MD, PhD, Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01680250
- Sponsor's protocol ID
- SILVER
- Acronym
- SILVER
- Status last verified
- Sep 2012
- First submitted
- 30 Aug 2012
- Last updated
- 7 Sep 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic Liver
- ClinicalTrials.gov ID
- NCT01680250
- Acronym / sponsor ID
- SILVER / SILVER
- Conditions
- Polycystic Kidney Diseases
- Keywords
- Total liver volume, Liver cyst
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- Seoul National University HospitalUniversity, hospital or other
- Collaborators
- Wyeth is now a wholly owned subsidiary of Pfizer
- Study start
- Sep 2011
- Primary completion
- Sep 2014 (estimated)
- Study completion
- Aug 2015 (estimated)
- First posted
- 7 Sep 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Experimental | Drug: SirolimusSirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: RapamuneSirolimus administration for 12 months followed by conventional therapy alone for additional 12 months | Drug | Sirolimus |
Primary outcome
Total liver volume
Time frame: 12 months
Change in total liver volume
7 secondary outcomes
Total liver volume
Time frame: 24 months
Total kidney volume
Time frame: 12 month
Estimated glomerular filtration rate
Time frame: 12 month
Urinary biomarker
Time frame: 12 month
Total kidney volume
Time frame: 24 month
Estimated glomerular filtration rate
Time frame: 24 month
Urinary biomarker
Time frame: 24 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Sep 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Curie Ahn, MD, PhD principal investigator
Seoul National University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Sirolimus in people with Polycystic Kidney Diseases, run by Seoul National University Hospital at 1 site in South Korea.
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 9 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 7 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Curie Ahn, MD, PhD (82-2-2072-2222, curie@snu.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total liver volume (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Sep 2012.