Autosomal Dominant Polycystic Kidney DiseasePhase 2/3NCT00920309
Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
A Phase 2/3 study testing Rapamycin in people with Autosomal Dominant Polycystic Kidney Disease, run by Yale University at 1 site in United States.
Trial summary
Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation. This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney Disease
- What is being tested?
- Rapamycin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2009to Jul 2010
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- adult ADPKD patients aged 18-70
- combined kidney volume >1200 ml
- estimated creatinine clearance >60 ml/min
- absence of implanted ferromagnetic objects
Who cannot join
- Age >70
- uncontrolled hyperlipidemia
- Proteinuria >500 mg/day
- unstable cerebral aneurysm
- active coronary artery disease
- diagnosis of another systemic condition affecting kidney function (ie: diabetes, hepatitis B or C, HIV)
Show all 9Show fewer
- diagnosis of cancer other than skin cancer
- pregnancy or lactation
- presence of implanted ferromagnetic objects
Trial locations and contacts
1 site in 1 country.
United States1 site
Yale Center for Clinical Investigation
New Haven, Connecticut, 06520
About this study
Official title: Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy
Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation.
This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Patients will be followed by volumetric magnetic resonance imaging (MRI) to observe for change in kidney (and cyst) size. Blood and urine samples will also be collected to evaluate for change in biomarkers with treatment.
Read the detailed description
This is a 2 year, open label trial to evaluate the role of rapamycin as treatment for ADPKD. Patients will be randomized 2:1 to the rapamycin arm or to standard therapy. The dose of rapamycin will be adjusted so that patients obtain 24 trough levels of 4-6ng/ml. There will be a volumetric MRI measurement at the start and end of the treatment period. Patients will be monitored every 4 months throughout the study.
- Being tested
- Rapamycin, Standard of Care-Placebo
- Keywords
- Autosomal Dominant Polycystic Kidney Disease, rapamycin, ADPKD
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RapamycinDrug
Also called sirolimus
The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.
Standard of Care-PlaceboOther
| Study group | Role | What they receive |
|---|---|---|
| Rapamycin | Experimental | Drug: Rapamycin
Other Names:
sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml. Receives: Drug: Rapamycin |
| Standard of Care-Placebo | Placebo comparator | Standard of Care Receives: Other: Standard of Care-Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Kidney Volume (mL)
Measured: 2 years
Secondary outcomes (1)
Glomerular Filtration Rate (Kidney Function)
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2009 (estimated)
- Study startJun 2009
- Primary completionJul 2010
- Study completionJul 2010
Why it stopped: This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Autosomal Dominant Polycystic Kidney DiseaseStudies of the same condition, open and under way.
- More trials run by Yale UniversityThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yale University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Neera K Dahl, MD, PhD, Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00920309
- Sponsor's protocol ID
- HIC#0903004934
- Status last verified
- Mar 2014
- First submitted
- 12 Jun 2009
- Last updated
- 14 Apr 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy
- ClinicalTrials.gov ID
- NCT00920309
- Acronym / sponsor ID
- HIC#0903004934
- Keywords
- Autosomal Dominant Polycystic Kidney Disease, rapamycin, ADPKD
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Yale UniversityUniversity, hospital or other
- Study start
- Jun 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jul 2010 (actual)
- First posted
- 15 Jun 2009 (estimated)
- Results
- Posted 14 Apr 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation.
This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Patients will be followed by volumetric magnetic resonance imaging (MRI) to observe for change in kidney (and cyst) size. Blood and urine samples will also be collected to evaluate for change in biomarkers with treatment.
Detailed description
This is a 2 year, open label trial to evaluate the role of rapamycin as treatment for ADPKD. Patients will be randomized 2:1 to the rapamycin arm or to standard therapy. The dose of rapamycin will be adjusted so that patients obtain 24 trough levels of 4-6ng/ml. There will be a volumetric MRI measurement at the start and end of the treatment period. Patients will be monitored every 4 months throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rapamycin | Experimental | Drug: RapamycinDrug: Rapamycin Other Names: sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml. |
| Standard of Care-Placebo | Placebo comparator | Other: Standard of Care-PlaceboStandard of Care |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- adult ADPKD patients aged 18-70
- combined kidney volume >1200 ml
- estimated creatinine clearance >60 ml/min
- absence of implanted ferromagnetic objects
Key exclusion criteria (9 listed)
- Age >70
- uncontrolled hyperlipidemia
- Proteinuria >500 mg/day
- unstable cerebral aneurysm
- active coronary artery disease
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 |
|---|---|---|
| RapamycinAlso: sirolimusThe starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml. | Drug | Rapamycin |
| Standard of Care-Placebo | Other | Standard of Care-Placebo |
Primary outcome
Total Kidney Volume (mL)
Time frame: 2 years
1 secondary outcome
Glomerular Filtration Rate (Kidney Function)
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Neera K Dahl, MD, PhD principal investigator
Yale University
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 Rapamycin in people with Autosomal Dominant Polycystic Kidney Disease, run by Yale University at 1 site in United States.
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: stopped early, as of the sponsor's last update on 14 Apr 2014. 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 United States. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 14 Apr 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Total Kidney Volume (mL) (time frame: 2 years). 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 14 Apr 2014.