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This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Standard of Care-Placebo | Placebo Comparator | Standard of Care |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rapamycin | Drug | 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Total Kidney Volume (mL) | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Glomerular Filtration Rate (Kidney Function) | 2 years |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Neera K Dahl, MD, PhD | Yale University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale Center for Clinical Investigation | New Haven | Connecticut | 06520 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Rapamycin | 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. |
| FG001 | Standard of Care-Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Standard of Care-Placebo | Other |
|
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Rapamycin | 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. |
| BG001 | Standard of Care-Placebo | |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Total Kidney Volume (mL) | Outcome Measures were not analyzed due to study termination | Posted | 2 years |
|
| |||||||||||||||||||||||
| Secondary | Glomerular Filtration Rate (Kidney Function) | Outcome measures were not analyzed due to study termination | Posted | 2 years |
|
|
The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Rapamycin | 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. | 3 | 14 | 14 | 14 | ||
| EG001 | Standard of Care-Placebo | 0 | 7 | 0 | 7 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hyperglycemia | Endocrine disorders | Non-systematic Assessment |
| ||
| Flank Pain | General disorders | Non-systematic Assessment |
| ||
| Proteinuria | Renal and urinary disorders | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cellulitis of the ear | Infections and infestations | Non-systematic Assessment |
| ||
| Upper Respiratory Infesion | Infections and infestations | Non-systematic Assessment |
| ||
| Heart Palipitations with Hypertension | Cardiac disorders | Non-systematic Assessment |
| ||
| Edema/Weight gain | Vascular disorders | Non-systematic Assessment |
| ||
| Headache | Nervous system disorders | Non-systematic Assessment |
| ||
| Apothous Ulcer | Infections and infestations | Non-systematic Assessment |
| ||
| Increased Liver Function Test | Hepatobiliary disorders | Non-systematic Assessment |
| ||
| Hyperglycemia | Endocrine disorders | Non-systematic Assessment |
| ||
| Emesis/Diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Impaired Depth Perception | Nervous system disorders | Non-systematic Assessment |
| ||
| Back Pain | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
| ||
| Arthralgias | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
| ||
| Insomnia | General disorders | Non-systematic Assessment |
| ||
| Sinus Headache | General disorders | Non-systematic Assessment |
| ||
| Acne | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Skin Abscess | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Allergic Rhinitis | General disorders | Non-systematic Assessment |
| ||
| Dysuria | Endocrine disorders | Non-systematic Assessment |
| ||
| Fatigue | General disorders | Non-systematic Assessment |
| ||
| Epistaxis | General disorders | Non-systematic Assessment |
| ||
| Abdominal Pain | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Thrombocytopenia | Blood and lymphatic system disorders | Non-systematic Assessment |
| ||
| Neutropenia | Blood and lymphatic system disorders | Non-systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Anemia | Blood and lymphatic system disorders | Non-systematic Assessment |
| ||
| Left Hip Stiffness | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
| ||
| Weakness | General disorders | Non-systematic Assessment |
| ||
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| ||
| Pedal Edema | Vascular disorders | Non-systematic Assessment |
| ||
| Hives | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Myalgia | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
| ||
| Ear/Neck Pain | General disorders | Non-systematic Assessment |
| ||
| Bloating/Flatulence | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Hematuria | Renal and urinary disorders | Non-systematic Assessment |
| ||
| Facial Sensitivity | General disorders | Non-systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Neera Dahl | Yale University School of Medicine | 203-785-4184 | neera.dahl@yale.edu |
| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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| >=65 years |
|
| Male |
|