Trial Outcomes & Findings for Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy (NCT NCT00920309)

NCT ID: NCT00920309

Last Updated: 2014-04-14

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2/PHASE3
• Target enrollment: 21 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2014-04-14

Participant Flow

Participant milestones

Measure	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.	Standard of Care-Placebo
Overall Study STARTED	14	7
Overall Study COMPLETED	14	7
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

Baseline characteristics by cohort

Measure	Rapamycin n=14 Participants 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 n=7 Participants	Total n=21 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=93 Participants	7 Participants n=4 Participants	20 Participants n=27 Participants
Age, Categorical >=65 years	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Sex: Female, Male Female	5 Participants n=93 Participants	2 Participants n=4 Participants	7 Participants n=27 Participants
Sex: Female, Male Male	9 Participants n=93 Participants	5 Participants n=4 Participants	14 Participants n=27 Participants

PRIMARY outcome

Timeframe: 2 years

Population: Outcome Measures were not analyzed due to study termination

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: Outcome measures were not analyzed due to study termination

Outcome measures

Outcome data not reported

Adverse Events

Rapamycin

Serious events: 3 serious events

Other events: 14 other events

Deaths: 0 deaths

Standard of Care-Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Rapamycin n=14 participants at risk 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 n=7 participants at risk
Endocrine disorders Hyperglycemia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Flank Pain	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Renal and urinary disorders Proteinuria	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.

Other adverse events

Measure	Rapamycin n=14 participants at risk 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 n=7 participants at risk
Infections and infestations Cellulitis of the ear	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Infections and infestations Upper Respiratory Infesion	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Cardiac disorders Heart Palipitations with Hypertension	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Vascular disorders Edema/Weight gain	21.4% 3/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Nervous system disorders Headache	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Infections and infestations Apothous Ulcer	14.3% 2/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Hepatobiliary disorders Increased Liver Function Test	14.3% 2/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Endocrine disorders Hyperglycemia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Gastrointestinal disorders Emesis/Diarrhea	14.3% 2/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Nervous system disorders Impaired Depth Perception	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Musculoskeletal and connective tissue disorders Back Pain	28.6% 4/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Musculoskeletal and connective tissue disorders Arthralgias	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Insomnia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Sinus Headache	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Skin and subcutaneous tissue disorders Acne	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Skin and subcutaneous tissue disorders Skin Abscess	14.3% 2/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Allergic Rhinitis	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Endocrine disorders Dysuria	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Fatigue	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Epistaxis	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Gastrointestinal disorders Abdominal Pain	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Blood and lymphatic system disorders Thrombocytopenia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Blood and lymphatic system disorders Neutropenia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Gastrointestinal disorders Nausea	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Blood and lymphatic system disorders Anemia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Musculoskeletal and connective tissue disorders Left Hip Stiffness	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Weakness	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Respiratory, thoracic and mediastinal disorders Dyspnea	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Vascular disorders Pedal Edema	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Skin and subcutaneous tissue disorders Hives	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Musculoskeletal and connective tissue disorders Myalgia	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Ear/Neck Pain	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Gastrointestinal disorders Bloating/Flatulence	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Renal and urinary disorders Hematuria	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
General disorders Facial Sensitivity	7.1% 1/14 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.	0.00% 0/7 • The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.

Additional Information

Dr. Neera Dahl

Yale University School of Medicine

Phone: 203-785-4184

Email: neera.dahl@yale.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place