Clinical trials: Ciliopathies
Autosomal Dominant Polycystic Kidney Disease
The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
This is a Phase 2, open-label, parallel-group, multiple dose study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and tolerability of multiple doses of lixivaptan in Autosomal Dominant Polycystic Kidney…
- Trial name
- NCT03487913
- Testing
- Lixivaptan
- Where
- 13 sites in United States
- Volunteers
- 31 people
- Sponsor
- Palladio Biosciences
Autosomal Dominant Polycystic Kidney Disease
A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (2) [Extension of Study 156-05-002]
ADPKD patients who were enrolled in Study 156-05-002 will receive repeated oral administration of OPC-41061 at doses of 15 mg twice daily (morning and evening).
- Trial name
- NCT01022424
- Testing
- OPC-41061
- Where
- Japan
- Volunteers
- 13 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Polycystic Kidney, Autosomal Dominant
Feasibility Study of Metformin Therapy in ADPKD
This study is being done to determine if treatment with metformin, a drug widely used for the treatment of diabetes type 2, is safe and well tolerated by individuals with Autosomal Dominant Polycystic Kidney Disease (ADPKD) who…
- Trial name
- NCT02903511
- Testing
- Metformin
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- University of Colorado, Denver
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Diet as a Potential Treatment for Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to learn if dietary habits can affect vasopressin secretion in patients with autosomal dominant polycystic kidney disease.
- Trial name
- NCT02225860
- Testing
- Diet and water adjustment
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Tufts Medical Center
Von Hippel-Lindau Syndrome, Renal Cell Carcinoma
Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow
The goal of this clinical research study is to learn if sunitinib malate (SU011248) can help to control VHL. The safety of this drug will also be studied.
- Conditions
- Von Hippel-Lindau Syndrome, Renal Cell Carcinoma, Hemangioblastoma
- Trial name
- NCT00330564
- Testing
- SU011248
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- M.D. Anderson Cancer Center
Chronic Kidney Diseases, Alport Syndrome
An Extended Access Program for Bardoxolone Methyl in Patients With CKD (EAGLE)
This extended access study will assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with…
- Conditions
- Chronic Kidney Diseases, Alport Syndrome, Autosomal Dominant Polycystic Kidney
- Trial name
- EAGLE (NCT03749447)
- Testing
- Bardoxolone methyl
- Where
- 87 sites in United States, Australia and 4 more
- Volunteers
- 270 people
- Sponsor
- Biogen
Autosomal Dominant Polycystic Kidney Disease
Short-term Effects of an SGLT2 Inhibitor on Divalent Ions in Autosomal Dominant Polycystic Kidney Disease
This study aims to better understand electrolyte handling in patients with autosomal dominant polycystic kidney disease treated with the SGLT2 inhibitor Empagliflozin.
- Trial name
- SIDIA (NCT06435858)
- Testing
- Empagliflozin
- Where
- 2 sites in Switzerland
- Volunteers
- 16 people
- Sponsor
- Cantonal Hospital Graubuenden
Healthy Volunteers
A Study of RGLS8429 in Healthy Volunteers
Primary Objective To assess the safety and tolerability of single ascending doses of RGLS8429 Secondary Objectives To identify dose-limiting toxicity (DLT) and to determine the maximum tolerated dose (MTD) of a single SC dose of…
- Trial name
- NCT05429073
- Testing
- RGLS8429
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Regulus Therapeutics Inc.
Bardet-Biedl Syndrome, POMC Deficiency Obesity
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
This is a phase 3 open-label, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric participants aged 2 to <6 years with obesity due to either biallelic variants…
- Conditions
- Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Trial name
- NCT04966741
- Testing
- Setmelanotide
- Where
- 6 sites in United States, Australia and 2 more
- Volunteers
- 12 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Polycystic Kidney Disease, Adult, ADPKD
Safety of Lixivaptan in Subjects Previously Treated With Tolvaptan for Autosomal Dominant Polycystic Kidney Disease
This is a Phase 3, open-label, repeat-dose study designed to assess liver safety, non-liver safety, and efficacy of lixivaptan in participants who previously experienced liver chemistry test abnormalities while treated with…
- Trial name
- ALERT (NCT04152837)
- Testing
- Lixivaptan
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Palladio Biosciences
Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease
Octreotide in Severe Polycystic Liver Disease
This study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection…
- Conditions
- Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease, Hepatomegaly, Liver Diseases
- Trial name
- NCT00426153
- Testing
- Octreotide
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Mayo Clinic
Von Hippel-Lindau Disease
Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. These tumors are more aggressive in some people than in others.
- Trial name
- NCT00062166
- Where
- United States
- Volunteers
- 340 people
- Sponsor
- National Cancer Institute (NCI)
Polycystic Kidney, Autosomal Dominant
Daily Caloric Restriction and Intermittent Fasting in Overweight and Obese Adults With Autosomal Dominant Polycystic Kidney Disease
The proposed research will determine the feasibility of delivering two behavioral weight loss interventions for 1 year in adults with autosomal dominant polycystic kidney disease (ADPKD) who are overweight or obese.
- Trial name
- NCT03342742
- Testing
- Weight Loss
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- University of Colorado, Denver
Autosomal Dominant Polycystic Kidney, ADPKD
A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON
This international, multi-center, randomized, double-blind, placebo-controlled Phase 3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with ADPKD.
- Trial name
- FALCON (NCT03918447)
- Testing
- Bardoxolone methyl oral capsule
- Where
- 137 sites in United States, Australia and 8 more
- Volunteers
- 667 people
- Sponsor
- Biogen
Kidney, Polycystic, Autosomal Dominant
High Water Intake to Slow Progression of Polycystic Kidney Disease
Polycystic kidney disease (PKD) is a genetic disease that occurs in 1 in 500 individuals and leads to kidney failure in half of all affected. Currently, no treatments exist for PKD.
- Trial name
- NCT00784030
- Testing
- Water
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Polycystic Kidney, Autosomal Dominant
Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease
This study will test to see if metformin is safe and if it is tolerated compared to placebo in adult Autosomal Dominant Polycystic Kidney Disease (ADPKD) patients with beginning stages of chronic kidney disease.
- Trial name
- TAME (NCT02656017)
- Testing
- Metformin
- Where
- 2 sites in United States
- Volunteers
- 97 people
- Sponsor
- Kyongtae Ty Bae, M.D., Ph.D.
Autosomal Dominant Polycystic Kidney, ADPKD
Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD
The goal of the study was to compare and evaluate safety and efficacy of tesevatinib 50 milligrams (mg) versus placebo in participants with autosomal dominant polycystic kidney disease (ADPKD).
- Trial name
- NCT03203642
- Testing
- Tesevatinib
- Where
- 19 sites in United States
- Volunteers
- 80 people
- Sponsor
- Kadmon, a Sanofi Company
Polycystic Kidney, Autosomal Dominant
Tolvaptan Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
This study's purpose is to evaluate the long-term safety of open-label tolvaptan regimens to determine the maximally-tolerated dose and acquire pilot efficacy data in patients with autosomal dominant polycystic kidney disease…
- Trial name
- TEMPO 2:4 (NCT00413777)
- Testing
- Tolvaptan
- Where
- 11 sites in United States
- Volunteers
- 46 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Autosomal Dominant Polycystic Kidney Disease
A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) [Extension of Study 156-04-001]
Investigation into the long-term safety and efficacy of OPC-41061 in repeated oral administrations at doses of 15 mg twice daily in patients with ADPKD who completed the preceding dose-finding study (156-04-001).
- Trial name
- NCT00841568
- Testing
- OPC-41061
- Where
- Japan
- Volunteers
- 17 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Polycystic Kidney, Autosomal Dominant
Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of this study is to determine whether the medication pravastatin will ameliorate renal and cardiovascular disease over a 3-year period in children and young adults with autosomal dominant polycystic kidney disease…
- Trial name
- NCT00456365
- Testing
- pravastatin
- Where
- United States
- Volunteers
- 110 people
- Sponsor
- University of Colorado, Denver