Clinical trials: Ciliopathies
Kidney, Polycystic
HALT Progression of Polycystic Kidney Disease Study B
The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two…
- Trial name
- HALT PKD B (NCT01885559)
- Testing
- Lisinopril, Telmisartan
- Where
- 7 sites in United States
- Volunteers
- 486 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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
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
Hippel-Lindau Disease, Kidney Cancer
17AAG to Treat Kidney Tumors in Von Hippel-Lindau Disease
This study will examine whether the drug 17AAG (17-allylamino 17-demethoxygeldanamycin) can shrink kidney tumors in patients with Von Hippel-Lindau disease (VHL), a rare, inherited syndrome in which patients develop tumors in…
- Trial name
- NCT00088374
- Testing
- 17 allylamino-17-demethoxygeldanamycin, 18 FDG (Fludeoxyglucose 18F), [15-O] H2O
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- National Cancer Institute (NCI)
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, 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
Chronic Kidney Disease, Autosomal Dominant Polycystic Kidney Disease
Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease
The purpose of the study is to determine whether tolvaptan is effective and safe for the treatment of late-stage chronic kidney disease due to autosomal dominant polycystic kidney disease (ADPKD)
- Trial name
- NCT02160145
- Testing
- Tolvaptan (OPC-41061)
- Where
- 230 sites in United States, Argentina and 20 more
- Volunteers
- 1,370 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
IgA Nephropathy, CKD Associated With Type 1 Diabetes
A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX
This multi-center, open-label Phase 2 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with the following rare chronic kidney diseases (CKD): CKD associated with type 1 diabetes…
- Conditions
- IgA Nephropathy, CKD Associated With Type 1 Diabetes, Focal Segmental Glomerulosclerosis, Autosomal Dominant Polycystic Kidney
- Trial name
- PHOENIX (NCT03366337)
- Testing
- Bardoxolone methyl capsules
- Where
- 30 sites in United States
- Volunteers
- 103 people
- Sponsor
- Biogen
Von Hippel-Lindau Syndrome
TKI 258 in Von Hippel-Lindau Syndrome (VHL)
The goal of this clinical research study is to learn if dovitinib can safely be given to patients who have VHL with a measurable hemangioblastoma (tumor of the central nervous system).
- Trial name
- NCT01266070
- Testing
- Dovitinib
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- M.D. Anderson Cancer Center
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in participants with Bardet Biedl…
- Trial name
- NCT03746522
- Testing
- Setmelanotide
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 52 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Autosomal Dominant Polycystic Kidney, Hypertension
Effect of Beetroot Juice on Reducing Hypertension in Autosomal Dominant Polycystic Kidney Disease
People with autosomal dominant polycystic kidney disease (ADPKD) develop high blood pressure and kidney disease. Previous studies have shown that a commonly occurring chemical, nitric oxide (NO), is reduced in ADPKD, and may…
- Conditions
- Autosomal Dominant Polycystic Kidney, Hypertension, Endothelial Dysfunction
- Trial name
- BEET-PKD (NCT05401409)
- Testing
- Beetroot juice, Nitrate-depleted beetroot juice
- Where
- 2 sites in Australia
- Volunteers
- 60 people
- Sponsor
- Western Sydney Local Health District
Von Hippel-Lindau Syndrome
Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome
This open-label study will pilot the use of systemic sunitinib malate, a dual inhibitor of vascular endothelial growth factor (VEGF) and platelet derived growth factor (PDGF), in five participants with Von Hippel-Lindau (VHL) to…
- Trial name
- VHL3 (NCT00673816)
- Testing
- Sunitinib Malate
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Eye Institute (NEI)
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Tolvaptan Extension Study in Participants With ADPKD
To demonstrate whether tolvaptan modifies ADPKD progression as measured by changes from Baseline (from Study 156-04-251) in total kidney volume (TKV) and renal function.
- Trial name
- TEMPO 4/4 (NCT01214421)
- Testing
- Tolvaptan
- Where
- 97 sites in United States, Argentina and 11 more
- Volunteers
- 1,083 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
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.
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
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 Disease, Adult
Roll-over Study to Assess Safety of Lixivaptan in Participants With ADPKD Who Completed Study PA-ADPKD-303
This is a Phase 3, open-label, roll-over study to demonstrate the continued hepatic and non-hepatic safety and renal efficacy of lixivaptan in participants with ADPKD who previously experienced abnormal liver chemistry test…
- Trial name
- NCT05208866
- Testing
- Lixivaptan
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Palladio Biosciences
Autosomal Dominant Polycystic Kidney Disease
Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of the trial was to determine the short-term effects of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) at various levels of renal function.
- Trial name
- NCT01336972
- Testing
- Tolvaptan
- Where
- Netherlands
- Volunteers
- 29 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
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
Autosomal Dominant Polycystic Kidney Disease
8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of this study is to compare the short-term effects of two tolvaptan formulations in patients with ADPKD.
- Trial name
- NOCTURNE (NCT01451827)
- Testing
- Tolvaptan MR, Tolvaptan IR
- Where
- 35 sites in United States
- Volunteers
- 178 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.