175 completed, 78 recruiting, 40 unknown, 27 active not recruiting. 71 Phase 2, 66 Not applicable, 47 Phase 3, 36 Phase 1, 9 Phase 4.
Follow Ciliopathies: new trials by email Literature Review of Ciliopathies trials Research Report on Ciliopathies
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose 14C-VX-407 - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Not yet recruiting | Vertex Pharmaceuticals Incorporated | 8 | 2026-10-19 | 2026-10-09 |
| Effects of VX-407 on the Pharmacokinetics of Combined Oral Contraceptives VX-407, LNG/EE, NGM/EE - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Recruiting | Vertex Pharmaceuticals Incorporated | 148 | 2026-09-01 | 2026-09-30 |
| Bempedoic Acid Therapy for Polycystic Kidney Disease Bempedoic Acid 180 MG Oral Tablet, Placebo Capsule(s) - ADPKD (Autosomal Dominant Polycystic Kidney Disease) | Phase 2 | Recruiting | Kenneth Hallows | 120 | 2026-09-01 | 2026-09-29 |
| Malnutrition and Sarcopenia Among Primary Ciliary Dyskinesia Physical assessments of individuals - Primary Ciliary Dyskinesia (PCD) | Not yet recruiting | Izmir Democracy University | 15 | 2026-08-03 | 2026-07-31 | |
| Comparison of Individuals With Primary Ciliary Dyskinesia and Healthy Controls Physical Evaluations in Children with Primary Ciliary Dyskinesia, Physical Evaluations in Healthy Children - Primary Ciliary Dyskinesia (PCD) | Not yet recruiting | Izmir Democracy University | 30 | 2026-08-01 | 2026-07-31 | |
| Clearance in Primary Ciliary Dyskinesia Exercise, HS pretreatment with Exercise - Primary Ciliary Dyskinesia, PCD | Not applicable | Not yet recruiting | University of North Carolina, Chapel Hill | 8 | 2026-07-30 | 2026-07-13 |
| Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas Von Hippel-Lindau Disease, Central Nervous System Hemangioblastoma | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 25 | 2026-07-15 | 2026-07-15 | |
| Dietary Modulation of Urinary MCP-1 in ADPKD Carbohydrate Restricted, Plant Dominant Diet, usual diet - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Not applicable | Not yet recruiting | Assaf-Harofeh Medical Center | 36 | 2026-07-10 | 2026-06-26 |
| A Phase 1 Study of Single and Multiple Ascending Doses of GV-100 GV-100, Placebo - ADPKD (Autosomal Dominant Polycystic Kidney Disease) | Phase 1 | Recruiting | Gilva Therapeutics Inc. | 80 | 2026-06-23 | 2026-08-26 |
| Managing Depressive Symptoms in ADPKD Mayo Clinic-Approved Education, Digital Neuropathy, Capacity Coaching - Autosomal Dominant Polycystic Kidney Disease | Not applicable | Recruiting | Mayo Clinic | 105 | 2026-06-12 | 2026-08-07 |
| Drug-Drug Interactions of JMKX003142 in Healthy Participants JMKX003142 tablets, Fluconazole Capsules, Cocktail Substrates (Midazolam Oral Solution, Rosuvastatin Calcium Tablets, and Digoxin Tablets) - ADPKD (Autosomal Dominant Polycystic Kidney Disease) | Phase 1 | Not yet recruiting | Jemincare | 112 | 2026-05-11 | 2026-05-04 |
| Di-PKD: A Pilot Trial of Dietary Intervention in Patients With Autosomal Dominant Polycystic Kidney Disease Interventional Diet - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Not applicable | Not yet recruiting | Loma Linda University | 30 | 2026-05-01 | 2026-01-30 |
| Quality of Life, Psychological Impact, and Care-related Challenges in Patients Affected by Von Hippel-Lindau Syndrome. VHL - Von Hippel-Lindau Syndrome | Active not recruiting | IRCCS Ospedale San Raffaele | 200 | 2026-05-01 | 2026-05-11 | |
| Digital Physiotherapy for Pediatric Chronic Suppurative Lung Diseases Videoconferencing (Vsee platform), Specially designed website (FysΑΩ) - Chronic Suppurative Lung Disease, Protracted Bacterial Bronchitis | Not applicable | Not yet recruiting | University of Thessaly | 32 | 2026-05-01 | 2026-01-30 |
| 18F-T2 PET/CT Imaging for CAIX Positive Solid Tumors 18F-T2 PET/CT - Clear Cell Renal Cell Cancer (ccRCC), Urothelial Carcinoma (UC) | Not applicable | Recruiting | Peking University First Hospital | 200 | 2026-04-27 | 2026-05-14 |
| Developing Resource Interventions for Healthcare Professionals and Patients to Improve Knowledge About Fertility in Primary Ciliary Dyskinesia Online educational resource, Toolkit for healthcare professionals - Primary Ciliary Dyskinesia (PCD), Fertility | Not applicable | Not yet recruiting | University of Southampton | 30 | 2026-04-01 | 2026-04-15 |
| A Study to Investigate the Effects of JMKX003142 on QTc Interval in Healthy Adults JMKX003142 tablets, Placebo - ADPKD (Autosomal Dominant Polycystic Kidney Disease) | Phase 1 | Not yet recruiting | Jemincare | 24 | 2026-03-27 | 2026-03-13 |
| Extension Study for Participants in Studies That Include Belzutifan (MK-6482-043/LITESPARK-043) Belzutifan, Nivolumab, Lenvatinib - Von Hippel-Lindau Disease, Malignant Neoplasms | Phase 3 | Recruiting | Merck Sharp & Dohme LLC | 450 | 2026-03-23 | 2026-10-21 |
| Mass Balance and Absolute Bioavailability Study of JMKX003142 In Healthy Volunteers [14C]JMKX003142, JMKX003142 tablets, [14C]JMKX003142 solution for infusion - ADPKD (Autosomal Dominant Polycystic Kidney Disease) | Phase 1 | Not yet recruiting | Jemincare | 8 | 2026-03-18 | 2026-03-10 |
| Metabolic Impacts of Ren-Nu: A Dietary Program for Polycystic Kidney Disease Ren-Nu and KetoCitra - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Not applicable | Recruiting | Richard Fatica | 20 | 2026-02-25 | 2026-03-06 |
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Polycystic Kidney, Autosomal Dominant (182) · Ciliary Motility Disorders (79) · Polycystic Kidney Diseases (58) · von Hippel-Lindau Disease (55) · Kartagener Syndrome (21) · Cystic Fibrosis (20) · Bardet-Biedl Syndrome (18) · Kidney Diseases (15) · Renal Insufficiency, Chronic (15) · Carcinoma, Renal Cell (14) · Polycystic liver disease (13) · Hypertension (11) · Hemangioblastoma (10) · Obesity (9) · Polycystic Kidney, Autosomal Recessive (8)
Abnormalities, Multiple (374) · Congenital Abnormalities (374) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (374) · Genetic Diseases, Inborn (370) · Female Urogenital Diseases and Pregnancy Complications (241) · Urogenital Diseases (241) · Female Urogenital Diseases (240) · Male Urogenital Diseases (239) · Urologic Diseases (237) · Kidney Diseases (222) · Kidney Diseases, Cystic (214) · Polycystic Kidney Diseases (159) · Cardiovascular Diseases (94) · Nervous System Diseases (88)
Tolvaptan (21) · Blood Specimen Collection (9) · Sirolimus (9) · belzutifan (5) · lixivaptan (5) · setmelanotide (5) · Everolimus (4) · Metformin (4) · Sodium Chloride (4) · XL647 (4) · lanreotide (4) · Diet (3) · Diet, Ketogenic (3) · Pravastatin (3) · Ranibizumab (3)
Mayo Clinic (15) · Otsuka Pharmaceutical Development & Commercialization, Inc. (12) · University of Colorado, Denver (12) · Mario Negri Institute for Pharmacological Research (11) · University of North Carolina, Chapel Hill (11) · Assistance Publique - Hôpitaux de Paris (9) · National Eye Institute (NEI) (7) · Vertex Pharmaceuticals Incorporated (7) · Jemincare (6) · Gazi University (5) · M.D. Anderson Cancer Center (5) · National Cancer Institute (NCI) (5) · Palladio Biosciences (5) · ReCode Therapeutics (5) · The Rogosin Institute (5)
United States (170) · France (45) · Italy (39) · United Kingdom (38) · Germany (34) · Canada (31) · Netherlands (30) · Spain (25) · Belgium (23) · Australia (21) · Turkey (Türkiye) (20) · Denmark (19) · Japan (18) · China (16) · Switzerland (13)
Jon Blumenfeld, MD (4) · David Meeker, MD (3) · Eric Jonasch, MD (3) · John Matthews, MBBS, MCRP, PhD (3) · Marva Moxey-Mims, M.D. (3) · Naris Nilubol, M.D. (3) · Vicente Torres, MD, PhD (3) · Arlene Chapman, M.D. (2) · Arlene Chapman, MD (2) · Changlin Mei, MD (2) · Claudia E Kuehni, Prof (2)
PMID 39356039, PMID 41385299, PMID 37250503, PMID 41348481, PMID 36191725, PMID 37349694, PMID 38837240, PMID 31014270, PMID 34783716, PMID 21903984, PMID 23477994, PMID 37729939, PMID 37986188, PMID 38072392, PMID 40377984, PMID 11562511, PMID 16221708, PMID 16627867, PMID 16858015, PMID 17059358, PMID 17449765, PMID 17515466, PMID 17804777, PMID 17868472, PMID 18026621, PMID 18372389, PMID 19141364, PMID 19525519, PMID 19540516, PMID 20350728, PMID 20581391, PMID 20581392, PMID 20876670, PMID 21270641, PMID 21354816, PMID 21514598, PMID 21694639, PMID 21757630, PMID 21926392, PMID 21926394, PMID 21926395, PMID 21926396, PMID 22105611, PMID 22113127, PMID 22177992, PMID 22184204, PMID 22205355, PMID 22276227, PMID 22328589, PMID 22417271
U54HL096458 (5), 2017 (3), 2018 (3), 2023 (3), 2025 (2), U01DK062401 (2), U01DK082230 (2), 2005 (1), 2014 (1), 2015 (1), 2016 (1), 2019 (1), P30DK048520 (1), P30DK074038 (1), P30DK090868 (1)