Clinical trial • Phase II • Nephrology|Rare Disease
VX-407 for Autosomal dominant polycystic kidney disease (ADPKD)
Phase II trial of VX-407 for Autosomal dominant polycystic kidney disease (ADPKD). open-label. 13 participants.
Overview
- Trial Therapeutic Area
- Nephrology|Rare Disease
- Trial Disease
- Autosomal dominant polycystic kidney disease (ADPKD)
- Trial Stage
- Phase II
- Drug Modality
- Small molecule
Key dates
- Initial CTIS Submission Date
- 08-08-2025
- First CTIS Authorization Date
- 28-11-2025
Trial design
open-label Phase II trial in Italy, Netherlands, Spain and others.
- Open Label
- Yes
- Target Sample Size
- 13
- Trial Duration For Participant
- 364
Eligibility
Recruits 13 The trial does not select vulnerable populations (isVulnerablePopulationSelected: false). Participants are adults (18–65). Informed consent is obtained via adult subject information and informed consent forms (L1_SIS and ICF Main Adult and related ICF documents). No assent/child consent procedures are described in the available documentation..
- Vulnerable Population
- The trial does not select vulnerable populations (isVulnerablePopulationSelected: false). Participants are adults (18–65). Informed consent is obtained via adult subject information and informed consent forms (L1_SIS and ICF Main Adult and related ICF documents). No assent/child consent procedures are described in the available documentation.
Inclusion criteria
- {"criterion_text":"- Subjects between the ages of 18 and 65 years, inclusive at the time of signing the Screening Period ICF.\n- A body mass index (BMI) of 18 to 45 kg/m2, inclusive (Note: this may be adjusted based on the available data from Study 001).\n- Subjects with a pre-existing diagnosis of ADPKD defined as: •\tEvidence of polycystic kidney disease in 1 or both biological parents AND meeting the Pei-Ravine classification criteria14 as defined below The minimum number of cysts present for each age category as follows: \t≥3 cysts in between both kidneys for participants <40 years of age \t≥2 cysts in each kidney for participants ≥40 years of age and ≤59 years of age \t≥4 cysts in each kidney for participants ≥60 years of age OR •\tIn the absence of a family history of ADPKD: The presence of ≥10 cysts per kidney\n- Subjects with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E15 confirmed by abdominal MRI obtained during screening. A maximum of 5 subjects with MIC status of 1B who have htTKV ≥250 mL/m may be enrolled.\n- Subject has a non-truncating variant that is pathogenic, likely pathogenic, or variant of uncertain significance (VUS) within a sub-region of PKD1 and no additional truncating PKD1 variants confirmed by results obtained with a Vertex-designated investigational clinical study assay.\n- eGFR ≥25 ml/min/1.73 m2 based on the Modified Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) (2021) creatinine equation without the race adjustment."}
Exclusion criteria
- {"criterion_text":"- History of any illness or any clinical condition that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to: •\tKidney disease other than ADPKD that in the opinion of the investigator would independently impact the natural history of ADPKD •\tSolid organ or bone marrow transplantation, or nephrectomy •\tAny condition possibly affecting drug absorption (e.g., gastrectomy, gastrointestinal tract surgery except appendectomy and cholecystectomy) •\tClinically significant liver dysfunction •\tCancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years) •\tStroke or myocardial infarction within 6 months before Day 1 •\tHospitalization for heart failure exacerbation within the past year •\tRisk factors for Torsades de Pointes (e.g., familial long QT syndrome, chronic hypokalemia, heart failure) or concomitant medications that prolong QT/QTc interval or history of any cardiac disorders that, in the opinion of the investigator, might put the subject at risk or may confound the results of the study"}
- {"criterion_text":"- Subjects who, in the opinion of the investigator, have evidence of atypical ADPKD presentation (MIC 2)."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Proportion of subjects with percent change from baseline in htTKV ≤0 on MRI over time","definition_or_measurement_approach":"Percent change from baseline in height-adjusted total kidney volume (htTKV) measured by MRI over time; endpoint expressed as proportion of subjects with percent change ≤ 0 on MRI over time."}
Secondary endpoints
- {"endpoint_text":"- Percent change from baseline in htTKV on MRI over time","definition_or_measurement_approach":"Percent change in htTKV measured by MRI over time compared with baseline."}
- {"endpoint_text":"- Safety and tolerability of VX¬407 based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, coagulation), standard 12-lead ECGs, and vital signs","definition_or_measurement_approach":"Safety assessed by recording adverse events, clinical laboratory parameters (hematology, serum chemistry, coagulation), standard 12-lead ECGs, and vital signs."}
- {"endpoint_text":"- Plasma PK parameters of VX 407","definition_or_measurement_approach":"Plasma pharmacokinetic parameters of VX-407 measured from plasma samples (PK sampling and analysis as per protocol)."}
Recruitment
- Planned Sample Size
- 13
- Recruitment Window Months
- 20
- Consent Approach
- Informed consent is obtained from adult participants using subject information sheets and ICF documents (L1_SIS and ICF Main Adult, Genotype Determination Visit ICF, Pregnancy ICF and privacy annexes). ICF and recruitment materials are provided in country- and language-specific versions (documents available in Italian, Dutch, Spanish, French, German and English as indicated by document titles). As participants are adults (18–65), consent is by the participant; no assent/child consent procedures are described.
Methods
- Country-specific recruitment arrangements documents (K1) outlining local recruitment plans (documents available for IT, NL, ES, BE, FR, DE).
- Study flyers and site posters (K2_Recruitment material_Study Flyer, Site Poster) in country/language-specific versions targeting potential participants at sites.
- Physician brochures and physician letters (K2_Recruitment material_Physician Brochure, Physician Letter) distributed to referring clinicians to identify eligible patients.
- Patient letters, new participant brochures, and returning participant brochures for site-based participant outreach and engagement.
- Congress cards and study flyers for outreach at conferences and professional meetings.
- Educational material about genotyping (K2_Recruitment material_Understanding Genotyping) to inform potential participants about genotype-based eligibility and the investigational genotyping assay.
- Informed consent flipbooks and ICF materials to support consent discussions at sites (language-specific versions).
Geography
- Total Number Of Sites
- 12
- Total Number Of Participants
- 11
Italy
- Earliest CTIS Part Ii Submission Date
- 04-11-2025
- Latest Decision Or Authorization Date
- 01-12-2025
- Processing Time Days
- 27
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Nephrology
- Principal Investigator Name
- Giuseppe Grandaliano
- Principal Investigator Email
- giuseppe.grandaliano@unicatt.it
- Contact Person Name
- Giuseppe Grandaliano
- Contact Person Email
- giuseppe.grandaliano@unicatt.it
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-11-2025
- Latest Decision Or Authorization Date
- 01-12-2025
- Processing Time Days
- 28
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Nephrology
- Principal Investigator Name
- Madhi Salih
- Principal Investigator Email
- m.salih@erasmusmc.nl
- Contact Person Name
- Madhi Salih
- Contact Person Email
- m.salih@erasmusmc.nl
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- Nephrology
- Principal Investigator Name
- Ronald Gansevoort
- Principal Investigator Email
- r.t.gansevoort@umcg.nl
- Contact Person Name
- Ronald Gansevoort
- Contact Person Email
- r.t.gansevoort@umcg.nl
Spain
- Earliest CTIS Part Ii Submission Date
- 04-11-2025
- Latest Decision Or Authorization Date
- 03-12-2025
- Processing Time Days
- 29
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Hospital Universitario Fundacion Jimenez Diaz
- Department Name
- Nefrología e Hipertensión
- Principal Investigator Name
- María Vanessa Pérez Gómez
- Principal Investigator Email
- MVANESSA@fjd.es
- Contact Person Name
- María Vanessa Pérez Gómez
- Contact Person Email
- MVANESSA@fjd.es
- Site Name
- Fundacio Puigvert
- Department Name
- Nefrología
- Principal Investigator Name
- Mónica María Furlano
- Principal Investigator Email
- mfurlano@fundacio-puigvert.es
- Contact Person Name
- Mónica María Furlano
- Contact Person Email
- mfurlano@fundacio-puigvert.es
Belgium
- Earliest CTIS Part Ii Submission Date
- 30-10-2025
- Latest Decision Or Authorization Date
- 02-12-2025
- Processing Time Days
- 33
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Cliniques Universitaires Saint-Luc
- Department Name
- Nephrology
- Principal Investigator Name
- Nathalie Demoulin
- Principal Investigator Email
- nathalie.demoulin@saintluc.uclouvain.be
- Contact Person Name
- Nathalie Demoulin
- Contact Person Email
- nathalie.demoulin@saintluc.uclouvain.be
- Site Name
- UZ Leuven
- Department Name
- Nephrology
- Principal Investigator Name
- Bert Bammens
- Principal Investigator Email
- Bert.bammens@uzleuven.be
- Contact Person Name
- Bert Bammens
- Contact Person Email
- Bert.bammens@uzleuven.be
France
- Earliest CTIS Part Ii Submission Date
- 13-11-2025
- Latest Decision Or Authorization Date
- 28-11-2025
- Processing Time Days
- 15
- Number Of Sites
- 3
- Number Of Participants
- 2
Sites
- Site Name
- Centre Hospitalier Regional Universitaire De Tours
- Department Name
- Service de Néphrologie-Hypertension artérielle- Dialyse-Transplantation rénale
- Principal Investigator Name
- Jean-Michel Halimi
- Principal Investigator Email
- jean-michel.halimi@univ-tours.fr
- Contact Person Name
- Jean-Michel Halimi
- Contact Person Email
- jean-michel.halimi@univ-tours.fr
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Service de Néphrologie-Dialyse Adultes
- Principal Investigator Name
- Bertrand Knebelmann
- Principal Investigator Email
- bertrand.knebelmann@aphp.fr
- Contact Person Name
- Bertrand Knebelmann
- Contact Person Email
- bertrand.knebelmann@aphp.fr
- Site Name
- Centre Hospitalier Regional Et Universitaire De Brest
- Department Name
- Department of Nephrology, Renal Transplant and Hemodialysis
- Principal Investigator Name
- Emilie Cornec-Le Gall
- Principal Investigator Email
- emilie.cornec-legall@chu-brest.fr
- Contact Person Name
- Emilie Cornec-Le Gall
- Contact Person Email
- emilie.cornec-legall@chu-brest.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 07-10-2025
- Latest Decision Or Authorization Date
- 01-12-2025
- Processing Time Days
- 55
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Nephrology and Medical Intensive Care
- Principal Investigator Name
- Jan Halbritter
- Principal Investigator Email
- jan.halbritter@charite.de
- Contact Person Name
- Jan Halbritter
- Contact Person Email
- jan.halbritter@charite.de
- Site Name
- University Hospital Cologne AöR
- Department Name
- Nephrology, Rheumatology, Endocrinology and General Medicine
- Principal Investigator Name
- Roman-Ulrich Mueller
- Principal Investigator Email
- roman-ulrich.mueller@uk-koeln.de
- Contact Person Name
- Roman-Ulrich Mueller
- Contact Person Email
- roman-ulrich.mueller@uk-koeln.de
Sponsor
Primary sponsor
- Full Name
- Vertex Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Investigational products
- Investigational Product Name
- VX-407 tablet
- Active Substance
- VX-407
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
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