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Phase 2a Study of VX-407 in Subjects with ADPKD Who Have a Subset of PKD1 Gene Variants
EU CTIS ID: 2024-517393-13-00
What this study is testing
Treatment of ADPKD with VX-407
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Subjects between the ages of 18 and 65 years, inclusive at the time of signing the Screening Period ICF.
- 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).
- Subjects with a pre-existing diagnosis of ADPKD defined as: • Evidence 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: ≥3 cysts in between both kidneys for participants <40 years of age ≥2 cysts in each kidney for participants ≥40 years of age and ≤59 years of age ≥4 cysts in each kidney for participants ≥60 years of age OR • In the absence of a family history of ADPKD: The presence of ≥10 cysts per kidney
- 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.
- 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.
- 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.
You likely can't join if
- 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: • Kidney disease other than ADPKD that in the opinion of the investigator would independently impact the natural history of ADPKD • Solid organ or bone marrow transplantation, or nephrectomy • Any condition possibly affecting drug absorption (e.g., gastrectomy, gastrointestinal tract surgery except appendectomy and cholecystectomy) • Clinically significant liver dysfunction • Cancer, 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) • Stroke or myocardial infarction within 6 months before Day 1 • Hospitalization for heart failure exacerbation within the past year • Risk 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
- Subjects who, in the opinion of the investigator, have evidence of atypical ADPKD presentation (MIC 2).
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Canada. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.