Phase 2
N=80
Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD
Autosomal Dominant Polycystic Kidney · ADPKD
Bottom Line
View on ClinicalTrials.gov: NCT03203642 ↗Enrolled (actual)
80
Serious AEs
10.3%
Results posted
Feb 2023
Primary outcome: Primary: Change From Baseline in Height Adjusted Total Kidney Volume (htTKV) at Month 12 — 0.0236; 0.0288 milliliters per meter — p=0.5265
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Tesevatinib (Drug); Placebo (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Kadmon, a Sanofi Company
- Primary completion
- Jan 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Height Adjusted Total Kidney Volume (htTKV) at Month 12 |
0.0236; 0.0288 | 0.5265 |
| PRIMARY Change From Baseline in Height Adjusted Total Kidney Volume at Month 18 |
0.0383; 0.0425 | 0.7076 |
| PRIMARY Change From Baseline in Height Adjusted Total Kidney Volume at Month 24 |
0.0485; 0.0607 | 0.3895 |
| PRIMARY Change From Baseline in Height Adjusted Total Kidney Volume at End of Study |
0.0604; 0.0589 | 0.9093 |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
37; 40 | — |
| SECONDARY Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Months 12, 18, 24 and End of Study |
-6.8032; -2.1780; -7.3626; -3.8950; -9.7307; -6.4070 | — |
Summary
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).
Eligibility Criteria
Inclusion Criteria
- ADPKD diagnosis based on Ravine's criteria.
- Cysts of at least 1 centimeter.
- Estimated glomerular filtration rate greater than or equal to (>=) 25 milliliter per minute per 1.73 square meter (mL/min/1.73 m^2) and less than or equal to ( = 500 milliliters (mL) for participants 18-35 years of age; >= 750 mL for participants 36-49 years of age; >= 900 mL for participants 50-60 years of age.
- The participant had the following laboratory values:
Platelets greater than (>) lower limit of normal (LLN); Hemoglobin > 9 grams per deciliter; Total bilirubin = LLN; Amylase 450 milliseconds.
- History of torsade de pointes, ventricular tachycardia or fibrillation, pathologic sinus bradycardia (< 50 beats per minute), heart block (excluding first-degree block, being PR interval prolongation only), congenital long QT syndrome or new ST segment elevation or depression or new Q wave on electrocardiogram.
- Participants with a history of atrial arrhythmias were discussed with the Medical Monitor.
- Family history of congenital long QT syndrome or unexplained cardiac death.
- Symptomatic heart failure (per New York Heart Association guidelines), unstable angina, myocardial infarction, or cerebrovascular accident within 6 months prior to study entry.
- History of ventricular rhythm disturbances.
- History of cardiac arrhythmias, stroke, or myocardial infarction.
- Has a cardiac pacemaker.
- History of pericardial effusion or presence of pericardial effusion on screening echocardiogram.
- Taking any medication known to inhibit the cytochrome P450 (CYP)3A4 isozyme or any drugs that are CYP3A4 inducers, or any drugs associated with torsade de pointes or known to prolong the QTcF interval, including anti-arrhythmic medications within 2 weeks prior to screening.
- Uncontrolled intercurrent illness that would limit compliance with study requirements.
- Participant was pregnant, planed to become pregnant, or nursing.
- Human immunodeficiency virus positive.
- Hepatitis B or C positive.
- Immunocompromised.
- Documented renal vascular disease resulting in uncontrolled hypertension.
- Previously received an epithelial growth factor receptor (EGFR).
- Allergy or hypersensitivity to components of tesevatinib or placebo or their formulations.
- Been aphakic due to previous cataract surgery or congenital abnormality.
Data sourced from ClinicalTrials.gov (NCT03203642). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.