neonetal diseases kidney disease Polycystic kidney disease
Conditions
Interventions
Patients should visit the clinics and be willing to receive their study drug
comparator an/or placebo according to the dosing scheme. Furthermore their data
of Medical history and demographic data w
Sponsors
Galagapos NV
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: Main inclusion criteria for the double-blind period of the study: 1. Male and female subject aged 18 to 50 years, inclusive. 2. Documented diagnosis of typical ADPKD, using the Ravine criteria. 3. Rapidly progressive disease, defined as presence of all of the following: -TKV >750 mL, as determined on imaging not older than 5 years before screening. If historical imaging is not available or older than 5 years, imaging can be performed during the screening period according to local clinical practice (i.e. echography, magnetic resonance imaging [MRI]). -Mayo ADPKD Classification Classes 1C to 1E. 4. eGFR at screening between 30-90 mL/min/1.73 m2 for subjects aged 18 to 40 years (inclusive), and between 30-60 mL/min/1.73 m2 for subjects aged 40 to 50 years. 5. Blood pressure
Exclusion criteria
Exclusion criteria: Main exclusion criteria for the double-blind period of the study: 1. Congenital absence of 1 kidney, or subject had a previous nephrectomy or has a transplanted kidney or a transplantation is planned in the foreseeable future. 2. Administration of polycystic kidney disease-modifying agents (e.g. tolvaptan, somatostatin analogues) or interventions (such as cystaspiration or cyst fenestration) within 12 weeks prior to the screening visit and during the screening period. In case tolvaptan is not being administered, this should be because of e.g. non-availability, intolerance, or physician's clinical judgement. 3. Any condition or circumstances that, in the opinion of the investigator, may make a subject unlikely or unable to complete the study or comply with study procedures and requirements (e.g. unable to undergo MRI. For example subject's weight exceeds weight capacity of the MRI, ferromagnetic metal prostheses, aneurysm clips, severe claustrophobia, etc.). Main exclusion criterion for the open-label extension period of the study: 1. Clinically significant abnormalities detected on 12-lead ECG of either rhythm or conduction, QTcF >450 ms, or long QT syndrome.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Mean percent change from baseline of height-adjusted TKV (htTKV). - Frequency and severity of treatment-emergent adverse events (TEAEs), treatment-emergent serious AEs (SAEs), and TEAEs leading to treatment discontinuation. | — |
Secondary
| Measure | Time frame |
|---|---|
| - Mean change from baseline and estimated GFR (eGFR). - Estimated exposure (area under the curve [AUC], maximum plasma concentration [Cmax]), based on population PK analyses of GLPG2737 and its major metabolite M4. | — |
Countries
Netherlands
Outcome results
None listed