An exploratory, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy, safety, tolerability and pharmacokinetics of orally administered GLPG2737 for 52 weeks, followed by an open-label extension period of 52 weeks in subjects with autosomal dominant polycystic kidney disease (ADPKD).
- Conditions
- neonetal diseaseskidney diseasePolycystic kidney disease1003836010029149
- Registration Number
- NL-OMON55300
- Lead Sponsor
- Galagapos NV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 15
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 <= 150/90 mmHg. In case the subject is treated for
hypertension, he/she should be on a stable treatment regimen of
antihypertensive therapy for at least 8 weeks prior to the screening visit, and
during the screening period.
Main inclusion criteria for the open-label extension period of the study:
1. Male and female subjects who completed the 52-week double-blind treatment
period on IP.
2. Subject, according to the investigator's judgment, may benefit from
long-term treatment with GLPG2737.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>- Mean percent change from baseline of height-adjusted TKV (htTKV).<br /><br>- Frequency and severity of treatment-emergent adverse events<br /><br>(TEAEs), treatment-emergent serious AEs (SAEs), and TEAEs leading to<br /><br>treatment discontinuation.</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Mean change from baseline and estimated GFR (eGFR).<br /><br>- Estimated exposure (area under the curve [AUC], maximum plasma<br /><br>concentration [Cmax]), based on population PK analyses of GLPG2737<br /><br>and its major metabolite M4.</p><br>