A study to evaluate the effects of GLPG2737 in subjects with autosomal dominant polycystic kidney disease

Phase 1

• Conditions: Autosomal dominant polycystic kidney disease; MedDRA version: 20.0Level: LLTClassification code 10036046Term: Polycystic kidney, autosomal dominantSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-003521-21-DE
• Lead Sponsor: Galapagos NV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-03-10
• Last Update Posted: 24 January 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 60

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 = 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.

Reference is made to the protocol for a complete overview of the inclusion criteria.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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 cyst aspiration 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 judgment.
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.

Reference is made to the protocol for a complete overview of the exclusion criteria.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified