A study into the efficacy and toleratability of leuprorelin by patients suffering from severe polycystic liver disease

Phase 1

Conditions: Severe polycystic liver disease in female patients
MedDRA version: 20.1Level: PTClassification code 10048834Term: Polycystic liver diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10036045Term: Polycystic kidneySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10036046Term: Polycystic kidney, autosomal dominantSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 23.0Level: LLTClassification code 10083939Term: Autosomal dominant polycystic liver diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2020-005949-16-NL

Lead Sponsor: niversity Medical Center Groningen

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Female

Target Recruitment: 36

Inclusion Criteria

- Female patients with PLD
- Age 18 to 45 years
- Very large height adjusted liver volume for age: 18-30 yr >2.0 L/m; 30-35 yr >2.2 L/m, 35-40 yr > 2.5 L/m and >40 years >3.0 L/m
- Confirmed ongoing liver growth
- Since somatostatin analogues are proven efficacious therapy for PLD at this time it is required that patients use a somatostatin analogue and still have liver growth (as mentioned above) or the patient has a specific reason not to use this medication (e.g. patient used a somatostatin analogue in the past, but had to stop it due to inefficacy or because they did not tolerate it, or they have a contra-indication for using somatostatin analogues)
- Availability of at least 1 historical MRI or CT scan made between 5 to 1 years before baseline visit
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 36
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Post-menopausal status or (vasomotor) symptoms indicating upcoming menopause;
- AMH measurement at screening < 0.3
- Active desire to have pregancy;
- Contra-indications for leuproreline, such as history of cardiovascular disease, history of osteoporosis or osteoporosis at the dexa-scan at screening;
- Liver transplantation expected in the next 1.5 years
- Use of estrogen or progesterone containing medication