A GnRH Agonist IN Pre-menopausal Women STudy to Treat Severe Polycystic Liver Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-06-01

Study Completion Date

2027-10-01

Brief Summary

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Multicenter trial on the effect of the GnRH analogue leuprorelin on the growth of total liver volume in pre-menopausal women with very severe polycystic liver disease who, despite available therapy, experience growth and are heading for liver transplantation.

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Detailed Description

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Conditions

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Polycystic Liver Disease Autosomal Dominant Polycystic Kidney

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomized, controlled trial in which patients are randomized between either 1. Direct start with treatment or 2. Delayed start with treatment which will start after 18 months of standard care with the study medication

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Patient blinding is not possible, since leuprorelin will induce menopause related complaints.

Primary outcome is not reported to investigators until the study has finished. Outcome assessors are blinded for study subject and treatment allocation.

Study Groups

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Direct start

36 months of treatment with study medication

Group Type ACTIVE_COMPARATOR

Leuprorelin

Intervention Type DRUG

Treatment consist of leuprorelin 3.75 mg once monthly for the first 3 months followed by 3-monthly injections of 11.25 mg. The direct start group will use leuprorelin for 36 months. The delayed start group will use standard of care in the first 18 months.

Delayed start

First 18 months standard care, hereafter 18 months treatment with study medication

Group Type OTHER

Leuprorelin

Intervention Type DRUG

Treatment consist of leuprorelin 3.75 mg once monthly for the first 3 months followed by 3-monthly injections of 11.25 mg. The direct start group will use leuprorelin for 36 months. The delayed start group will use standard of care in the first 18 months.

Interventions

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Leuprorelin

Treatment consist of leuprorelin 3.75 mg once monthly for the first 3 months followed by 3-monthly injections of 11.25 mg. The direct start group will use leuprorelin for 36 months. The delayed start group will use standard of care in the first 18 months.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Female patients
* Diagnosis of polycystic liver disease defined as the presence of more than 10 liver cysts
* Age between 18 to 45 (inclusive) years;
* Very large liver for age, defined as the upper 10% of liver volumes in specific age categories (based on a retrospective polycystic liver disease registry, n=1.600 patients)

* 18-30 yr; height adjusted TLV \> 2.0 L/m
* 30-35 yr; height adjusted TLV \> 2.2 L/m
* 35-40 yr; height adjusted TLV \> 2.5 L/m
* 40-45 years; height adjusted TLV \> 3.0 L/m
* Availability of at least 1 historical MRI or CT scan made between 5 to 1 years before baseline visit of this study
* Ongoing liver growth, defined as an increase in absolute total liver volume between the historical MRI or CT scan and the MRI at screening of this trial
* Since somatostatin analogues are proven efficacious therapy for polycystic liver disease at this time it is required that:

* patients use a somatostatin analogue and still have confirmed liver growth; OR
* patient have 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 he/she did not tolerate it, patient has a contra-indication for using somatostatin analogues, no availability of somatostatin analogues
* Voluntary written informed consent before performance of any study-related procedures not part of standard medical care, and able to read, comprehend, and respond to study questionnaires.

Exclusion Criteria

Post-menopausal status or (vasomotor) symptoms indicating upcoming menopause

* Anti Mullerian Hormone (AMH) measurement at screening visit \<0.03 ng/ml.
* Active desire to have children, pregnancy or breast-feeding
* Contra-indications for leuprorelin, such as history of cardiovascular disease, history of osteoporosis or osteoporosis determined by DEXA-scan at screening (T score ≤ - 2.5), or a known intolerance for leuprorelin
* Liver transplantation or liver surgery expected within 1.5 years, to the discretion of the study doctor
* Contra-indications for MRI assessments (such as implants) or not able or willing to undergo MRI scan for other reasons (e.g. claustrophobia, profound obesity)
* Kidney transplantation or chronic use of immunosuppressive agents (such as cyclosporine, mycophenolic acid, tacrolimus but not prednisolone) for other indications
* Severe hypertension, defined as a systolic blood pressure \>160 mmHg and/or diastolic blood pressure \> 100 mm Hg.
* Clinically significant, uncontrolled medical condition that, in the opinion of the investigator, would put the safety of the patient at risk through participation, or which would affect the efficacy of safety analysis if the condition exacerbated during the study, or that may significantly interfere with study compliance, such as, but not restricted to, recurrent cholangitis, recurrent ascites or hepato-venous outflow obstruction, (history of) depression
* Participation in other interventional studies at the same time.

Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No