BridgeMD
The AGAINST-PLD Study; An investigator-driven, randomized, open label clinical trial assessing efficacy of leuprorelin to halt disease progression in PLD
EU CTIS ID: 2023-506637-30-00
What this study is testing
To determine whether lowering estrogen and progesterone levels with the GnRH agonist leuprorelin decreases liver growth rates (in percent per year) in pre-menopausal women with severe polycystic liver disease during 18 months of treatment, comparing the direct start group (treated) and delayed start group (untreated).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Female patients
- 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) o 18-30 yr; height adjusted TLV > 2.0 L/m o 30-35 yr; height adjusted TLV > 2.2 L/m o 35-40 yr; height adjusted TLV > 2.5 L/m o 40-45 years; height adjusted TLV > 3.0 L/m OR Age independent hTLV > 2.0 L/m and PLD-Q score >30.
- With regard to the use of somatostatin analogues: o patients use a somatostatin analogue and still have confirmed liver growth; OR o patient have a reason not to use this medication, .e.g. patient used a somatostatin analogue in the past but had to stop due to inefficacy or because he/she did not tolerate it, patient has a contra-indication for using somatostatin analogues, patient or treating physician chose not to try 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.
- Diagnosis of polycystic liver disease defined as the presence of more than 10 liver cysts
You likely can't join if
- Post-menopausal status or (vasomotor) symptoms indicating upcoming menopause
- 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.
- 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), unexplained vaginal bleeding, or a known intolerance for leuprorelin
See the full eligibility criteria
Who can join
- Female patients
- 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) o 18-30 yr; height adjusted TLV > 2.0 L/m o 30-35 yr; height adjusted TLV > 2.2 L/m o 35-40 yr; height adjusted TLV > 2.5 L/m o 40-45 years; height adjusted TLV > 3.0 L/m OR Age independent hTLV > 2.0 L/m and PLD-Q score >30.
- With regard to the use of somatostatin analogues: o patients use a somatostatin analogue and still have confirmed liver growth; OR o patient have a reason not to use this medication, .e.g. patient used a somatostatin analogue in the past but had to stop due to inefficacy or because he/she did not tolerate it, patient has a contra-indication for using somatostatin analogues, patient or treating physician chose not to try 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.
- Diagnosis of polycystic liver disease defined as the presence of more than 10 liver cysts
What rules you out
- Post-menopausal status or (vasomotor) symptoms indicating upcoming menopause
- 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.
- 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), unexplained vaginal bleeding, or a known intolerance for leuprorelin
- Liver transplantation or liver surgery expected within 1.5 years, to the discretion of the study doctor
- Use of hormonal oral contra-conception containing estrogen and/or progesterone. In contrast, a hormone containing uterine device is not an exclusion criteria.
- Contra-indications for both MRI and CT assessments (such as implants) or not able or willing to undergo MRI and CT 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
- Therapy resistant severe hypertension, defined as a systolic blood pressure >160 mmHg and/or diastolic blood pressure > 100 mm Hg.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling female, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.