2023-505313-24-00招募中3 期
A randomized, placebo-controlled, double-blind, multi-center trial to assess efficacy and safety of octreotide subcutaneous depot (CAM2029) in patients with symptomatic polycystic liver disease
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Camurus AB
- 入组人数
- 31
- 试验地点
- 5
- 主要终点
- Change from baseline to Week 53 in height-adjusted total liver volume (htTLV) as determined by magnetic resonance imaging (MRI) volumetry
研究概览
简要总结
To evaluate the treatment effect of CAM2029 compared to placebo on liver volume in patients with polycystic liver disease (PLD).
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Voluntary and valid written informed consent to participate in the trial provided by the patient before any trial related procedures are performed
- •Male or female patient, ≥18 years at screening
- •Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) with htTLV ≥1800 mL/m at screening
- •Presence of at least 1 of the following PLD-related symptoms within 2 weeks before screening: bloating, fullness in abdomen, lack of appetite, feeling full quickly after beginning to eat, acid reflux, nausea, rib cage pain or pressure, pain in side, abdominal pain, back pain, shortness of breath after physical exertion, limited in mobility, concern about abdomen getting larger, dissatisfied by the size of abdomen
- •Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial
- •Female patients of childbearing potential must be willing to use an acceptable method of contraception from screening and during the entire trial
- •Male patients must be willing to use condom as method of contraception from screening and throughout the trial unless they have been sterilized by vasectomy (with an appropriate post-vasectomy documentation of the absence of sperm in the ejaculate)
排除标准
- •Surgical intervention for PLD within 3 months before screening. For sclerotherapy patients, at least 6 months before screening
- •Patients with a known history of hypothyroidism, unless they have been on adequate and stable replacement thyroid hormone therapy for at least 3 months before the first dose of the IMP.
- •Uncontrolled hypertension defined by a systolic blood pressure of >160 mmHg and/or diastolic blood pressure of >100 mmHg at screening
- •History of significant cardiac disease or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the trial, such as uncontrolled or significant cardiac disease, including any of the following: a. History of myocardial infarction, angina pectoris or coronary artery bypass graft within 6 months before screening b. Clinically significant cardiac arrhythmias (e.g. ventricular tachycardia), complete left bundle branch block or high-grade atrioventricular block (e.g. bifascicular block, Mobitz type II and third-degree atrioventricular block) c. Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following: i. Risk factors for Torsades de Pointes including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure or history of clinically significant/symptomatic bradycardia ii. Treatment with concomitant medication(s) with a "Known risk of Torsades de Pointes" per www.crediblemeds.org that cannot be discontinued or replaced by safe alternative medication at least 5 half-lives or 7 days (whichever is longer) before the first dose of IMP iii. Patients with a baseline QTc interval corrected by Fridericia's formula (QTcF) >450 msec for males and >470 msec for females at screening
- •Patients with vascular compromise, including, but not limited to, mesenteric thrombosis, portal hypertension and thrombocytopenia (platelet counts less than 100x109/L)
- •Pregnant, lactating or planning to be pregnant during the trial
- •Clinically significant laboratory abnormalities, which in the opinion of the Investigator may prevent the patient from safely participating in the trial
- •History of solid organ transplantation. Exception for kidney transplant patients
- •Any known allergy, hypersensitivity or intolerance to octreotide or any related drug, or other components of CAM2029, or history of any drug hypersensitivity or intolerance that, in the opinion of the Investigator, would compromise the safety of the patient
- •Contraindications to, or interference with, MRI assessments, as dictated by local hospital regulations
- •Previously treated/randomized in the current clinical trial
- •Treatment with an Somatostatin analogue (SSA) within 3 months before screening
- •Participation in any other clinical trial to test an investigational drug or device within the last 30 days before screening or during the trial
- •Any other contraindicated serious medical condition that, in the Investigator’s opinion, may prevent the patient from safely participating in the trial
- •Any other current or prior medical condition that may interfere with the conduct of the trial or the evaluation of its results in the opinion of the Investigator
- •Unwilling or unable to comply with the requirements of the protocol or in a situation or condition that, in the opinion of the Investigator, may interfere with participation in the trial
- •On the staff, affiliated with, or a family member of the personnel directly involved with this trial
- •Non-responsive to previous treatment of PLD with an SSA as per the Investigator’s assessment
- •Symptomatic cholelithiasis within 3 months before screening or previous medical history of cholelithiasis induced by SSAs unless treated with cholecystectomy
- •Presence of extrahepatic cysts that, in the Investigator’s opinion, may prevent the patient from safely participating in the trial
- •Severe kidney disease, as defined by estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m2
- •Severe liver disease defined as liver cirrhosis of Child-Pugh class C
- •Use of oral estrogen contraceptives or supplementation within 3 months before screening
- •Poorly controlled diabetes (hemoglobin A1c ≥10%) at screening
结局指标
主要结局
Change from baseline to Week 53 in height-adjusted total liver volume (htTLV) as determined by magnetic resonance imaging (MRI) volumetry
Change from baseline to Week 53 in height-adjusted total liver volume (htTLV) as determined by magnetic resonance imaging (MRI) volumetry
次要结局
- Change from baseline to Week 53 in the Polycystic Liver Disease Symptoms (PLD-S) measure score
- Change from baseline in htTLV as determined by MRI volumetry
- Change from baseline in the PLD-S measure score
- Change from baseline in height-adjusted total kidney volume (htTKV) as measured by MRI volumetry
- Change from baseline in estimated glomerular filtration rate (eGFR), assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) cystatin C equation using serum concentrations of creatinine and cystatin C
- Change from baseline in the Polycystic Liver Disease Impact (PLD-I) measure score
- Change from baseline in the Clinical Global Impression of Severity (CGI-S) score
- Change from baseline in the Patient Global Impression of Severity (PGI-S) score
- Change from baseline in the Patient Global Impression of Change (PGI-C) score
- Change from baseline in the Short Form-36 (SF-36) scores
- Change from baseline in the Polycystic Liver Disease Questionnaire (PLD-Q) score
- Incidence of adverse events (AEs)
- Changes from baseline in laboratory values, vital signs and electrocardiogram (ECG) readings
- Octreotide plasma concentrations over time
- Change from baseline in total liver cyst volume determined by MRI volumetry
研究者
Senior VP Research & Development
Scientific
Camurus AB
研究点 (5)
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