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
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- Camurus AB
- 入组人数
- 71
- 试验地点
- 22
- 主要终点
- Height-adjusted total liver volume (htTLV)
研究概览
简要总结
The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD (ADPLD) or associated with autosomal dominant polycystic kidney disease (ADPKD).
In the Treatment Period of the trial, participants will be allocated at random to 1 of the 3 treatment arms in a 1:1:1 ratio. After completing the Treatment Period (53 weeks) participants may proceed to a 120-week open-label extension part of the trial and then only receive the same CAM2029 treatment.
The active ingredient in CAM2029, octreotide, is administered as a subcutaneous depot using Camurus' FluidCrystal® technology.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patient, ≥18 years at screening
- •Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) as defined by 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
排除标准
- •Surgical intervention for PLD within 3 months before screening
- •Treatment with a somatostatin analogue (SSA) within 3 months before screening
- •Non-responsive to previous treatment of PLD with an SSA as per the Investigator's assessment
- •Systematic 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 eGFR <30 mL/min/1.73^m2
- •Severe liver disease defined as liver cirrhosis of Child-Pugh class C
- •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
研究组 & 干预措施
CAM2029 once every 2 weeks
0.5 mL CAM2029 10 mg, SC injection, every 2 weeks and 0.5 mL placebo, SC injection, once every 2 weeks (alternating with CAM2029 dosing)
干预措施: Placebo (Drug)
Placebo
0.5 mL placebo, SC injection, once weekly
干预措施: Placebo (Drug)
CAM2029 once weekly
0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly
干预措施: CAM2029 (Drug)
CAM2029 once every 2 weeks
0.5 mL CAM2029 10 mg, SC injection, every 2 weeks and 0.5 mL placebo, SC injection, once every 2 weeks (alternating with CAM2029 dosing)
干预措施: CAM2029 (Drug)
结局指标
主要结局
Height-adjusted total liver volume (htTLV)
时间窗: From screening until treatment week 53
Change from baseline to Week 53 in htTLV as determined by MRI volumetry
次要结局
- Patient Global Impression of Severity (PGI-S) score(From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173)
- Height-adjusted total kidney volume (htTKV)(From screening until treatment weeks 13, 25, 53, 77, 125 and 173)
- PLD symptom (PLD-S) score(From screening to week 53)
- Polycystic Liver Disease Questionnaire (PLD-Q)(From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173)
- PLD-S(From screening to weeks 13, 21, 25, 39, 77, 101, 125, 149 and 173)
- Total liver cyst volume(From screening to treatment weeks 13, 25, 53, 77, 125 and 173)
- PLD impact (PLD-I) score(From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173)
- Patient Global Impression of Change (PGI-C) score(At treatment weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173)
- Estimated glomerular filtration rate (eGFR)(From treatment week 1 to weeks 13, 25, 53, 65, 77, 101, 125, 149 and 173)
- Short Form-36 (SF-36) score(From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173)
- htTLV(From screening until treatment weeks 13, 25, 77, 125 and 173)
- Clinical Global Impression of Severity (CGI-S) score(From treatment week 1 to weeks 13, 21, 25, 53, 77, 101, 125, 149 and 173)
- Adverse events (AEs)(From screening to the safety follow-up, assessed up to approximately 43 months)