A Phase 3, Randomized, Active Controlled Study to Evaluate Maintenance of Response, Safety and Patient Reported Outcomes in Acromegaly Patients Treated With Octreotide Capsules vs. Parenteral Somatostatin Receptor Ligands
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 146
- 试验地点
- 53
- 主要终点
- Proportion of Patients Who Are Biochemically Controlled Throughout the RCT Phase
研究概览
简要总结
Octreotide capsule is a novel, orally-administered formulation of the commercially-available injectable drug octreotide. In a recent phase 3 trial (OPTMAL; NCT03252353), oral octreotide capsules demonstrated sustained biochemical response up to 13 months in patients with acromegaly previously managed with somatostatin analog injections (ref).
The objective of this study was to compare the efficacy, safety, and patient reported outcomes (PROs) between oral octreotide capsules and injectable somatostatin receptor ligands (SRLs).
详细描述
This was phase 3, randomized, open-label, active controlled, multicenter study to evaluate the maintenance of response, safety and patient reported outcomes (PROs) in acromegaly patients treated with octreotide capsules and in patients treated with standard of care parenteral somatostatin receptor ligands (SRLs), who previously tolerated and demonstrated biochemical control on both treatments.
The core study consisted of three phases: a Screening phase, Run-in phase and a Randomized Controlled Treatment (RCT) phase.
Eligible patients who were biochemically controlled on parenteral SRLs were switched to octreotide capsules for a 26-week period Run-in phase. During this phase the effective dose for each patient was determined through dose titration.
Patients whose acromegaly has been controlled biochemically on octreotide capsules at the end of the Run-in phase entered a 36-week open-label RCT phase, where they randomized to continue on octreotide capsules or switch back to their injectable SRL treatment (as received prior to Screening).
Following the completion of the core study (Screening, Run-in and RCT phases), eligible patients were offered to enter the Study Extension phase and receive octreotide capsules until product marketing or study termination.
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
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of acromegaly
- •Treatment with Somatostatin analogs injections (octreotide or lanreotide) for at least 6 months
- •Biochemical control (IGF -1 < 1.3 x ULN and GH < 2.5ng/mL)
排除标准
- •Injections of long-acting somatostatin analogs, at a dosing interval > 8 weeks.
- •Pituitary radiotherapy within 5 years
- •Pituitary surgery within six months
- •Patients who previously participated in CH-ACM-01 study
- •Any clinically significant uncontrolled concomitant disease
- •Symptomatic cholelithiasis
- •Previous treatment with:
- •Pegvisomant, within 12 weeks
- •Dopamine agonists, within 6 weeks
- •Pasireotide, within 12 weeks
研究组 & 干预措施
Run-in phase
Oral octreotide capsules
干预措施: Octreotide capsules (Drug)
RCT phase - Oral
Oral octreotide capsules
干预措施: Octreotide capsules (Drug)
RCT phase - Injectables
Injectable somatostatin analogs (octreotide or lanreotide)
干预措施: Octreotide capsules (Drug)
Combination phase (sub-study)
Octreotide capsules plus cabergoline
干预措施: Octreotide capsules (Drug)
结局指标
主要结局
Proportion of Patients Who Are Biochemically Controlled Throughout the RCT Phase
时间窗: 62 weeks
Proportion of patients who are biochemically controlled throughout the RCT phase. A patient was considered biochemically controlled if IGF-1 Time Weighted Average (TWA) during the RCT phase is \<1.3 ULN
次要结局
- Proportion of Patients With Clinical and Biochemical Control at the End of the RCT Phase(Week 62/ End of treatment; EOT)
- Proportion of Patients Who Maintain or Reduce the Overall Number of Active Acromegaly Symptoms at the End of the RCT Phase(62 weeks)
- Proportion of Patients Who Maintain or Improve Their Overall Acromegaly Index of Severity (AIS) Score at the End of the RCT Phase(62 weeks)
- Proportion of Patients of Those Completing the RCT Phase Who Entered the Study Extension Phase(62 weeks)
- Change in IGF-1 Levels in the RCT Phase(Change from Week 26 to week 62)
- Change in GH Levels in the RCT Phase(Change from Week 26 to week 62)