2023-508350-26-00招募中3 期
Phase 3 Randomized, Placebo-Controlled Study to Assess Safety, Tolerability and Efficacy of Garetosmab in Patients with Fibrodysplasia Ossificans Progressiva
Regeneron Pharmaceuticals Inc.6 个研究点 分布在 6 个国家目标入组 24 人开始时间: 2024年5月13日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 24
- 试验地点
- 6
- 主要终点
- Number of new HO lesions
研究概览
简要总结
The primary efficacy objective of the study is to assess the effect of garetosmab versus placebo on the formation of new HO lesions, as determined by low-dose computerized tomography (CT). The primary safety objective of the study is to assess the safety and tolerability of garetosmab versus placebo.
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
- 否
入选标准
- •Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) [(based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)].
- •Confirmation of FOP diagnosis with documentation of Type I activin A receptor (ACVR1) FOP causing mutation.
- •FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, or other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of HO lesions (increase in size or number of HO lesions) with/without being associated with flare-up episodes.
- •Willing and able to undergo CT imaging procedures and other procedures as defined in the protocol.
- •Note: Other protocol defined Inclusion Criteria apply
排除标准
- •Cumulative Analog Joint Involvement Scale (CAJIS) score at screening >
- •Prior use in the past year and concomitant use of bisphosphonates.
- •Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures (eg, collection of blood or tissue samples).
- •Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer.
- •Pregnant or breastfeeding women.
- •Women of childbearing potential (WOCBP) who are unwilling to practice highly effective contraception, as defined in the protocol.
- •Male patients with WOCBP partners who are not willing to use condoms with WOCBP partners to prevent potential fetal exposure, as defined in the protocol.
- •Note: Other protocol defined Exclusion Criteria apply
- •Participant has significant concomitant illness or history of significant illness such as but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study.
- •Previous history or diagnosis of cancer.
- •Severely impaired renal function defined as estimated glomerular filtration rate <30 milliliter per minute (mL/min) (/1.73 m^2 calculated by the Modification of Diet in Renal Disease equation.
- •Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) >9% at screening.
- •History of poorly controlled hypertension, as defined by: a. Systolic blood pressure ≥180 mm Hg or diastolic blood pressure ≥110 mm Hg at the screening visit b. Systolic blood pressure of 160 mm Hg to 179 mm Hg or diastolic blood pressure of 100 mm Hg to 109 mm Hg at the screening visit, AND a history of end-organ damage (including history of left-ventricular hypertrophy, heart failure, angina, myocardial infarction, stroke, transient ischemic attack, peripheral arterial disease, end-stage renal disease, and moderate-to-advanced retinopathy.
- •Known history of cerebral vascular malformation.
- •Cardiovascular conditions such as New York Heart Association class III or IV heart failure, cardiomyopathy, intermittent claudication, myocardial infarction, or acute coronary syndrome within 6 months prior to screening; symptomatic ventricular cardiac arrhythmia.
- •History of severe respiratory compromise requiring oxygen, respiratory support (eg, bilevel positive airway pressure [biPAP] or continuous positive airway pressure [CPAP]), or a history of aspiration pneumonia requiring hospitalization.
结局指标
主要结局
Number of new HO lesions
Number of new HO lesions
Incidence and severity of treatment-emergent adverse events of special interest (AESIs)
Incidence and severity of treatment-emergent adverse events of special interest (AESIs)
次要结局
- Number of clinician-assessed flare-ups.
- Occurrence of new HO lesions
- Total volume of new HO lesions
- Occurrence of patient-reported flare-ups
- Number of new HO lesions
- Occurrence of clinician-assessed flare ups
- Number of patient reported flare-ups
- Change in joint function assessment by physician using cumulative analog joint involvement scale (CAJIS)
- Change in pulmonary function as assessed by spirometry
- Change in disease severity as assessed by the Patient Global Impression of Severity (PGIS)
- Change in disease severity as assessed by the Patient’s Global Impression of Change (PGIC).
- Change in disease severity as assessed by the Clinician’s Global Impression of Change (CGIC).
- Concentration of total activin A in serum over time.
- Concentrations of garetosmab in serum over time.
- Incidence of anti-drug antibodies (ADA) to garetosmab over time.
- Titer of ADA to garetosmab over time.
研究者
Medical Affairs
Scientific
Regeneron Pharmaceuticals Inc.
研究点 (6)
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