NCT05394116进行中（未招募）3 期
Phase 3 Randomized, Placebo-Controlled Study to Assess Safety, Tolerability, and Efficacy of Garetosmab in Patients With Fibrodysplasia Ossificans Progressiva
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 63
- 试验地点
- 22
- 主要终点
- Number of new HO lesions
研究概览
简要总结
This study is researching an experimental drug called garetosmab. The study is focused on adult patients with fibrodysplasia ossificans progressiva (FOP).
The aim of the study is to see how safe and effective the study drug is in patients with FOP.
The study is looking at several other research questions, including:
- What side effects may happen from receiving the study drug
- How much study drug is in the blood at different times
- Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
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
- •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
排除标准
- •Cumulative Analog Joint Involvement Scale (CAJIS) score at screening >19
- •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:
- •Systolic blood pressure ≥180 mm Hg or diastolic blood pressure ≥110 mm Hg at the screening visit
- •Systolic blood pressure of 160 mm Hg to 179 mm Hg or diastolic blood pressure of 100 mm Hg to 10^9 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
- •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 Inclusion/Exclusion Criteria apply
研究组 & 干预措施
High dose Garetosmab
Experimental
Garetosmab is administered by intravenous (IV) administration every 4 weeks (Q4W)
干预措施: Garetosmab (Drug)
Placebo
Experimental
Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV Q4W.
干预措施: Placebo (Drug)
Low dose Garetosmab
Experimental
Garetosmab is administered by IV administration Q4W
干预措施: Garetosmab (Drug)
结局指标
主要结局
Number of new HO lesions
时间窗: At Week 56
Incidence and severity of treatment-emergent adverse events of special interest (AESIs)
时间窗: Baseline to Week 56
次要结局
- Number of clinician-assessed flare-ups(Through Weeks 28, 56 and 84)
- Occurrence of new HO lesions(At Weeks 28, 56 and 84)
- Total volume of new HO lesions(At Weeks 28, 56 and 84)
- Occurrence of patient-reported flare-ups(Through Weeks 28 and 56)
- Number of new HO lesions(At week 28 and 84)
- Occurrence of clinician-assessed flare-ups(Through Weeks 28, 56 and 84)
- Number of patient-reported flare-ups(Through Weeks 28 and 56)
- Change in joint function assessment by physician using cumulative analog joint involvement scale (CAJIS)(Baseline to Weeks 28 and 56)
- Change in pulmonary function as assessed by spirometry(Baseline at Weeks 28 and 56)
- Change in disease severity as assessed by the Patient Global Impression of Severity (PGIS)(Baseline to Weeks 28 and 56)
- Change in disease severity as assessed by the Patient's Global Impression of Change (PGIC)(At Weeks 28 and 56)
- Change in disease severity as assessed by the Clinician's Global Impression of Change (CGIC)(At Weeks 28 and 56)
- Concentration of total activin A in serum over time(Through Week 56)
- Concentration of garetosmab in serum over time(Through Week 56)
- Incidence of anti-drug antibodies (ADA) to garetosmab over time(Through Week 56)
- Titer of ADA to garetosmab over time(Through Week 56)
研究者
研究点 (22)
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