NCT04577820撤回3 期
Evaluation of Efficacy and Safety of Garetosmab in Japanese Adult 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
- 撤回
- 主要终点
- Incidence and severity of treatment-emergent adverse event (TEAEs)
研究概览
简要总结
The primary safety objective of the study is to assess the safety and tolerability of garetosmab in Japanese male and female adult patients with FOP.
The primary efficacy objective of the study is to assess the effect of garetosmab on Heterotopic ossification (HO) in Japanese adult patients with FOP, as determined by the number of new heterotopic bone lesions identified by computed tomography (CT).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive HO)
- •Confirmation of FOP diagnosis with documentation of any Type I activin A receptor (ACVR1) mutation
- •FOP disease activity, as defined in the protocol, within 1 year of screening visit
- •Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study
- •Able to understand and complete study-related questionnaires and diaries (assistance from caregivers is allowed)
排除标准
- •Patient 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 mL/min/1.73 m2 calculated by the Modification of Diet in Renal Disease equation (1 retest is allowed)
- •Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) >9% at screening (1 retest allowed)
- •History of severe respiratory compromise, as defined in protocol
- •Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures
- •Pregnant or breastfeeding women
- •NOTE: Other protocol defined inclusion/exclusion criteria apply.
研究组 & 干预措施
garetosmab
Experimental
干预措施: garetosmab (Drug)
结局指标
主要结局
Incidence and severity of treatment-emergent adverse event (TEAEs)
时间窗: Through week 28
Number of new HO lesions as assessed by CT
时间窗: At week 28
次要结局
- Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET(Baseline and week 56)
- Percent of patients with investigator-assessed flare-ups(Baseline to week 56)
- Percent change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET(Baseline and week 56)
- Percent change in total lesion activity by PET(Baseline and week 56)
- Total lesion activity in new HO lesions as assessed by PET(At week 56)
- Percent of patients with flare-ups assessed by patient e-diary(Baseline to week 56)
- Number of new HO lesions as assessed by CT(At week 56)
- Number of new HO lesions as assessed by PET(At week 56)
- Time-weighted average (standardized area under curve [AUC]) change in daily pain due to FOP, as measured using the daily numeric rating scale (NRS)(Baseline through week 28)
- Incidence and severity of TEAEs(Through week 56)
- Percent of patients with new HO lesions as assessed by PET(At week 56)
- Total volume of new HO lesions as assessed by CT(At week 56)
- Number of new HO lesions as assessed by positron emission tomography (PET)(At week 28)
- Percent of patients with new HO lesions as assessed by CT(At week 56)
- Change in number of HO lesions as assessed by PET(Baseline and week 56)
- Time-weighted average (standardized AUC) change in daily pain due to FOP, as measured using the daily NRS(Baseline through week 56)
- Pharmacokinetic (Pk) Profile - concentrations of garetosmab in serum over time(Through week 56)
- Immunogenicity as measured by Anti-drug antibodies (ADA) to garetosmab over time(Through week 28)
- Percent change from baseline in biomarkers of bone formation levels in serum(Through week 28)
- Change in total lesion activity by PET(Baseline and week 56)
- Change in the total volume of HO lesions as assessed by CT(Baseline and week 56)
- Percent change in the total volume of HO lesions as assessed by CT(Baseline and week 56)
- Change in the number of HO lesions detectable by CT(Baseline and week 56)
- Total dosage of glucocorticoids use(Through week 56)
- Concentration of total activin A in serum over time(Through week 56)
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