EUCTR2021-006255-34-DK进行中（未招募）1 期
A PHASE II, MULTICENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO EVALUATE THE PHARMACODYNAMICS, SAFETY, TOLERABILITY, PHARMACOKINETICS, AND EFFICACY OF RO7204239 IN PARTICIPANTS WITH FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 48
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •Age >=18 and <=65 years at the time of signing the Informed Consent Form
- •Genetic confirmation of FSHD1 or FSHD2
- •Clinical findings consistent with FSHD according to the investigator’s clinical judgment
- •Ability to walk unassisted 10 meters (Timed 10-Meter Walk Test [10MWT])
- •Ricci Clinical Severity Scale score =2.5 and = 4
- •Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study
- •Ability and willingness to comply with the study protocol and to complete all study procedures, measurements, and visits
- •For female participants of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception, during treatment with study drug and for 17 months after the final dose of RO7204239
- •For male participants: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods, and agree to refrain from donating sperm, during treatment with study drug and for 4 months after the final dose of RO7204239
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 47
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239
- •Current or previous treatment (or receipt) of anti-myostatin therapies
- •Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer
- •Contraindications to MRI scans, difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI
- •Presence of clinically significant ECG abnormalities from average of triplicate measurement at screening indicating a safety risk for participants
- •Presence of clinically significant cardiovascular disease indicating a safety risk for participants
- •Presence of clinically significant abnormal findings in echocardiography at screening, with the exception of mitral valve prolapse, which does not exclude participants from the study
- •Any major illness within 1 month before screening
- •Ascertained or presumptive hypersensitivity to RO7204239, or to the constituents of its formulation
- •Concurrent disease or a medical condition or abnormality in clinical laboratory tests that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would pose an unacceptable risk to the participant in this study
- •History of malignancy
- •Any clinically relevant history of anaphylactic reaction requiring inotropic support
- •Any abnormal skin conditions, pigmentation, or lesions in the area intended for SC injection (abdomen) and that would prevent visualization of potential injection-site reactions to RO7204239
- •Immobilization, surgical procedures, fracture, or trauma to the upper or lower limbs within 90 days prior to screening or longer, if judged by the investigator that it may affect motor function assessment
- •Any planned surgery that may affect a participant’s motor function assessment, including participants who have had surgery of scapular fixation within the 12 months preceding screening or that are planned during the study
- •Substance abuse within 12 months prior to screening or are at risk of substance abuse per investigator’s judgment
- •Use of the following medications within 90 days prior to enrollment:
- •– Salbutamol or another ß2-adrenergic agonist taken orally
- •– Recombinant human growth hormone
- •– Recombinant human insulin growth factor-1
- •– Testosterone, oxandrolone or other anabolic steroid
- •– Chronic oral or parenteral use of corticosteroids (inhaled corticosteroid use is allowed) unless required to manage injection reactions
- •– Agents anticipated to increase or decrease muscle volume or strength
研究者
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