NL-OMON49538招募中2 期
An Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with Extension - Open-Label study of Losmapimod for FSHD1
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 14
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 18 至 64（—）
入选标准
- •1.FSHD1 subjects age 18-65 years., 2.Subject will sign and date an informed
- •consent form (ICF)., 3.3. Subjects will have a confirmed diagnosis of FSHD1
- •with 1 to 9 repeats via assessment of the size of the D4Z4 array on chromosome
- •4 using the calculator provided by the sponsor. Genetic confirmation must be
- •obtained prior to the screening MRI and baseline muscle biopsy; genetic
- •confirmation can come from previous testing if verified with appropriate
- •documentation. Due to stable transmission of repeat sizes within families,
- •subjects with a clinical diagnosis of FSHD who have a first degree relative
- •with a genetically confirmed diagnosis of FSHD1 may be entered into the study
- •for screening and MRI. During screening, a confirmatory genetic diagnosis is
- •conducted. If genetic testing during screening is necessary, the 4-week
- •screening window will not start until the results are obtained and verified by
- •the principal investigator. , 4.Subject will be willing and able to comply with
- •scheduled visits, treatment plan, study restrictions, laboratory tests,
- •contraceptive guidelines, scheduled needle muscle biopsies, and other study
- •procedures., 5.Male or female subjects:, a.A female subject is eligible to
- •participate if she is of non-childbearing potential defined as pre-menopausal
- •females with a documented tubal ligation or hysterectomy; or postmenopausal
- •defined as 12 months of spontaneous amenorrhoea or if of childbearing potential
- •is using a highly effective method for avoidance of pregnancy (refer to Section
- •5.5) for the duration of the clinical trial and until 90 days following the
- •last dose. The decision to include or exclude women of childbearing potential
- •may be made at the discretion of the investigator and in accordance with local
- •practice in relation to adequate contraception., b.Male subjects must agree to
- •use one of the contraception methods listed in Section 5.5. This criterion must
- •be followed from the time of the first dose of study medication until 90 days
- •after the last study drug dose., 6. Subject has a Clinical Severity Score
- •between 2 and 4 on Ricci*s scale (scale range is from 0 to 5). Patients that
- •use a wheelchair or walker for any activity are not permitted to enroll in the
- •study., 7. Subject commitment to complete the 2 visits for skeletal muscle
- •needle biopsy and all visits for whole body MRI., 8. Subject is able to
- •complete the RWS, TUG, and FSHD PROs (FSHD-RODS and FSHD HI) at the screening
- •visit., 9. Subject has an MRI-eligible muscle for biopsy as determined by the
- •central reader.
排除标准
- •1. Subject has a history of any illness or any clinical condition that, in the
- •opinion of the investigator, might confound the results of the study or pose an
- •additional risk in administering study drug to the subject. This may include,
- •but is not limited to, history of relevant drug or food allergies; history of
- •cardiovascular or central nervous system disease; history or presence of
- •clinically significant pathology; clinically significant history of mental
- •disease; and history of cancer, except for squamous cell skin cancer, basal
- •cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no
- •recurrence for the last 5 years)., 2. Subject has a known or clinically
- •suspected infection with human immunodeficiency virus or hepatitis B or C
- •viruses., 3. Subject has current clinically significant liver or kidney
- •dysfunction., 4. Subject screens positive for hepatitis B surface antigen,
- •hepatitis C virus (HCV) antibody, or antibodies against human immunodeficiency
- •viruses 1 and 2 (HIV 1/HIV 2 antibodies)., 5. Subject has any condition
- •possibly affecting drug absorption (eg, gastrectomy, cholecystectomy, or other
- •gastrointestinal tract surgery, except appendectomy)., 6. Subject has a
- •standard 12-lead ECG demonstrating QT interval by Fredericia (QTcF) >450 msec
- •for male subjects and QTcF >470 msec for female subjects at Screening. If QTcF
- •exceeds 450 msec for males or 470 msec for females, the ECG will be repeated 2
- •more times, and the average of the 3 QTcF values will be used to determine the
- •subject*s eligibility., 7. Subject has a history of cardiac dysrhythmias
- •requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs
- •that, in the opinion of the investigator or Medical Monitor, would preclude the
- •subject*s participation in the study., 8. Male subject has a female partner who
- •is planning to become pregnant during the study or within 90 days after the
- •last study drug dose., 9. Subject has donated blood (of approximately 1 pint
- •[500 mL] or more) or has had any significant loss of blood within 90 days
- •before the first study drug dose, as determined by the investigator., 10.
- •Vaccination with a live attenuated vaccine within 6 weeks of randomization.,
- •11. Subject has a history of alcohol, analgesic/opioid, and/or illicit drug
- •abuse as defined by the American Psychiatric Association Diagnostic and
- •Statistical Manual of Mental Disorders, Fifth Edition, in the last 6 months
- •before screening, or a positive test for drugs of abuse at screening.
- •12. Subject has participated in a clinical trial in which they have received an
- •investigational product within the following time period prior to enrolment in
- •the current study: 30 days, 5 half-lives or twice the duration of the
- •biological effect of the investigational product (whichever was longer)., 13.
- •For subjects that are on drug(s) or supplements that may affect muscle function
- •as determined by the treating physician or included in the list of drugs
- •presented in Appendix 15.1: subjects must be on a stable dose of that drug(s)
- •or supplement for at least 3 months prior to enrolment in the study and remain
- •on that stable dose for the duration of the study (list of drugs presented in
- •Appendix 15.1). Changes to the dose or treatment discontinuation during the
- •study can only be done for strict medical reasons
研究者
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