EUCTR2022-000389-16-DK进行中（未招募）1 期
A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients 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
- 260
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
- •1. Patient will sign and date an informed consent form (ICF).
- •2. Patients will have a diagnosis of FSHD1 or FSHD2 verified by genetic testing
- •- Randomization will be stratified for FSHD1 to ensure that treatment allocation is balanced across FSHD repeat number categories (ie, 1 to 3 repeats versus 4 to 9 repeats).
- •- Randomization will be stratified for FSHD2 to ensure that an equal number of patients will be allocated to treatment and placebo.
- •3. Patients will have a Clinical Severity Score of 2 to 4 (Ricci score; range 0 to 5) at screening. Patients who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study.
- •4. Patients with screening total RSA (Q1-Q4) without weight in the dominant UE assessed by RWS = 0.2 and = 0.7.
- •5. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
- •6. No contraindications to MRI.
- •7. Patients (male and female) will be between the ages of 18 and 65 years at the time of consent, inclusive.
- •- A female patient is eligible to participate if she is of non-child bearing potential, defined as pre-menopausal females with a documented tubal ligation or hysterectomy; bilateral salpingectomy, or bilateral oophorectomy; if she is postmenopausal, defined as no menses for 12 months without an alternative medical cause; OR
- •- if of child-bearing potential, she is using a highly effective method for avoidance of pregnancy for the duration of dosing and until 90 days after 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.
- •- Male patients must agree to use one of the contraception methods. This criterion must be followed from the time of the first dose of study medication and until 90 days after the last dose of study drug.
- •1. Patient completed 48 weeks of treatment during Part A.
- •2. Patient will sign and date an ICF.
- •3. Patient is willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and
- •other study procedures.
- •4. Patients agree to the following methods of contraception:
- •- Female patients of childbearing potential agree to continue using a highly effective method for avoidance of pregnancy for the duration of
- •dosing and until 90 days after the last dose. For details regarding contraception requirements. 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.
- •- Male patients must agree to use one of the contraception methods listed in Section 5.5.1 of the protocol. This criterion must be followed
- •from the time of the first dose of study medication and until 90 days after the last dose of study drug.
- •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 260
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Hx of any illness or any clinical condition that might confound the
- •results of the study or pose an additional risk in administering study
- •drug to the patient.
- •2. Previously diagnosed cancer that has not been in complete remission for at least 5 years. Localized carcinomas of the skin and carcinoma in situ of the cervix that have been resected or ablated for cure are not exclusionary.
- •3. For patients who are on drug(s) or supplements that may affect
- •muscle function or that are included in the list of drugs presented in
- •Appendix 3 of the Protocol: pt must be on a stable dose of that drug(s) or supplement for at least 3 months prior to the first dose of study drug and remain on that stable dose for the duration of the study.
- •4. History of febrile illness within 5 days before the first study drug dose
- •5. Known active opportunistic or life-threatening infections including
- •HIV and hepatitis B or C
- •6. Known active or inactive tuberculosis infection.
- •7. Current acute liver disease or chronic liver disease as defined by any of the following: current ALT =2 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN (unless participant has Gilbert's syndrome
- •characterized by the combination of total bilirubin < 3 × ULN, direct
- •bilirubin within the normal range and normal ALT and AST, or the
- •presence of mutations in the UDP-glucuronosyltransferase 1 gene,
- •indicative of Gilbert's syndrome); or Positive for hepatitis B or surface
- •antigen; or Positive for hepatitis C antibody unless additional testing forhepatitis C viral RNA is negative, ALT is < 2 × ULN and total bilirubin is = 1.5 × ULN, indicating inactive/resolved hepatitis C infection
- •8. Known severe renal impairment (defined as a glomerular filtration
- •rate of < 30 mL/min/1.73 m2).
- •9. Standard 12-lead ECG demonstrating QTcF >450 msec for male
- •patients and QTcF >470 msec for female patients 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 patient's eligibility.
- •10. History of cardiac dysrhythmias requiring anti-arrhythmia
- •treatment(s); or history or evidence of abnormal ECGs
- •11. Male patients with a female partner who is planning to become
- •pregnant during the study or within 90 days after the last study drug
- •12. Concomitant use of cytotoxic chemotherapy for cancer or known
- •ongoing or anticipated use of chronic severe immunosuppressive agents.
- •13. Positive pregnancy test or known to be pregnant or lactating or
- •planning to become pregnant during study drug administration and until 90 days after last dose
- •14. Any current mental condition (psychiatric disorder, senility or
- •15. Patient has any condition possibly affecting drug absorption
- •16. History of alcohol, analgesic/opioid, and/or illicit drug abuse, as
- •defined by the Diagnostic and Statistical Manual of Mental Disorders,
- •Fifth Edition (American Psychiatric Association, 2013), in the last 6
- •months before screening or a positive test for drugs of abuse at
- •screening. Use of CBD/THC is permitted
- •17. Use of another IP within 30 days or 5 half-lives
- •18. Current or anticipated participation in a natural hx study.
- •19. Known hypersensitivity or intolerance to losmapimod or any of its excipients
- •20. Previous participation in a Fulcrum-sponsored FSHD losmapimod
- •21. Anticipated inability to comply with any study procedures, study
- •visits according to the visit schedule through 48 weeks.
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