EUCTR2022-000389-16-NL招募中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
- 招募中
- 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 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 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
- •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 of study drug
- •- 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. Patient 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.
- •- 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 for hepatitis 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 dose
- •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 dementia)
- •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 study
- •21. Anticipated inability to comply with any study procedures, study visits according to the visit schedule through 48 weeks.
研究者
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