EUCTR2020-000105-92-ES进行中（未招募）1 期
A Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease
ovartis Farmacéutica S.A.0 个研究点目标入组 75 人开始时间: 2021年8月26日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 75
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Signed informed consent must be obtained prior to participation in the study
- •2. Must be capable of providing informed consent
- •3. Clinically diagnosed Stage 1 or 2 HD with a diagnostic confidence interval (DCL) = 4 and a UHDRS Total Functional Capacity (TFC) >8 at screening
- •4. Genetically confirmed HD, with presence of =40 CAG repeats in the huntingtin gene
- •- For participants without prior documentation, a sample must be sent to the CAG lab vendor and confirmation of the CAG repeat length for these participants must be obtained prior to randomization
- •- For participants with previously existing documentation of their CAG repeat length, it is acceptable to use this prior data to qualify for randomization. These participants must also submit a sample for CAG repeat testing to be conducted by the central study laboratory.
- •5. Male and female participants between 25 to 75 years of age, inclusive, on the day of Informed Consent signature
- •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 50
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 25
排除标准
- •1. Use of other investigational drugs within 5 half-lives of the first dose of study drug or within 30d, whichever is longer.
- •2. Prior participation in clinical trial investigating a huntingtin-lowering therapy (unless participant received only placebo)
- •3. History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes
- •4. Participants taking medications prohibited by the protocol
- •5. Any medical history, lumbar surgery or condition that would interfere with the ability to complete the protocol specified assessments
- •6. History of malignancy of any organ system, treated or untreated, regardless of whether there is evidence of local recurrence or metastases
- •7. Participant has other severe, acute or chronic medical conditions including unstable psychiatric conditions or laboratory abnormalities that in the opinion of the Investigator may increase the risk associated with study participation, or that may interfere with the interpretation of study results
- •8. Score yes on item 4 or 5 of the C-SSRS, if this ideation occurred in the past 6m from screening or yes on any item of the Suicidal Behavior section, except for the Non-Suicidal Self-Injurious Behavior, if this behavior occurred in the past 2y
- •9. Pregnant or nursing (lactating) women. WOCB potential should not become pregnant during the study or 7 months after stopping study medication.
- •10. Sexually active males unwilling to use a condom together with a spermicidal agent during intercourse while taking study drug and for 120d (in total) after the last dose of the study drug. A condom is required to be used also by vasectomized men as well during intercourse with a male partner of the study subject in order to prevent delivery of the genotoxic drug via semen.
- •11. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using one highly effective methods of contraception during dosing and for 7 months after stopping the study medication. Highly effective methods of birth control are those methods that have a less than 1% chance of an unwanted pregnancy during 1 year (see protocol).
- •In addition to one highly effective method of contraception, a condom is required for all male partners of female participants to prevent fathering a child AND to prevent exposure of study treatment via vaginal fluid to your partner, until at least 7 months following the last dose of study treatment.
- •Total abstinence, Periodic abstinence and withdrawal are NOT acceptable methods of contraception. Oral contraception cannot be considered due to potential decreased efficacy as potential DDI with branaplam.
- •In case local regulations deviate from the contraception methods listed above, local regulations apply and will be described in the ICF.
- •12. History of:
- •- Gene therapy or cell transplantation or any other experimental brain surgery
- •- Hepatitis B or hepatitis C or serologic evidence for active viral hepatitis
- •- Immunodeficiency diseases, including a positive HIV test
- •- History or current evidence of drug or alcohol abuse in the 12m prior to screening, as defined by the DSM-V criteria for substance abuse. For former abusers, abstinence should be confirmed by laboratory tests
- •13. Any surgical or medical condition, which might put the participant at risk in case of participation in the study. The Investigator should make this determination in consideration of the participant’s medical history and/or clinical o
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