CTIS2023-505241-10-00招募中1 期
A phase IIa, open label, single centre study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of orally dosed MBF-015 in Huntington's disease patients - MBF-015CT-02
Medibiofarma S.L.0 个研究点目标入组 10 人开始时间: 2023年6月6日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
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
- 年龄范围
- 18 至 64（—）
- 性别
- All
入选标准
- •1. Ambulatory male or nonpregnant, nonlactating females, age =25 to =60 years old, 2. Males and females of childbearing potential must agree to use adequate birth control measures during the study. Females of childbearing potential must have a negative serum pregnancy test prior to Visit 2 and either be sexually abstinent or must use a hormonal (oral, implantable, or injectable) or double barrier method of birth control throughout the study, and until 60 days after the last dose of study drug. Females unable to bear children must have documentation of such in the source records (i.e., tubal ligation, hysterectomy, or postmenopausal [defined as a minimum of 1 year since the last menstrual period])., 3. Documented CAG triplet repeats =39 in the HTT gene. Clinical diagnostic motor features of HD, defined as UHDRS-TMS > 5 with Diagnostic Confidence Score = 4, 4. UHDRS Total Functional Capacity (TFC) scores =7 and =13 with mild-moderate cognitive impairment (MoCA score 10-25)., 5. In the opinion of the Investigator, the patient can tolerate all study procedures and is willing to comply with all other protocol requirements., 6. Able to undergo MRI scans and able to tolerate them (e.g., no metal implants including MRI incompatible IUDs, chorea of a severity that precludes MRI scans or any condition that renders testing intolerable for the patient., 7. Able to tolerate blood draws and lumbar puncture (LP)., 8. If participants are using a treatment for HD, they should be on a stable dose for at least 3 months prior to study commencement. Acceptable treatments include Dopamine D2 receptor blockers or reverse agonists, vesicular monoamine transporter 2 blockers or ?-aminobutyric acid (GABA) agonists., 9. Ability to participate fully, in the opinion of the Investigator, in all aspects of this clinical trial. Full comprehension of consent language and written informed consent must be obtained from the participant and documented.
排除标准
- •1. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures., 10. Contraindication for MRI (claustrophobia, pacemaker, aneurism clips, cardiac mechanical valve)., 11. Contraindication for lumbar puncture (anticoagulation, coagulation disease): Must not be taking anticoagulant treatment., 12. Concurrent or previous participation in another clinical trial and received investigational therapy within 4 weeks or 5 half-lives (whichever is longer) prior to Screening., 13. Any major surgery, in the investigator’s opinion, performed within 8 weeks prior to randomization or planned during the study (i.e., any surgical procedure requiring general anesthesia)., 14. Unwillingness to withhold protocol-prohibited medications during the trial., 15. Started or changed dose for concomitant medication for the treatment of HD symptoms or psychiatric disorders within 30 days prior to the Screening Visit (concomitant medications that have been administered on a stable regimen for =30 days are permitted)., 16. History of excessive alcohol or drug abuse that, in the opinion of the investigator, may interfere with the participant’s ability to comply with the study procedures., 17. Positive for opioids (unprescribed), cannabinoids, cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit., 18. Known or suspected allergy, anaphylaxis, hypersensitivity or intolerance to the study drug excipients., 19. Prior enrolment in the current study and had received study treatment., 2. Clinically significant laboratory abnormality at Screening., 20. The participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., spouse, parent, child, sibling)., 3. Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) =450 msec for males or =470 msec for females. Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the patient’s safe participation in the study or would interfere with the study assessments. Mental status, psychiatric medical history, and eligibility for the study must be documented in the screening questionnaire., 4. Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the course of the study., 5.Deemed to be at significant risk for suicidal behaviour based on any the following criteria: a.The opinion of the Investigator b.Answers yes” to Actual Suicide Attempts or Suicidal Behaviors in the Suicidal Behaviors section of the Columbia-Suicide Severity Rating Scale (C-SSRS) with reference to a 2-year period prior to the Screening Visit c.Answers yes” on any items in the Suicidal Ideation section of the C-SSRS with reference to a 6-month period prior to the Screening Visit. d.Answers yes” on any items in the Suicidal Ideation section of the C-SSRS at the Baseline Visit since the last visit (Screening Visit)., 6. Positive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV)., 7.
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