2025-524674-42-00招募中2 期
A Phase 2, Randomized, Double-blind, Placebo-Controlled, Single-Center Study to Assess the effects of SUL-238 on High Energy Phosphates with Magnetic Resonance Spectroscopy (31P-MRS) in patients with Early, untreated Parkinson’s Disease (“SHEPHERD” STUDY)
Gen Ilac Ve Saglik Urunleri Sanayi Ve Ticaret A.S.1 个研究点 分布在 1 个国家目标入组 45 人开始时间: 2026年2月13日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- The (mean) change from baseline to Week 4 in each active dose group as compared to placebo in the concentration of mitochondria-related brain metabolites (ATP, phosphocreatine and inorganic phosphate), as measured by ³¹P-Magnetic Resonance Spectroscopic (MRS) imaging, in the following regions of interest (ROIs): • Putamen • Substantia Nigra • Motor Cortex
研究概览
简要总结
To assess the effect of SUL-238 on mitochondria-related brain metabolites in Parkinson’s Disease Patients.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Untreated Parkinson's Disease patients diagnosed in accordance with the UK PDS Brain Bank Criteria for the diagnosis of Parkinson's Disease. Patients must have bradykinesia and at least one of the following: a. muscular rigidity b. rest tremor (4–6 Hz) c. postural instability unrelated to primary visual, cerebellar, vestibular or proprioceptive dysfunction.
- •Women of non-childbearing potential must be post-menopausal (the last menstrual period was at least 12 months ago, and follicle-stimulating hormone [FSH] at screening confirms post-menopausal status), or have no uterus, ovaries, or fallopian tubes (or have their fallopian tubes tied). All women must have a negative pregnancy test result before administration of test article. Women who are surgically sterile must provide documentation of the procedure by an operative report or by ultrasound.
- •The duration of Parkinson's Disease since diagnosis is ≤ 1 year.
- •Patients with Modified Hoehn and Yahr stage ≤ 1.
- •Patients with Montreal Cognitive Assessment (MOCA) score of ≥22
- •Men and women aged ≥40 years at screening.
- •Able to understand the nature of the study and provide signed and dated written informed consent in accordance with local regulations before the conduct of any study related procedures.
- •Able to complete all study related testing and evaluations.
- •Patients must be, in the opinion of the Investigator, able to participate in all scheduled evaluations, likely to complete all required tests, and likely to be compliant.
- •Men and women of child-bearing potential with partners of child-bearing potential must agree to use highly effective contraception. For male patients, contraception should continue for 3 months after the last dose of investigational medicinal product (IMP, one spermatic cycle). For female patients, contraception should continue for 6 months after the last dose of IMP (one oocyte cycle). Male and female patients must refrain from sperm or oocyte donation in this period.
排除标准
- •Atypical parkinsonism, including that due to drugs, metabolic disorders, encephalitis, cerebrovascular disease, normal pressure hydrocephalus, or other neurodegenerative disease.
- •Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels greater than 1.5 times the upper limit of normal (ULN) at screening or between screening and first dose administration.
- •Received or used an investigational product (including placebo) or device within the following time period prior to day -1 in the current study: 90 days, 5 half-lives, or twice the duration of the biological effect of the investigational product (whichever is longer).
- •Use of non-prescription drugs, vitamins, herbal, and dietary supplements which has potential to influence the mitochondrial function (such as coenzyme Q10, carnitine, creatine, lipoic acid and vitamin E) within 30 days prior to day -
- •History of clinically significant sensitivity to any of the study medications, or components thereof or a history of drug or other allergies that, in the opinion of the Investigator or Medical Monitor, contraindicates their participation.
- •Women with a positive pregnancy test, are lactating, or are planning to become pregnant during the study or within 6 months of the end of this study.
- •A history or presence of any disease, condition, or surgery likely to affect drug absorption, distribution, metabolism, or excretion. Patients with a history of cholecystectomy should be excluded.
- •A Columbia-Suicide Severity Rating Scale (C-SRRS) score of >3 at screening.
- •A history or presence of a clinically significant hepatic, renal, gastrointestinal, cardiovascular, endocrine, pulmonary, ophthalmologic, immunologic, hematologic, dermatologic, or neurologic (other than PD) abnormality.
- •At screening, any clinically significant abnormalities in rhythm, conduction, or morphology of the resting ECG and any abnormalities in the 12-lead ECG that, in the judgement of the Investigator or Medical Monitor, may interfere with the interpretation of QTc interval changes, including abnormal ST-T-wave morphology or left ventricular hypertrophy.
- •A clinically significant vital signs abnormality at screening or day -
- •This includes, but is not limited to, the following, in the sitting position (3 measurements, each 5 minutes apart): a. systolic blood pressure (SBP) < 90 or >140 mmHg, b. diastolic blood pressure (DBP) < 50 or > 95 mmHg, or c. heart rate < 45 or > 100 beats per minute.
- •In the opinion of the Investigator or Medical Monitor, the patient is unlikely to comply with the protocol or is unsuitable for any reason, e.g., known issues with ability to swallow tablets.
- •Women of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control for up to 6 months following study participation.
- •Contraindications for undergoing an MRI.
- •Any history of intellectual disability or psychiatric disorders, including substance use disorders, according to the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria, except a history of mild depression/anxiety that has been resolved for at least the past 3 months.
- •A positive Hepatitis B surface antigen, Hepatitis C antibody, or Human Immunodeficiency Virus (HIV) antibody test at screening.
结局指标
主要结局
The (mean) change from baseline to Week 4 in each active dose group as compared to placebo in the concentration of mitochondria-related brain metabolites (ATP, phosphocreatine and inorganic phosphate), as measured by ³¹P-Magnetic Resonance Spectroscopic (MRS) imaging, in the following regions of interest (ROIs): • Putamen • Substantia Nigra • Motor Cortex
The (mean) change from baseline to Week 4 in each active dose group as compared to placebo in the concentration of mitochondria-related brain metabolites (ATP, phosphocreatine and inorganic phosphate), as measured by ³¹P-Magnetic Resonance Spectroscopic (MRS) imaging, in the following regions of interest (ROIs): • Putamen • Substantia Nigra • Motor Cortex
次要结局
- Mean change from baseline to Week 4 in each active dose group as compared to placebo in the predefined mitochondria-related plasma targeted metabolomics and quantitative proteomics.
- To assess safety and tolerability of SUL-238 Parkinson’s Disease Patients. a- Frequency, seriousness and intensity of adverse events (AEs). b- Changes in safety laboratory measurements, c- Clinically significant changes in vital signs d- Clinically significant changes in electrocardiogram (ECG) e- Clinically significant changes in physical and neurological examination.
研究者
Nadir Ulu
Scientific
Gen Ilac Ve Saglik Urunleri Sanayi Ve Ticaret A.S.
研究点 (1)
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