ISRCTN10176313进行中（未招募）Unknown
Phase 0 biomarker study: assessment of day-to-day, within-day and inter-individual variability in ß-Glucocerebrosidase activity and pathway biomarkers in healthy adults and patients with Parkinson’s disease with and without heterozygous GBA1-mutations
适应症
试验速览
- 阶段
- Unknown
- 状态
- 进行中（未招募）
- 入组人数
- 28
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 性别
- All
入选标准
- •Group 1 (healthy volunteers)
- •1. Male or female 50-100 years of age at screening (inclusive)
- •2. BMI in the range of 18 – 32 kg/m2
- •Group 2 and 3 (patients with Parkinson’s disease with and without a GBA1 mutation)
- •3. Male or female 50-80 years of age at screening (inclusive)
- •4. BMI in the range of 18 – 32 kg/m2
- •5. Confirmed clinical diagnosis of Parkinson's disease by a neurologist, based on the presence of bradykinesia and either resting tremor and/or muscular rigidity in at least one limb
- •6. Hoehn and Yahr stage I-III, inclusive
- •7. GBA-PD group (2): confirmed presence of heterozygous GBA1 mutation via (historic) genotyping
- •8. IPD group (3): confirmed absence of GBA1 mutation via (historic) genotyping
- •Groups 1, 2 and 3
- •9. Able to speak, read, and understand study procedures in Dutch sufficiently to allow completion of all study assessments
- •10. Must understand and provide written informed consent prior to the initiation of any protocol-specific procedures
- •11. Willing and able to maintain stable doses and regimens for all medications, herbal treatments, medical marijuana, dietary supplements and caffeine intake from the screening visit through the last study visit
- •12. Willing and able to abstain from alcohol 24 hours prior to all study procedures at study Day 1 and Day 8
- •13. Women of childbearing potential must use a form of birth control (e.g., oral contraceptive, condom use, IUD, abstinence of heterosexual intercourse).
排除标准
- •Groups 1, 2 and 3
- •1. Significant haematological abnormalities during screening such as anaemia (haemoglobin level <7.0 mmol/L (males) or <6.0 mmol/L (females)), leukopenia, or any other significant abnormalities in clinical laboratory test values. A WBC distribution will be determined to ensure (near) normal WBC distribution as determined by the investigator.
- •2. Recent participation (<90 days / 5x T1/2) in an interventional study
- •3. Any other clinically significant neuro-degenerative disorder
- •4. Recent blood loss or blood donation (>500mL whole blood) in the past 30 days
- •5. Recent infection with hospital admission (<1 month) or clinical evidence of infection at screening and study visits
- •6. Alcohol or drugs abuse in the past 12 months (a negative test for alcohol and drugs of abuse at screening and prior to the first blood sample collection will be required for inclusion)
- •7. Clinically abnormal findings in the resting ECG at screening as determined by the Investigator
- •8. Vital sign measurements must be within the following ranges during screening:
- •8.1. Body temperature, =35C to =38C
- •8.2. Systolic blood pressure, =90 to =160 mm Hg
- •8.3. Diastolic blood pressure, =40 to =95 mm Hg
- •8.4. Pulse rate, =40 to =100 bpm
- •9. Positive serology for human immunodeficiency virus (HIV), hepatitis B virus (HBV) (positive hepatitis B core antibody [anti-HBc] with negative hepatitis B DNA is acceptable), or hepatitis C virus (HCV) (treated/resolved hepatitis C with negative polymerase chain reaction [PCR] RNA is allowed)
- •10. Any other issue that, in the opinion of the investigator, would make the participant ineligible for study participation
- •11. Previous exposure to gene therapy
- •12. For CSF sampling, any of the criteria below:
- •12.1. History of clinically significant hypersensitivity to local anesthetics that may be used for LP (e.g., lidocaine)
- •12.2. Criteria that would preclude an LP, such as a local infection at the site of the LP, <100× 103/µl platelet count at screening or clinically significant coagulation abnormality or significant active bleeding, or treatment with an anticoagulant or treatment with more than two antiplatelet agents
- •12.3. History of clinically significant back pathology and/or back injury (e.g., degenerative disease, spinal deformity, or spinal surgery) that may predispose to complications or technical difficulty with LP
- •13. Clinical evidence or history of Parkinson's disease, parkinsonism or Gaucher disease
- •14. First-order relative with Parkinson's disease or Gaucher disease
- •15. Any history of unstable or poorly controlled psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematologic, immunologic, or allergic disease, or other major disorders. Well-controlled conditions are permitted if the investigator and Sponsor agree.
- •16. Presence of clinical features suggestive of atypical Parkinsonian syndromes
- •17. Presence of homozygous or compound heterozygous GBA-1 mutation or PD-related risk variant
研究者
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