A Long-Term Follow-up Safety Study for Patients With Idiopathic Parkinson's Disease (PD) Implanted With the DDS and/or Who Received Treatment in the Main Study and/or Extension Study
试验速览
- 阶段
- 1 期
- 发起方
- Renishaw
- 入组人数
- 15
- 试验地点
- 6
- 主要终点
- Changes in clinical laboratory safety screen: activated partial thromboplastin time (aPTT) [safety-tolerability]
研究概览
简要总结
This study is a follow up to the HP-CD-CL-2002 clinical study. It evaluates the long-term safety in patients with Parkinson's disease after implantation of an investigational drug delivery system (DDS) with or without infusions of CDNF. All patients will have at least the port explanted.
详细描述
A patients participation in the study will last for up to 4 years and will include up to nine visits:
- Screening (1 visit, same as HP-CD-CL-2003 End-of-Study visit)
- Pre-surgery / explantation surgery / post-surgery (3 visits)
- Safety (4 visits)
- DAT-PET (Dopamine Transporter - Positron Emission Tomography) examination (1 visit)
- End-of-study visit (1 visit)
Study examinations and assessments:
- Physical examination: pulse rate, blood pressure, temperature, body weight and height, body mass index (BMI), neurological exam
- ECG (electrocardiography) and blood and urine tests
- Pregnancy tests for women of childbearing age
- Completion of a patient diary to record mobility and time asleep
- Parkinson's Kinetigraph (PKGTM) Data Logger: a watch-type movement recording device
- Questionnaires, rating scales and forms: quality of life, mood, memory, impulse control, mental health.
- Magnetic resonance imaging (MRI)
- Positron emission tomography scans (PET)
- Skin condition over any remaining implanted subcutaneous portion of the device.
- Skin healing after port / device removal.
For more information: https://treater.eu/clinical-study/
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 35 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of visit 4 (implantation of DDS) within main study HP-CD-CL-
- •Patients who:
- •Discontinued main study after visit 4 of main study or discontinued extension study.
- •Received 6 doses in main study but didn't participate in extension study.
- •Received 12 doses including extension study.
- •Provision of informed consent.
排除标准
- 未提供
结局指标
主要结局
Changes in clinical laboratory safety screen: activated partial thromboplastin time (aPTT) [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: aPTT (sec) . Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Changes in physical examination: clinical standard neurological examination
时间窗: Week 71, and Months 25, 34, 58
A clinical standard neurological examination by study investigator. Changes in motor function, sensory function, cranial nerve function (visual fields), cortical functions and reflexes are followed in the examination, scored as normal - abnormal without clinical relevance - abnormal with clinical relevance
Changes in vital signs: body temperature [safety-tolerability]
时间窗: Weeks 55, 57, 58, 75
Changes in body temperature during the study (in degrees celcius)
Incidence of treatment-emergent adverse events (AEs)[safety-tolerability]
时间窗: Until study completion, up to month 58
Total number, causality and severity of adverse events at any time during the study period
Change in Electrocardiogram (ECG): Ventricular rate (bpm), [safety-tolerability]
时间窗: Week 71 and Month 25
Changes in electrical activity of heartbeat measured by electrocardiogram: Ventricular rate (bpm),
Change in Electrocardiogram (ECG): PR (pulse rate) interval, qRS duration, QT, QTc (msec) [safety-tolerability]
时间窗: Week 71 and Month 25
Changes in electrical activity of heartbeat measured by electrocardiogram: PR interval (msec), QRS duration (msec), QT (msec), QTc (msec)
Changes in vital signs: body weight [safety-tolerability]
时间窗: Weeks 55, 57, 58, 75
Changes in body weight during the study (in kilograms)
Changes in vital signs: blood pressure [safety-tolerability]
时间窗: Weeks 55, 57, 58, 75
Changes in blood pressure during the study , measured as systolic and diastolic blood pressure (in mmHg)
Changes in vital signs: pulse rate [safety-tolerability]
时间窗: Weeks 55, 57, 58, 75
Changes in pulse rate during the study (in beats per minute)
Changes in clinical laboratory safety screen: mean cell haemoglobin of RBC (MHC) [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: MCH (Mean cell hemoglobin) (pg). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Device related occurrence of adverse device effects [safety-tolerability]
时间窗: Week 49 and Month 58
Occurrence of adverse device effects (ADE) at any time of the study period, for either the whole system or the individual sub systems (guide tubes/catheters, subcutaneous components, port), serious adverse device effect (SADE) including long term effects, neurological deficit (seizures), infection (local to components, in CNS), severe skin breakdown or necrosis requiring component removal life threatening or major (requiring intervention) intracerebral haemorrhage.
Change in Beck Depression Inventory (BDI) score [safety-tolerability]
时间窗: Week 71 and Month 25
Assessment of change in depression using Beck Depression Inventory (BDI) score: Sadness: Pessimism; Past Failure; Loss of pleasure; Guilty feelings; Punishment Feelings; Self-dislike; Self-criticalness;Suicidal thoughts or wishes; Crying; Agitation; Loss of interest; Indecisiveness;Worthlessness; Loss of energy; Changes in sleeping pattern; Irritability; Changes in appetite; Concentration difficulty; Tiredness or fatique; Loss of interest in sex. Rated on a 4-point scale ranging from 0 to 3 based on severity of each item (0=low intensity; 3=highest intensity). The maximum total score is 63.
Change in Questionnaire for impulsive-compulsive disorder in Parkinson's disease rating scale (QUIP_RS) [safety-tolerability]
时间窗: Week 71 and Month 25
Assessment of changes in impulsive-compulsive disorders using QUIP_RS. Questions scored 0-4 (0=never; 4=very often) on gambling, sex, buying, eating, performing tasks/hobbies, repeating simple activities, and taking Parkinson's disease medication. Total QUIP-RS Score 0-112 Assessment of changes in impulsive-compulsive disorders using QUIP_RS. Questions scored 0-4 (0=never; 4=very often) on gambling, sex, buying, eating, performing tasks/hobbies, repeating simple activities, and taking Parkinson's disease medication. Total QUIP-RS Score 0-112 Assessment of changes in impulsive-compulsive disorders using QUIP_RS. Questions scored 0-4 (0=never; 4=very often) on gambling, sex, buying, eating, performing tasks/hobbies, repeating simple activities, and taking Parkinson's disease medication. Total QUIP-RS Score 0-112
Changes in clinical laboratory safety screen: mean cell volume (MCV) of red blood cells [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: MCV of red blood cells (fL). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Changes in clinical laboratory safety screen: Platelet count [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: Platelet count (10E9/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Formation of anti-CDNF antibodies [safety-tolerability]
时间窗: Weeks 58, 78 and Month 25
Formation and change in anti-CDNF antibody concentration (in ng/ml).
Changes in clinical laboratory safety screen: haematology - haemoglobin [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: hemoglobin (g/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Changes in clinical laboratory safety screen: white blood call (WBC) count [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: Cell counts (10E9/L) for total WBC, neutrophils, lymphocytes, monocytes, eosinophils and basophils. Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Changes in clinical laboratory safety screen: International Normalised Ratio (INR) [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: INR (standardized prothrombin time) to determine the effects of oral anticoagulants on the clotting system. Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Change in Montreal cognitive assessment (MoCA) [safety-tolerability]
时间窗: Week 71 and Month 25
Assessment of change in cognitive domains using MoCA test: attention and, concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. The total possible score is 30 points; a score of 26 or above is considered normal.
Changes in physical examination: anatomic findings [safety-tolerability]
时间窗: Week 71, and Months 25, 34, 58
Changes in anatomic findings found in physical examination of the following body systems: general inspection/upper extremities; head, eyes, ears, nose, throat, and superficial cervial lymph notes; neck, shoulders, back; chest and lungs; cardiovascular; abdomen; lower extremities
Changes in vital signs: body mass index (BMI) [safety-tolerability]
时间窗: Weeks 55, 57, 58, 75
Changes in body mass index during the study (in kg/m\^2)
Changes in clinical laboratory safety screen: clinical chemistry [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for clinical chemistry (Na, K, Urea, creatinine, creatine kinase, Ca, Bilirubin, IgG (Immunoglobulin G), Albumin, ALP(Alkaline phosphatase), ALT (Alanine transaminase), AST (Aspartate transaminase))
Changes in clinical laboratory safety screen: haematology - haematocrit [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: hematocrit (%, ratio of red blood cell volume to total blood volume). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Changes in clinical laboratory safety screen: haematology - red blood cell (RBC) count [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
Changes in laboratory variables for haematology: RBC count (10E12/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
Changes in clinical laboratory safety screen: urinanalysis [safety-tolerability]
时间窗: Weeks 53, 55, 57, 58, 71 and Month 25
Changes in laboratory variables for urinanalysis (blood/erythrocytes, glucose, ketones, leukocytes, nitrites, pH, protein) studied by dipstick and scored 0-3. Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance".
次要结局
- Change in TUG (Timed Up and Go) test [efficacy](Week 71 and Month 25)
- Change in PDQ-39 (Parkinson's Disease Questionnaire) score [efficacy](Week 71 and Month 25)
- Change in UPDRS (Unified Parkinson's Disease Rating Scale) Part III motor score [efficacy](Week 71 and Month 25)
- Change in home diary score [efficacy](Weeks 40, 45, 49, 53, 57, 58, 61, 65, 71, and Month 25)
- Change in CGI-I (Clinical Global Impression - Improvement) scale [efficacy](Weeks 40, 45, 49, 53, 57, 58, 61, 65, 71, and Month 25)
- Change in UPDRS Total score (Part I-IV) [efficacy](Week 71 and Month 25)
- Healing of patient skin after port removal(Month 25, 34 and 58)