A Phase I-II, Randomised, Double-Blind, Placebo Controlled, Safety and Tolerability Study of Intermittent Bilateral Intraputamenal Cerebral Dopamine Neurotrophic Factor (CDNF) Infusions Administered via an Investigational Drug Delivery System to Patients with Idiopathic Parkinson’s Disease (PD) of Moderate Severity.

Herantis Pharma Plc0 个研究点目标入组 17 人开始时间: 2016年7月7日最近更新: 2024年9月9日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方
入组人数: 17

研究概览入排标准研究者记录与标识符相似试验

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

•Eligible patients must fulfill all of the following criteria (at screening):
•1.Subjects diagnosed with idiopathic PD according to the UK Brain Bank Criteria. Bilateral findings must be present at study entry.
•2. Duration of PD motor symptoms 5-15 years (inclusive), verified by subject’s medical records.
•3. Age 35-75 years (inclusive).
•4. Presence of motor fluctuations. Subjects must have an average of at least 2.5 hours of Off-time per day on 3-day fluctuation diaries completed during screening.
•5. At least 5 daily doses of Levodopa
•6. Ability to reliably distinguish motor states (ON without dyskinesia, ON with non-troublesome dyskinesia, ON with troublesome dyskinesia and OFF) and accurately complete fluctuation diaries.
•7. UPDRS motor score (part III) in a practically defined OFF-state between 25-50 (inclusive).
•8. Hoehn and Yahr = stage III in the OFF-state.
•9. Responsiveness to levodopa (=30% improvement in motor UPDRS [part III] following a levodopa challenge)
•10. No change in anti-parkinsonian medication for 6 weeks before screening.
•11. Females of childbearing potential must have a negative pregnancy test at study entry and be willing to use a highly effective form of contraception until 30 days after the end of the study (hormonal contraception associated with inhibition of ovulation, IUD, IUS, bilateral tubal occlusion, vasectomised partner or sexual abstinence). Males (non-vasectomised) must be willing to use condom during intercourse and do not donate sperms for three months following each DAT-PET scan. Female partners of childbearing potential should be willing to use a highly effective form of contraception until three months after their male partner's DAT-PET scan.
•12.Provision of informed consent. The patient is judged by the investigator to be alert and oriented to person, place, time and situation when giving the informed consent.
•Post-surgery Randomization Criteria
•13. No relevant sequelae from catheter implantation such as clinically significant intracerebral trauma, haemorrhage, or infection.
•14. At least one functioning catheter tip in each putamen.
•15. Implanted catheter trajectories are satisfactory from a safety perspective and there have been no changes in pathology after implantation surgery(s), which give rise to safety concerns.
•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 11
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 6

排除标准

•1. Diagnosed with atypical parkinsonism or any known secondary parkinsonian syndrome including but not limited to medication induced, toxic, vascular, post-traumatic or post-infectious parkinsonism, progressive supranuclear palsy, multiple systems atrophy, or other neurodegenerative disorder associated with parkinsonism.
•2. Signs or symptoms suggestive of atypical parkinsonian syndrome including supranuclear gaze palsy, early postural instability and falls (within 3 years of disease onset), cerebellar signs, myoclonus, disproportionate antecollis, extensor plantar responses, cortical sensory loss, emotional incontinence (pseudobulbar affect), severe bulbar dysfunction (dysarthria, dysphonia or dysphagia) or respiratory symptoms such as stridor or inspiratory sighs.
•3. Drug-resistant rest tremor, severe dyskinesia or severe head tremor, which could interfere with treatment and test infusions.
•4. Prior neurosurgical treatment for PD, including previous treatment with platelet-derived growth factor (PDGF-BB), glial cell-line derived neurotrophic factor (GDNF), lesioning or deep brain stimulation.
•5. Significant neurological disorder other than PD including clinically significant head trauma, cerebrovascular disease, epilepsia, CSF shunt or other implanted CNS device.
•6. Presence of significant depression as defined as a Beck Depression Inventory (BDI) score = 20.
•7. Current psychosis requiring therapy. The presence of benign hallucinosis is not exclusionary.
•8. Presence of clinically significant impulse control disorder by a positive screen on the questionnaire for impulsive-compulsive disorders in Parkinson's Disease (QUIP-RS) score > 20, or presence of dopamine dysregulation syndrome.
•9. Montreal Cognitive Assessment (MoCA) score < 24.
•10. Use within 3 months of planned catheter insertion of concomitant medications known to affect PD symptoms other than prescribed PD therapy including but not limited to neuroleptics or antipsychotic medication prescribed for the treatment of psychosis, central dopamine receptor blockers, or other tricyclic antidepressants.
•11. Any medical condition, which might impair outcome measure assessments or safety measures including ability to undergo MRI or DAT-PET. Estimated Glomerular Filtration
•rate (eGFR) < 30 mL/min/1.73 m2.
•12.Hypersensitivity or allergy to gadolinium or to any of the excipients of the macrocyclic GBCA used for the surgical planning MRI.
•13. Screening and/or planning MRI demonstrating any abnormality, which would suggest an alternative cause for patient’s parkinsonism or preclude neurosurgery, including but not limited to brain atrophy, anatomical abnormalities within the catheter target area and inability to plan safe trajectories for 4 catheters (2 per putamen) to the target area of the putamen.
•14. Any medical condition that would put the subject at undue risk from surgical treatment or chronic implants including but not limited to bleeding disorders, chronic infections, or immunosuppressive illness.
•15. History within the last 5 years of cancer with the exception of basal cell carcinoma of the skin.
•16. History of drug or alcohol abuse within 2 years of screening.
•17. Use of any investigational drug or device within 90 days of screening.
•18. Active breastfeeding.

研究者

发起方

Herantis Pharma Plc

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