Clinical trial NCT04228653: Long-Term Follow-up Safety After DDS Implantation With/Without CDNF Infusions
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Parkinson Disease
Movement Disorders
Neuro-Degenerative Disease
Nervous System Diseases
Brain Diseases
Keywords: Parkinson
CDNF
Drug Delivery System
Intracerebral
Parkinson Disease
Nervous System Disease
Movement Disorders
Brain Diseases
Neurodegenerative Diseases
Parkinsonian Disorders
Basal Ganglia Diseases
Central Nervous System Diseases
Dopamine
Cardiotonic Agents
Sympathomimetrics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Protective Agents
Lead sponsor: Renishaw
Sponsor class: INDUSTRY
Collaborators: name: Herantis Pharma Plc.
First posted: 2020-01-14
Enrollment: 15
Sex: ALL
Minimum age (years): 35
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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/

Eligibility criteria: Inclusion Criteria:

1. Completion of visit 4 (implantation of DDS) within main study HP-CD-CL-2002.
2. 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.
3. Provision of informed consent.

Exclusion Criteria:

* None

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (AEs)[safety-tolerability]
timeFrame: Until study completion, up to month 58
measure: Change in Electrocardiogram (ECG): Ventricular rate (bpm), [safety-tolerability]
timeFrame: Week 71 and Month 25
measure: Change in Electrocardiogram (ECG): PR (pulse rate) interval, qRS duration, QT, QTc (msec) [safety-tolerability]
timeFrame: Week 71 and Month 25
measure: Change in Beck Depression Inventory (BDI) score [safety-tolerability]
timeFrame: Week 71 and Month 25
measure: Change in Questionnaire for impulsive-compulsive disorder in Parkinson's disease rating scale (QUIP_RS) [safety-tolerability]
timeFrame: Week 71 and Month 25
measure: Change in Montreal cognitive assessment (MoCA) [safety-tolerability]
timeFrame: Week 71 and Month 25
measure: Changes in physical examination: anatomic findings [safety-tolerability]
timeFrame: Week 71, and Months 25, 34, 58
measure: Changes in physical examination: clinical standard neurological examination
timeFrame: Week 71, and Months 25, 34, 58
measure: Changes in vital signs: blood pressure [safety-tolerability]
timeFrame: Weeks 55, 57, 58, 75
measure: Changes in vital signs: pulse rate [safety-tolerability]
timeFrame: Weeks 55, 57, 58, 75
measure: Changes in vital signs: body temperature [safety-tolerability]
timeFrame: Weeks 55, 57, 58, 75
measure: Changes in vital signs: body weight [safety-tolerability]
timeFrame: Weeks 55, 57, 58, 75
measure: Changes in vital signs: body mass index (BMI) [safety-tolerability]
timeFrame: Weeks 55, 57, 58, 75
measure: Changes in clinical laboratory safety screen: clinical chemistry [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: haematology - haemoglobin [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: haematology - haematocrit [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: haematology - red blood cell (RBC) count [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: mean cell volume (MCV) of red blood cells [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: mean cell haemoglobin of RBC (MHC) [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: Platelet count [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: white blood call (WBC) count [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: activated partial thromboplastin time (aPTT) [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: International Normalised Ratio (INR) [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Months 25, 34, 58
measure: Changes in clinical laboratory safety screen: urinanalysis [safety-tolerability]
timeFrame: Weeks 53, 55, 57, 58, 71 and Month 25
measure: Formation of anti-CDNF antibodies [safety-tolerability]
timeFrame: Weeks 58, 78 and Month 25
measure: Device related occurrence of adverse device effects [safety-tolerability]
timeFrame: Week 49 and Month 58
Secondary outcomes:
measure: Change in UPDRS (Unified Parkinson's Disease Rating Scale) Part III motor score [efficacy]
timeFrame: Week 71 and Month 25
measure: Change in TUG (Timed Up and Go) test [efficacy]
timeFrame: Week 71 and Month 25
measure: Change in UPDRS Total score (Part I-IV) [efficacy]
timeFrame: Week 71 and Month 25
measure: Change in home diary score [efficacy]
timeFrame: Weeks 40, 45, 49, 53, 57, 58, 61, 65, 71, and Month 25
measure: Change in PDQ-39 (Parkinson's Disease Questionnaire) score [efficacy]
timeFrame: Week 71 and Month 25
measure: Change in CGI-I (Clinical Global Impression - Improvement) scale [efficacy]
timeFrame: Weeks 40, 45, 49, 53, 57, 58, 61, 65, 71, and Month 25
measure: Healing of patient skin after port removal
timeFrame: Month 25, 34 and 58
Other outcomes:
measure: Change in DAT (dopamine transporter)-PET imaging [exploratory]
timeFrame: Week 63
measure: Change in daily activity measurement [exploratory]
timeFrame: Weeks 58, 71 and Month 25
Locations:
city: Helsinki
country: Finland
facility: Helsinki University Hospital
city: Lund,
country: Sweden
facility: Skåne University Hospital
city: Stockholm
country: Sweden
facility: Karolinksa University Hospital
Study officials:
References: