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Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease (SUNRISE-PD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03720418
Recruitment Status : Active, not recruiting
First Posted : October 25, 2018
Last Update Posted : January 29, 2021
Sponsor:
Information provided by (Responsible Party):
Sio Gene Therapies

Brief Summary:
This study consists of two parts. Part A will evaluate the safety and tolerability of multiple doses of OXB-102 (AXO-Lenti-PD) in participants with Parkinson's disease. Part B will assess the safety and efficacy of the selected dose of OXB-102 in participants with Parkinson's disease.

Condition or disease Intervention/treatment Phase
Parkinson Disease Drug: OXB-102 Other: Imitation Surgical Procedure (ISP) Phase 1 Phase 2

Detailed Description:
This study consists of two parts. Part A is an open-label dose-escalation phase in which participants are enrolled in cohorts and will receive one of approximately three escalating doses of OXB-102 (AXO-Lenti-PD). Part B is a randomized, double-blind phase in which participants will be randomized to either an active group receiving the selected dose from Part A, or to a control group receiving an imitation surgical procedure (ISP).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Study Part A open-label and Study Part B randomized, double-blind
Primary Purpose: Treatment
Official Title: A Phase I/II Safety and Dose Evaluation Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral Idiopathic Parkinson's Disease (SUNRISE-PD)
Actual Study Start Date : October 17, 2018
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : December 2031

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: OXB-102 Dose Level 1
OXB-102 Dose Level 1 Single Administration (Part A: open-label)
Drug: OXB-102
Neurosurgical delivery of OXB-102 (gene therapy) to the putamen
Other Name: AXO-Lenti-PD

Experimental: OXB-102 Dose Level 2
OXB-102 Dose Level 2 Single Administration (Part A: open-label)
Drug: OXB-102
Neurosurgical delivery of OXB-102 (gene therapy) to the putamen
Other Name: AXO-Lenti-PD

Experimental: OXB-102 Dose Level 3
OXB-102 Dose Level 3 Single Administration (Part A: open-label)
Drug: OXB-102
Neurosurgical delivery of OXB-102 (gene therapy) to the putamen
Other Name: AXO-Lenti-PD

Experimental: OXB-102 Selected Dose
Selected Dose of OXB-102 Single Administration (Part B: double-blind)
Drug: OXB-102
Neurosurgical delivery of OXB-102 (gene therapy) to the putamen
Other Name: AXO-Lenti-PD

Sham Comparator: Imitation Surgical Procedure
General anesthesia with bilateral skin incisions (Part B: double-blind)
Other: Imitation Surgical Procedure (ISP)
Participants randomized to the control group in Part B will receive an ISP




Primary Outcome Measures :
  1. Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events [ Time Frame: 3 months timepoint ]
    Treatment emergent adverse events and serious adverse events will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, for severity

  2. Safety of OXB-102 as measured by changes in clinical laboratory analysis [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in clinical laboratory analysis

  3. Safety of OXB-102 as measured by changes in vital signs [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in vital signs

  4. Safety of OXB-102 as measured by changes in brain MRI findings [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in brain MRI findings

  5. Safety of OXB-102 as measured by changes in physical examination [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in physical examination


Secondary Outcome Measures :
  1. Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states [ Time Frame: Baseline to 6 months ]
  2. Change in "OFF" time during waking day compared to baseline as assessed by participant diaries [ Time Frame: Baseline to 6 months ]
  3. Change in dyskinesia rating scale score [ Time Frame: Baseline to 6 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   30 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Diagnosed with bilateral idiopathic PD
  2. Males/females between 30 and 70 years at the time of surgery
  3. Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) score of between 30 and 60 in the "OFF" medication state
  4. Presence of motor fluctuations and/or dyskinetic movement
  5. Candidate for surgical intervention
  6. Hoehn and Yahr (H&Y) Stage 3 or 4 in the "OFF" medication state
  7. Stable dosing of PD medication, including L-DOPA, for four weeks prior to screening with Levodopa equivalent daily dose (LEDD) of at least 900 mg

Key Exclusion Criteria:

  1. History of psychosis or current treatment with dopamine blocking agents and prior regular exposure to antipsychotic agents
  2. History of stereotactic or other surgery for the treatment of PD, including Deep Brain Stimulation (DBS)
  3. Participation in a prior cell or gene transfer therapy study
  4. Contraindications to use of anaesthesia
  5. Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy that cannot be temporarily stopped around the time of surgery
  6. Diagnosis of multiple system atrophy
  7. Abnormal MRI findings such as mega cisterna, septum pellucidum, signs of severe cortical or subcortical atrophy, brain tumours, vascular diseases, trauma or arteriovenous malformations
  8. Presence of dementia

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03720418


Locations
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France
Service de Neurochirurgie, Hôpital Henri Mondor
Créteil, France, 94010
United Kingdom
University of Cambridge, Centre for Brain Repair
Cambridge, Cambridgeshire, United Kingdom, CB2 0PY,
The National Hospital for Neurology and Neurosurgery
London, United Kingdom, WC1N 3BG
Sponsors and Collaborators
Sio Gene Therapies
Investigators
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Study Director: Erika De Boever, DDS, PhD Sio Gene Therapies
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Sio Gene Therapies
ClinicalTrials.gov Identifier: NCT03720418    
Other Study ID Numbers: OXB-102-01
First Posted: October 25, 2018    Key Record Dates
Last Update Posted: January 29, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sio Gene Therapies:
Parkinson's disease
Gene therapy
OXB-102
AXO-Lenti-PD
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Synucleinopathies
Neurodegenerative Diseases