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Trial record 1 of 1 for:    NCT03720418
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Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease (SUNRISE-PD)

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ClinicalTrials.gov Identifier: NCT03720418
Recruitment Status : Recruiting
First Posted : October 25, 2018
Last Update Posted : April 17, 2020
Sponsor:
Collaborator:
Oxford BioMedica
Information provided by (Responsible Party):
Axovant Sciences Ltd.

Tracking Information
First Submitted Date  ICMJE October 16, 2018
First Posted Date  ICMJE October 25, 2018
Last Update Posted Date April 17, 2020
Actual Study Start Date  ICMJE October 17, 2018
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 24, 2018)
  • Safety of OXB-102-01 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
  • Safety of OXB-102-01 as measured by changes in clinical laboratory analysis [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in clinical laboratory analysis
  • Safety of OXB-102-01 as measured by changes in vital signs [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in vital signs
  • Safety of OXB-102-01 as measured by changes in brain MRI findings [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in brain MRI findings
  • Safety of OXB-102-01 as measured by changes in physical examination [ Time Frame: 3 months timepoint ]
    Number of clinically significant changes in physical examination
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 24, 2018)
  • The change in Unified Parkinson's Disease Rating Scale (UPDRS) scores compared to baseline in defined "OFF" and "ON" medication states [ Time Frame: 6 months timepoint ]
  • The change in motor fluctuations compared to baseline as assessed by patient diaries [ Time Frame: 6 months timepoint ]
  • The change in dyskinesia rating scale from baseline [ Time Frame: 6 months timepoint ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease
Official Title  ICMJE A Phase I/II Safety and Dose Evaluation Study of OXB-102 in Patients With Bilateral Idiopathic Parkinson's Disease
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 patients with Parkinson's disease. Part B will assess the safety and efficacy of the selected dose of OXB-102 in patients with Parkinson's disease.
Detailed Description This study consists of two parts. Part A is an open-label dose-escalation phase in which patients 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 patients 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).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE 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
Condition  ICMJE Parkinson Disease
Intervention  ICMJE
  • Drug: OXB-102
    Neurosurgical delivery of OXB-102 (gene therapy) to the striatum
    Other Name: AXO-Lenti-PD
  • Other: Imitation Surgical Procedure (ISP)
    Patients randomized to the control group in Part B will receive an ISP
Study Arms  ICMJE
  • Experimental: OXB-102 Dose Level 1
    OXB-102 Dose Level 1 Single Administration (Part A: open-label)
    Intervention: Drug: OXB-102
  • Experimental: OXB-102 Dose Level 2
    OXB-102 Dose Level 2 Single Administration (Part A: open-label)
    Intervention: Drug: OXB-102
  • Experimental: OXB-102 Dose Level 3
    OXB-102 Dose Level 3 Single Administration (Part A: open-label)
    Intervention: Drug: OXB-102
  • Experimental: OXB-102 Selected Dose
    Selected Dose of OXB-102 Single Administration (Part B: double-blind)
    Intervention: Drug: OXB-102
  • Sham Comparator: Imitation Surgical Procedure
    General anesthesia with bilateral skin incisions (Part B: double-blind)
    Intervention: Other: Imitation Surgical Procedure (ISP)
Publications * McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS: 1. GENE THERAPY FOR PARKINSON'S, 2. PHASE 3 STUDY IN FOCUS - INTEC PHARMA'S ACCORDION PILL, 3. CLINICAL TRIALS RESOURCES. J Parkinsons Dis. 2019;9(2):251-264. doi: 10.3233/JPD-199001.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 24, 2018)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2031
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
  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
  8. Levodopa equivalent daily dose (LEDD):

at least 900 mg

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 30 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Travis B Lewis, MD, PhD 1-646-593-8403 travis.lewis@axovant.com
Contact: Rizalyn Golnar 1 -646-495-5312 Rizalyn.Golnar@axovant.com
Listed Location Countries  ICMJE France,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03720418
Other Study ID Numbers  ICMJE OXB-102-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Axovant Sciences Ltd.
Study Sponsor  ICMJE Axovant Sciences Ltd.
Collaborators  ICMJE Oxford BioMedica
Investigators  ICMJE
Study Director: Travis B Lewis, MD, PhD Axovant Sciences
PRS Account Axovant Sciences Ltd.
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP