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

Primary Purpose

Parkinson Disease

Status

Terminated

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

OXB-102

Imitation Surgical Procedure (ISP)

About this trial

This is an interventional treatment trial for Parkinson Disease focused on measuring Parkinson's disease, Gene therapy, OXB-102, AXO-Lenti-PD

Eligibility Criteria

30 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Diagnosed with bilateral idiopathic PD
Males/females between 30 and 70 years at the time of surgery
Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) score of between 30 and 60 in the "OFF" medication state
Presence of motor fluctuations and/or dyskinetic movement
Candidate for surgical intervention
Hoehn and Yahr (H&Y) Stage 3 or 4 in the "OFF" medication state
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:

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

Sites / Locations

Service de Neurochirurgie, Hôpital Henri Mondor
University of Cambridge, Centre for Brain Repair
The National Hospital for Neurology and Neurosurgery

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Sham Comparator

Arm Label

OXB-102 Dose Level 1

OXB-102 Dose Level 2

OXB-102 Dose Level 3

OXB-102 Selected Dose

Imitation Surgical Procedure

Arm Description

OXB-102 Dose Level 1 Single Administration (Part A: open-label)

OXB-102 Dose Level 2 Single Administration (Part A: open-label)

OXB-102 Dose Level 3 Single Administration (Part A: open-label)

Selected Dose of OXB-102 Single Administration (Part B: double-blind)

General anesthesia with bilateral skin incisions (Part B: double-blind)

Outcomes

Primary Outcome Measures

Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events

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 as measured by changes in clinical laboratory analysis

Number of clinically significant changes in clinical laboratory analysis

Safety of OXB-102 as measured by changes in vital signs

Number of clinically significant changes in vital signs

Safety of OXB-102 as measured by changes in brain MRI findings

Number of clinically significant changes in brain MRI findings

Safety of OXB-102 as measured by changes in physical examination

Number of clinically significant changes in physical examination

Secondary Outcome Measures

Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states

Change in "OFF" time during waking day compared to baseline as assessed by participant diaries

Change in dyskinesia rating scale score

Full Information

NCT ID

NCT03720418

First Posted

October 16, 2018

Last Updated

April 27, 2022

Sponsor

Sio Gene Therapies

1. Study Identification

Unique Protocol Identification Number

NCT03720418

Brief Title

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

Acronym

SUNRISE-PD

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)

Study Type

Interventional

2. Study Status

Record Verification Date

April 2022

Overall Recruitment Status

Terminated

Why Stopped

As a result of termination of development of OXB-102-01, it is no longer possible for Sio to either commence or to continue any related clinical trials.

Study Start Date

October 17, 2018 (Actual)

Primary Completion Date

April 12, 2022 (Actual)

Study Completion Date

April 12, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sio Gene Therapies

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Parkinson Disease

Keywords

Parkinson's disease, Gene therapy, OXB-102, AXO-Lenti-PD

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Sequential Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Masking Description

Study Part A open-label and Study Part B randomized, double-blind

Allocation

Randomized

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

OXB-102 Dose Level 1

Arm Type

Experimental

Arm Description

OXB-102 Dose Level 1 Single Administration (Part A: open-label)

Arm Title

OXB-102 Dose Level 2

Arm Type

Experimental

Arm Description

OXB-102 Dose Level 2 Single Administration (Part A: open-label)

Arm Title

OXB-102 Dose Level 3

Arm Type

Experimental

Arm Description

OXB-102 Dose Level 3 Single Administration (Part A: open-label)

Arm Title

OXB-102 Selected Dose

Arm Type

Experimental

Arm Description

Selected Dose of OXB-102 Single Administration (Part B: double-blind)

Arm Title

Imitation Surgical Procedure

Arm Type

Sham Comparator

Arm Description

General anesthesia with bilateral skin incisions (Part B: double-blind)

Intervention Type

Drug

Intervention Name(s)

OXB-102

Other Intervention Name(s)

AXO-Lenti-PD

Intervention Description

Neurosurgical delivery of OXB-102 (gene therapy) to the putamen

Intervention Type

Other

Intervention Name(s)

Imitation Surgical Procedure (ISP)

Intervention Description

Participants randomized to the control group in Part B will receive an ISP

Primary Outcome Measure Information:

Title

Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events

Description

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

Time Frame

3 months timepoint

Title

Safety of OXB-102 as measured by changes in clinical laboratory analysis

Description

Number of clinically significant changes in clinical laboratory analysis

Time Frame

3 months timepoint

Title

Safety of OXB-102 as measured by changes in vital signs

Description

Number of clinically significant changes in vital signs

Time Frame

3 months timepoint

Title

Safety of OXB-102 as measured by changes in brain MRI findings

Description

Number of clinically significant changes in brain MRI findings

Time Frame

3 months timepoint

Title

Safety of OXB-102 as measured by changes in physical examination

Description

Number of clinically significant changes in physical examination

Time Frame

3 months timepoint

Secondary Outcome Measure Information:

Title

Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states

Time Frame

Baseline to 6 months

Title

Change in "OFF" time during waking day compared to baseline as assessed by participant diaries

Time Frame

Baseline to 6 months

Title

Change in dyskinesia rating scale score

Time Frame

Baseline to 6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

30 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Diagnosed with bilateral idiopathic PD Males/females between 30 and 70 years at the time of surgery Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) score of between 30 and 60 in the "OFF" medication state Presence of motor fluctuations and/or dyskinetic movement Candidate for surgical intervention Hoehn and Yahr (H&Y) Stage 3 or 4 in the "OFF" medication state 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: History of psychosis or current treatment with dopamine blocking agents and prior regular exposure to antipsychotic agents History of stereotactic or other surgery for the treatment of PD, including Deep Brain Stimulation (DBS) Participation in a prior cell or gene transfer therapy study Contraindications to use of anaesthesia Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy that cannot be temporarily stopped around the time of surgery Diagnosis of multiple system atrophy Abnormal MRI findings such as mega cisterna, septum pellucidum, signs of severe cortical or subcortical atrophy, brain tumours, vascular diseases, trauma or arteriovenous malformations Presence of dementia

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Erika De Boever, DDS, PhD

Organizational Affiliation

Sio Gene Therapies

Official's Role

Study Director

Facility Information:

Facility Name

Service de Neurochirurgie, Hôpital Henri Mondor

City

Créteil

ZIP/Postal Code

94010

Country

France

Facility Name

University of Cambridge, Centre for Brain Repair

City

Cambridge

State/Province

Cambridgeshire

ZIP/Postal Code

CB2 0PY,

Country

United Kingdom

Facility Name

The National Hospital for Neurology and Neurosurgery

City

London

ZIP/Postal Code

WC1N 3BG

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

31127735

Citation

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. No abstract available.

Results Reference

derived

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

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