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

NCT03720418 | Terminated Phase 1 DMC

• 1 other identifier: OXB-102-01
• Study Type: interventional
• Target: 6
• Locations: 2 countries
• Sites: 3

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.

Conditions

Parkinson Disease

Keywords

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

Outcome Measures

Primary Outcomes (5)

• 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

  3 months timepoint

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

  Number of clinically significant changes in clinical laboratory analysis

  3 months timepoint

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

  Number of clinically significant changes in vital signs

  3 months timepoint

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

  Number of clinically significant changes in brain MRI findings

  3 months timepoint

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

  Number of clinically significant changes in physical examination

  3 months timepoint

Secondary Outcomes (3)

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

  Baseline to 6 months

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

  Baseline to 6 months

• Change in dyskinesia rating scale score

  Baseline to 6 months

Study Arms (5)

OXB-102 Dose Level 1

EXPERIMENTAL

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

Drug: OXB-102

OXB-102 Dose Level 2

EXPERIMENTAL

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

Drug: OXB-102

OXB-102 Dose Level 3

EXPERIMENTAL

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

Drug: OXB-102

OXB-102 Selected Dose

EXPERIMENTAL

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

Drug: OXB-102

Imitation Surgical Procedure

SHAM COMPARATOR

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

Other: Imitation Surgical Procedure (ISP)

Interventions

OXB-102 DRUG

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

Also known as: AXO-Lenti-PD

OXB-102 Dose Level 1; OXB-102 Dose Level 2; OXB-102 Dose Level 3; OXB-102 Selected Dose

Imitation Surgical Procedure (ISP) OTHER

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

Imitation Surgical Procedure

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Sio Gene Therapies: lead

Study Sites (3)

Service de Neurochirurgie, Hôpital Henri Mondor

Créteil, 94010, France

Location

University of Cambridge, Centre for Brain Repair

Cambridge, Cambridgeshire, CB2 0PY,, United Kingdom

Location

The National Hospital for Neurology and Neurosurgery

London, WC1N 3BG, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 31127735 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Erika De Boever, DDS, PhD

  Sio Gene Therapies

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Study Part A open-label and Study Part B randomized, double-blind
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2018
• First Posted: October 25, 2018
• Study Start: October 17, 2018
• Primary Completion: April 12, 2022
• Study Completion: April 12, 2022
• Last Updated: May 3, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); FR (1)