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Parkinson DiseasePhase 1/2SUNRISE-PDNCT03720418

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

A Phase 1/2 study testing OXB-102 and Imitation Surgical Procedure (ISP) in people with Parkinson Disease, run by Sio Gene Therapies at 3 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 30 to 70 years
Conditions the study is for
Parkinson Disease
What is being tested?
OXB-102 and Imitation Surgical Procedure (ISP)
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
6 participants
Where
3 sitesin 2 countries
Study dates
17 Oct 2018to 12 Apr 2022
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 30 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

3 sites in 2 countries.

3 sites in 2 countries

United Kingdom2 sites

  • University of Cambridge, Centre for Brain Repair

    Cambridge, Cambridgeshire, CB2 0PY,

  • The National Hospital for Neurology and Neurosurgery

    London, WC1N 3BG

Other trials in United Kingdom

France1 site

  • Service de Neurochirurgie, Hôpital Henri Mondor

    Créteil, 94010

Other trials in France

About this study

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)

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.

Read the 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).

Being tested
OXB-102, Imitation Surgical Procedure (ISP)
Keywords
Parkinson's disease, Gene therapy, OXB-102, AXO-Lenti-PD
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OXB-102Drug

    Also called AXO-Lenti-PD

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

  • Imitation Surgical Procedure (ISP)Other

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

Study groups in NCT03720418
Study groupRoleWhat they receive
OXB-102 Dose Level 1ExperimentalOXB-102 Dose Level 1 Single Administration (Part A: open-label)
Receives: Drug: OXB-102
OXB-102 Dose Level 2ExperimentalOXB-102 Dose Level 2 Single Administration (Part A: open-label)
Receives: Drug: OXB-102
OXB-102 Dose Level 3ExperimentalOXB-102 Dose Level 3 Single Administration (Part A: open-label)
Receives: Drug: OXB-102
OXB-102 Selected DoseExperimentalSelected Dose of OXB-102 Single Administration (Part B: double-blind)
Receives: Drug: OXB-102
Imitation Surgical ProcedureSham comparatorGeneral anesthesia with bilateral skin incisions (Part B: double-blind)
Receives: Other: Imitation Surgical Procedure (ISP)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 3 months timepoint

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

    Number of clinically significant changes in clinical laboratory analysis

    Measured: 3 months timepoint

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

    Number of clinically significant changes in vital signs

    Measured: 3 months timepoint

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

    Number of clinically significant changes in brain MRI findings

    Measured: 3 months timepoint

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

    Number of clinically significant changes in physical examination

    Measured: 3 months timepoint

Secondary outcomes (3)

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

    Measured: Baseline to 6 months

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

    Measured: Baseline to 6 months

  3. Change in dyskinesia rating scale score

    Measured: Baseline to 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Oct 2018
  2. Study start17 Oct 2018
  3. Primary completion12 Apr 2022
  4. Study completion12 Apr 2022

Why it 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.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03720418
Sponsor's protocol ID
OXB-102-01
Acronym
SUNRISE-PD
Status last verified
Apr 2022
First submitted
16 Oct 2018
Last updated
3 May 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

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. No abstract available.

    PubMed 31127735

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing OXB-102 and Imitation Surgical Procedure (ISP) in people with Parkinson Disease, run by Sio Gene Therapies at 3 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 3 May 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United Kingdom, France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: 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.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events (time frame: 3 months timepoint). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03720418, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2022.

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