Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
80
Countries
1
Sites
1
Parkinson's disease
To investigate the disease modifying properties of ambroxol in PD patients with a GBA1-mutation
Key facts
- Sponsor
- Universitair Medisch Centrum Groningen
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 23 Sep 2024 → ongoing
- Decision date (initial)
- 2024-09-23
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-514976-41-00
- EudraCT number
- 2021-001833-38
- ClinicalTrials.gov
- NCT05830396
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
To investigate the disease modifying properties of ambroxol in PD patients with a GBA1-mutation
Conditions and MedDRA coding
Parkinson's disease
Regulatory references
- Scientific advice from competent authorities
- METC Universitair Medisch Centrum Groningen
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Diagnosis of Parkinson's Disease according to Movement Disorders Society (MDS) criteria
- GBA1-mutation
- Disease duration of PD < 10 years at time of inclusion
- Able to write written informed consent, understanding study protocol and perform protocol related actions
- Willingness and ability to self-administer oral ambroxol or placebo medication
Exclusion criteria 6
- The refusal to be informed about an unforeseen clinical finding
- Use of an implanted Deep Brain Stimulation (DBS) system
- History of known sensitivity to the study medication
- Pregnant or breastfeeding women
- MRI incompatible implants in the body
- Any clinically significant or unstable medical or surgical condition that in the opinion of the principal investigator may put the participant at risk when participating in the study or may influence the results of the study or affect the participant’s ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG), or laboratory tests.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Comparison of MDS-UPDRS part III motor subscale in the practically defined OFF medication state at 60 weeks
Secondary endpoints 7
- Safety and tolerability by examination of adverse events
- F-DOPA PET
- MRI
- Montreal Cognitive Assessment (MoCA)
- Non-Motor Symptoms scale (NMSS)
- Parkinson’s Disease Quality of life questionnaire with 39 items (PDQ-39)
- GCase activity measuring sphinglolipids in PBMCs and plasma
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
SCP101864681 · ATC
- Active substance
- Ambroxol Hydrochloride
- Substance synonyms
- TRANS-4-[(2-AMINO-3,5-DIBROMOBENZYL) AMINO] CYCLOHEXANOL HYDROCHLORIDE
- Route of administration
- ORAL
- Max daily dose
- 1800 mg milligram(s)
- Max total dose
- 605 g gram(s)
- Max treatment duration
- 48 Week(s)
- Authorisation status
- Authorised
- ATC code
- R05CB06 — AMBROXOL
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Capsules of 600mg instead of lozenges
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Universitair Medisch Centrum Groningen
- Sponsor organisation
- Universitair Medisch Centrum Groningen
- Address
- Hanzeplein 1
- City
- Groningen
- Postcode
- 9713 GZ
- Country
- Netherlands
Scientific contact point
- Organisation
- Universitair Medisch Centrum Groningen
- Contact name
- T. van Laar
Public contact point
- Organisation
- Universitair Medisch Centrum Groningen
- Contact name
- T. van Laar
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Ongoing, recruiting | 80 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Universitair Medisch Centrum Groningen
Neurology, Hanzeplein 1, 9713 GZ, Groningen
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Netherlands | 2024-09-23 | 2024-09-23 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 4 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-514976-41-00 | 4 |
| Recruitment arrangements (for publication) | Document is not required for transitional trial | 1 |
| Subject information and informed consent form (for publication) | L1_ Subject information + ICF GBA screening | 2 |
| Subject information and informed consent form (for publication) | L1_Patient information letter main study | 4 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-03 | Netherlands | Acceptable with conditions 2024-09-23
|
2024-09-23 |