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ParkinsonPhase 2/3GREATNCT05830396

GRoningen Early-PD Ambroxol Treatment

A Phase 2/3 study testing Ambroxol Hydrochloride in people with Parkinson and Parkinson Disease, run by University Medical Center Groningen at 1 site in Netherlands.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

The most common genetic risk factor for Parkinson's Disease is a heterozygous mutation of the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase). Reduced GCase activity is associated with aggregation of the protein alpha synucleine (aSyn) in the central nervous system, which is related to the… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Parkinson, Parkinson Disease
What is being tested?
Ambroxol Hydrochloride
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
80 participants
Where
1 siteNetherlands
Study dates
May 2023to Jul 2025
Phase
Phase 2/3
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of Parkinson's disease, according to Movement Disorders Society (MDS) criteria (27)
  • Disease duration of 10 years or less at time of inclusion
  • PD patients carrying a GBA1 mutation
  • Able to write written informed consent, understanding study protocol and perform protocol related actions
  • Willing and able to self-administer oral ambroxol or placebo medication

Who cannot join

  • The refusal to be informed about an unforeseen clinical finding
  • Use of an implanted Deep Brain Stimulation (DBS) system
  • Confirmed dysphagia that would preclude self-administration of ambroxol or placebo tablets
  • History of known sensitivity to the study medication
  • Pregnant or breastfeeding women
  • Participants of childbearing potential that would not use adequate birth control, consisting of a negative pregnancy test at the screening visit and use of accepted contraceptive methods defined as highly effective while participating in the study
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  • 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. Such conditions may include:
  • Impaired renal function (a positive urine dipstick test, and laboratory values below or above: a eGFR <45 ml/min 1,73M2, Sodium 135-145 mmol/L, Potassium 3.5-5.0 mmol/L, Urea 2.5-7.5mmol/L).
  • Moderate/severe hepatic impairment (laboratory values below or above: ASAT 0- 80U/L, ALAT0-90 U/L, GGT > 80 U/L, Alkaline Phosphatase 35-210 U/L).

Trial locations and contacts

1 site in 1 country.

Olav Siemeling contact

0031 (0)50 3615639

o.siemeling@umcg.nl

Netherlands1 site, 1 recruiting

  • University Medical Center Groningen

    Groningen

    Olav Siemeling, 0031 (0)50 3615639, o.siemeling@umcg.nl

    Teus van Laar, Prof. dr.

    Recruiting

Other trials in Netherlands

About this study

Official title: GRoningen Early-PD Ambroxol Treatment (GREAT) Trial: A Randomised, Double-blind, Placebo-controlled, Singlecenter Trial With Ambroxol in Parkinson Patients With a GBA Mutation

The most common genetic risk factor for Parkinson's Disease is a heterozygous mutation of the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase). Reduced GCase activity is associated with aggregation of the protein alpha synucleine (aSyn) in the central nervous system, which is related to the pathological cause of PD. Ambroxol is a mucolytic expectorant that appears to facilitate the refolding of the misfolded GBA protein thats acts as a chaperone for GCase.

This randomized placebo-controlled trial aims to investigate the disease-modifying properties of ambroxol in PD patients with a GBA1-mutation. Patients will undergo motor and cognitive tests, as well as imaging and blood tests.

Being tested
Ambroxol Hydrochloride, Placebo
Keywords
Ambroxol, GBA1, GBA
Medical subject headings
Parkinson Disease

What participants receive

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

  • Ambroxol HydrochlorideDrug

    Patients will either receive ambroxol or placebo. ambroxol will be given intially in a dosage of 600mg/day. After 1 week, this will be increased to 1200mg/day. After 2 weeks the maximum dosage of 1800mg/day will be given. In total, ambroxol will be administered for 48 weeks. This is followed by a 12 week washout period, after wich the final outcomes will be measured (week 60).

  • PlaceboDrug

    Patients will either receive ambroxol or placebo. ambroxol will be given intially in a dosage of 600mg/day. After 1 week, this will be increased to 1200mg/day. After 2 weeks the maximum dosage of 1800mg/day will be given. In total, ambroxol will be administered for 48 weeks. This is followed by a 12 week washout period, after wich the final outcomes will be measured (week 60).

Study groups in NCT05830396
Study groupRoleWhat they receive
AmbroxolExperimentalAmbroxol 1800mg/day
Receives: Drug: Ambroxol Hydrochloride
PlaceboPlacebo comparatorPlacebo
Receives: Drug: Placebo

What is being measured

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

  1. MDS-UPDRS3 motor scale

    Motor scale developed for PD patients, 0-132. 0 means good performance, 132 means very bad performance

    Measured: 60 weeks

Secondary outcomes (7)

  1. Safety and tolerability measured by incidence of adverse events and possible side effects

    AE will be monitored and patients will be questioned about side effects every week during the first 3 weeks and after that, every 3 months during the visits

    Measured: all throughout the study. specifically at: 1, 2, 3, 12, 24, 36, 48, 60 weeks

  2. Glucocerebrosidase (GCase) activity in blood mononuclear cells

    Measured by the level of sphingolipids in PBMCs

    Measured: 0, 12, 60 weeks

  3. Striatal F-DOPA uptake as measured by [18] F-DOPA PET scan

    Measured: 0, 60 weeks

  4. fMRI resting state to investigate the functional architecture and structural MRI for PET-scan

    Fluctuations in the BOLD signal can be used to investigate the functional architecture and connectivity within the brain.

    Measured: 0, 60 weeks

  5. Quality of Life (PDQ-39 questionnaire)

    Measured: 0, 60 weeks

  6. Non Motor Symptoms (NMSS scale)

    minimum value is 0, maximum value is 360. 0 indicating a good performance, 360 indicating a very bad performance

    Measured: 0, 60 weeks

  7. Cognition, using the Montreal Cognitive Assessment (MoCA)

    Range is 0-30, 0 indicating the worst performance, 30 indicating the best performance

    Measured: 0, 60 weeks

Timeline

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

  1. First posted26 Apr 2023
  2. Study startMay 2023 (estimated)
  3. Primary completionJul 2025 (estimated)
  4. Study completionJul 2025 (estimated)

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
80 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05830396
Sponsor's protocol ID
202100266
Acronym
GREAT
Status last verified
May 2023
First submitted
20 Mar 2023
Last updated
10 May 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Moes HR, Buskens E, van Laar T. Continuous subcutaneous levodopa-carbidopa infusion for Parkinson's disease. Lancet Neurol. 2024 Sep;23(9):856-857. doi: 10.1016/S1474-4422(24)00272-2. No abstract available.

    PubMed 39152017

  • Siemeling O, Slingerland S, van der Zee S, van Laar T. Study protocol of the GRoningen early-PD Ambroxol treatment (GREAT) trial: a randomized, double-blind, placebo-controlled, single center trial with ambroxol in Parkinson patients with a GBA mutation. BMC Neurol. 2024 May 1;24(1):146. doi: 10.1186/s12883-024-03629-9.

    PubMed 38693511

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Ambroxol Hydrochloride in people with Parkinson and Parkinson Disease, run by University Medical Center Groningen at 1 site in Netherlands.

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 5 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 10 May 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Netherlands. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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

Are results available for this study?

Results are not available in the source record.

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

Contact Olav Siemeling (0031 (0)50 3615639, o.siemeling@umcg.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

MDS-UPDRS3 motor scale (time frame: 60 weeks). 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 NCT05830396, which also lists 2 linked publications.

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 10 May 2023.

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