Parkinson's DiseasePhase 1NCT04875962
A Study to Test the Safety, Tolerability, and Pharmacokinetics of UCB0599 in Healthy Study Participants and Patients With Parkinson's Disease (PD)
A Phase 1 study testing UCB0599 in people with Parkinson's Disease, run by UCB Biopharma S.P.R.L. at 6 sites in United States.
Trial summary
The purpose of the study is to evaluate the safety and tolerability after administration of multiple doses and the pharmacokinetics (PK) of single and multiple doses of UCB0599 in healthy study participants and participants with Parkinson's Disease (PD). Read more about this study
- Who is it for?
- Ages 40 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- UCB0599
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 31 participants
- Where
- 6 sitesUnited States
- Study dates
- 6 May 2019to 19 Feb 2020
- Phase
- Phase 1
- 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 40 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Participant must be 40 to 80 years of age inclusive, at the time of signing the informed consent
- Study participant with Parkinson's disease (PD) must have a clinical diagnosis of PD. The following diagnostic criteria must be met: Bradykinesia AND at least ONE of the following: muscular rigidity or resting tremor
- Study participant with PD must have a historic brain image (magnetic resonance imaging (MRI) or computerized tomography (CT) obtained at any point from the time of clinical diagnosis to the time of Screening that does not show any brain abnormalities that could cause symptomatic Parkinsonism
- Study participant must have a Hoehn and Yahr Stage: 1 to 3
- Study participant must be either untreated, or treated with a stable regimen (at least 4 weeks prior to Baseline Visit) of antiparkinsonian drugs and is expected to remain on this regimen for the duration of the study
- Body weight >50 kg (110 lbs) and body mass index (BMI) within the range 18 to 32 kg/m^2 (inclusive)
Who cannot join
- Study participant has a known hypersensitivity to any components of the study medication or comparative drugs as stated in this protocol
- Study participant has a known relevant allergy, a pre-existing history of a relevant allergic condition, or a predisposition for an allergic reaction (ie, total immunoglobulin E [IgE] value above normal range at Screening); this study participant's inclusion should be discussed with the Medical Monitor
- Study participant has a history of levodopa-induced motor fluctuations or dyskinesia expected to interfere with his/her ability to participate in the study
- Study participant has ongoing significant inflammatory gastrointestinal disorders and/or clinical signs of significant gastrointestinal problems at Screening
- Study participant has a historic brain scan (MRI scan or CT scan) or an MRI scan performed at Screening indicative of a clinically significant abnormality
- Study participant has a diagnosis of a significant Central nervous system (CNS) disease other than PD or history of epilepsy or seizure disorder other than febrile seizures as a child
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- Abnormalities in lumbar spine previously known or determined by a screening lumbar x-ray (if conducted)
- History of clinically significant back pain, back pathology, and/or back injury (for example, degenerative disease, spinal deformity, or spinal surgery) that may predispose participant to complications or technical difficulty with lumbar puncture
- Evidence or history of significant active bleeding or coagulation disorder or use of drugs that affect coagulation or platelet function within 14 days prior to lumbar catheter insertion
- Current or chronic history of liver disease, or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones)
- Study participant has a history or present condition of respiratory or cardiovascular disorders at Screening (eg, cardiac insufficiency, coronary heart disease, uncontrolled hypertension, arrhythmia, tachyarrhythmia, or myocardial infarction) which is considered clinically significant by the Investigator
- Study participant has medical history or current diagnosis of diabetes
- Study participant has clinical significant electrocardiogram (ECG) abnormality at Screening, in the opinion of the Investigator
- Study participant has had prior treatment with an investigational vaccine for PD (including active immunization or passive immunotherapy with monoclonal antibodies)
- Study participant has had prior surgical treatment of PD involving intracranial surgery or implantation of a device (including deep brain stimulation) or duodopa
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Up0077 107
Atlanta, Georgia, 30331
Up0077 103
Bay Harbor Islands, Florida, 33154
Up0077 105
DeLand, Florida, 32720
Up0077 101
Farmington Hills, Michigan, 48334
Up0077 102
Long Beach, California, 90806
Up0077 104
Raleigh, North Carolina, 27612
About this study
Official title: A Double-Blind, Placebo-Controlled, Multiple Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of UCB0599 in Healthy Study Participants and Patients With Parkinson's Disease (PD)
The purpose of the study is to evaluate the safety and tolerability after administration of multiple doses and the pharmacokinetics (PK) of single and multiple doses of UCB0599 in healthy study participants and participants with Parkinson's Disease (PD).
- Conditions
- Parkinson's Disease
- Being tested
- UCB0599, Placebo
- Keywords
- UCB0599, Parkinson's Disease, Phase 1 study, Healthy participants, Patients
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UCB0599Drug
Participants will receive an assigned dosage regimen of UCB0599 during the Treatment Period.
PlaceboDrug
Participants will receive an assigned dosage regimen of Placebo during the Treatment Period to maintain the blinding.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - UCB0599 | Experimental | Participants will be randomized to receive a predefined dosage of UCB0599. Receives: Drug: UCB0599 |
| Cohort 2 - UCB0599 | Experimental | Participants will be randomized to receive a predefined dosage of UCB0599. Receives: Drug: UCB0599 |
| Cohort 1 - Placebo | Placebo comparator | Participants will be randomized to receive a predefined dosage of Placebo. Receives: Drug: Placebo |
| Cohort 2 - Placebo | Placebo comparator | Participants will be randomized to receive a predefined dosage of Placebo. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Emergent Adverse Avents (TEAEs) from Baseline to End of Study visit
An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Measured: From Baseline to End of study visit (up to Week 7)
Secondary outcomes (12)
Maximum observed plasma concentration (Cmax) in healthy participants on Day 1
Cmax: Maximum observed plasma concentration
Measured: Day 1: Predose up to 12 hours post dose
Maximum observed plasma concentration (Cmax) in healthy participants on Day 28
Cmax: Maximum observed plasma concentration
Measured: Day 28: Predose up to 24 hours post dose
Time to maximum observed plasma concentration (tmax) in healthy participants on Day 1
Tmax: Time of observed Cmax
Measured: Day 1: Predose up to 12 hours post dose
Time to maximum observed plasma concentration (tmax) in healthy participants on Day 28
Tmax: Time of observed Cmax
Measured: Day 28: Predose up to 24 hours post dose
Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in healthy participants on Day 1
AUC(0-12h): Area under the curve from time 0 to 12 hours
Measured: Day 1: Predose up to 12 hours post dose
Area under the concentration-time curve for the dosing interval (AUCtau) in healthy participants on Day 28
AUCtau: Area under the concentration-time curve for the dosing interval at steady state
Measured: Day 28: Predose up to 24 hours post dose
Maximum observed plasma concentration (Cmax) in patients on Day 1
Cmax: Maximum observed plasma concentration
Measured: Day 1: Predose up to 12 hours post dose
Maximum observed plasma concentration (Cmax) in patients on Day 28
Cmax: Maximum observed plasma concentration
Measured: Day 28: Predose up to 24 hours post dose
Time to maximum observed plasma concentration (tmax) in patients on Day 1
Tmax: Time of observed Cmax
Measured: Day 1: Predose up to 12 hours post dose
Time to maximum observed plasma concentration (tmax) in patients on Day 28
Tmax: Time of observed Cmax
Measured: Day 28: Predose up to 24 hours post dose
Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in patients on Day 1
AUC(0-12h): Area under the curve from time 0 to 12 hours
Measured: Day 1: Predose up to 12 hours post dose
Area under the concentration-time curve for the dosing interval (AUCtau) in patients on Day 28
AUCtau: Area under the concentration-time curve for the dosing interval at steady state
Measured: Day 28: Predose up to 24 hours post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2021
- Study start6 May 2019
- Primary completion19 Feb 2020
- Study completion19 Feb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma S.P.R.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04875962
- Sponsor's protocol ID
- UP0077
- Status last verified
- May 2021
- First submitted
- 3 May 2021
- Last updated
- 6 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Due to the small sample size in this trial, IPD cannot be adequately anonymized i.e., there is a reasonable likelihood that individual participants could be re-identified. For this reason, data from this trial cannot be shared.
Publications
Mercier J, Bani M, Colson AO, Germani M, Lalla M, Plisson C, Huiban M, Searle G, Mathy FX, Nicholl R, Otoul C, Smit JW, van Asch V, Wagneur M, Maguire RP. Evaluation and Application of a PET Tracer in Preclinical and Phase 1 Studies to Determine the Brain Biodistribution of Minzasolmin (UCB0599). Mol Imaging Biol. 2024 Apr;26(2):310-321. doi: 10.1007/s11307-023-01878-7. Epub 2023 Dec 18.
PubMed 38110790Smit JW, Basile P, Prato MK, Detalle L, Mathy FX, Schmidt A, Lalla M, Germani M, Domange C, Biere AL, Bani M, Carson S, Genius J. Phase 1/1b Studies of UCB0599, an Oral Inhibitor of alpha-Synuclein Misfolding, Including a Randomized Study in Parkinson's Disease. Mov Disord. 2022 Oct;37(10):2045-2056. doi: 10.1002/mds.29170. Epub 2022 Aug 12.
PubMed 35959805
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled, Multiple Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of UCB0599 in Healthy Study Participants and Patients With Parkinson's Disease (PD)
- ClinicalTrials.gov ID
- NCT04875962
- Acronym / sponsor ID
- UP0077
- Conditions
- Parkinson's Disease
- Keywords
- UCB0599, Parkinson's Disease, Phase 1 study, Healthy participants, Patients
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- UCB Biopharma S.P.R.L.Industry (pharma, biotech, devices)
- Study start
- 6 May 2019 (actual)
- Primary completion
- 19 Feb 2020 (actual)
- Study completion
- 19 Feb 2020 (actual)
- First posted
- 6 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the safety and tolerability after administration of multiple doses and the pharmacokinetics (PK) of single and multiple doses of UCB0599 in healthy study participants and participants with Parkinson's Disease (PD).
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - UCB0599 | Experimental | Drug: UCB0599Participants will be randomized to receive a predefined dosage of UCB0599. |
| Cohort 2 - UCB0599 | Experimental | Drug: UCB0599Participants will be randomized to receive a predefined dosage of UCB0599. |
| Cohort 1 - Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive a predefined dosage of Placebo. |
| Cohort 2 - Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive a predefined dosage of Placebo. |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Participant must be 40 to 80 years of age inclusive, at the time of signing the informed consent
- Study participant with Parkinson's disease (PD) must have a clinical diagnosis of PD. The following diagnostic criteria must be met: Bradykinesia AND at least ONE of the following: muscular rigidity or resting tremor
- Study participant with PD must have a historic brain image (magnetic resonance imaging (MRI) or computerized tomography (CT) obtained at any point from the time of clinical diagnosis to the time of Screening that does not show any brain abnormalities that could cause symptomatic Parkinsonism
- Study participant must have a Hoehn and Yahr Stage: 1 to 3
- Study participant must be either untreated, or treated with a stable regimen (at least 4 weeks prior to Baseline Visit) of antiparkinsonian drugs and is expected to remain on this regimen for the duration of the study
Key exclusion criteria (15 listed)
- Study participant has a known hypersensitivity to any components of the study medication or comparative drugs as stated in this protocol
- Study participant has a known relevant allergy, a pre-existing history of a relevant allergic condition, or a predisposition for an allergic reaction (ie, total immunoglobulin E [IgE] value above normal range at Screening); this study participant's inclusion should be discussed with the Medical Monitor
- Study participant has a history of levodopa-induced motor fluctuations or dyskinesia expected to interfere with his/her ability to participate in the study
- Study participant has ongoing significant inflammatory gastrointestinal disorders and/or clinical signs of significant gastrointestinal problems at Screening
- Study participant has a historic brain scan (MRI scan or CT scan) or an MRI scan performed at Screening indicative of a clinically significant abnormality
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UCB0599Participants will receive an assigned dosage regimen of UCB0599 during the Treatment Period. | Drug | Cohort 1 - UCB0599, Cohort 2 - UCB0599 |
| PlaceboParticipants will receive an assigned dosage regimen of Placebo during the Treatment Period to maintain the blinding. | Drug | Cohort 1 - Placebo, Cohort 2 - Placebo |
Primary outcome
Treatment-Emergent Adverse Avents (TEAEs) from Baseline to End of Study visit
Time frame: From Baseline to End of study visit (up to Week 7)
An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
12 secondary outcomes
Maximum observed plasma concentration (Cmax) in healthy participants on Day 1
Time frame: Day 1: Predose up to 12 hours post dose
Maximum observed plasma concentration (Cmax) in healthy participants on Day 28
Time frame: Day 28: Predose up to 24 hours post dose
Time to maximum observed plasma concentration (tmax) in healthy participants on Day 1
Time frame: Day 1: Predose up to 12 hours post dose
Time to maximum observed plasma concentration (tmax) in healthy participants on Day 28
Time frame: Day 28: Predose up to 24 hours post dose
Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in healthy participants on Day 1
Time frame: Day 1: Predose up to 12 hours post dose
Area under the concentration-time curve for the dosing interval (AUCtau) in healthy participants on Day 28
Time frame: Day 28: Predose up to 24 hours post dose
Maximum observed plasma concentration (Cmax) in patients on Day 1
Time frame: Day 1: Predose up to 12 hours post dose
Maximum observed plasma concentration (Cmax) in patients on Day 28
Time frame: Day 28: Predose up to 24 hours post dose
Time to maximum observed plasma concentration (tmax) in patients on Day 1
Time frame: Day 1: Predose up to 12 hours post dose
Time to maximum observed plasma concentration (tmax) in patients on Day 28
Time frame: Day 28: Predose up to 24 hours post dose
Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in patients on Day 1
Time frame: Day 1: Predose up to 12 hours post dose
Area under the concentration-time curve for the dosing interval (AUCtau) in patients on Day 28
Time frame: Day 28: Predose up to 24 hours post dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2021.
- Derived from this study: PubMed 38110790Mercier J, Bani M, Colson AO, Germani M, Lalla M, Plisson C, Huiban M, Searle G, Mathy FX, Nicholl R, Otoul C, Smit JW, van Asch V, Wagneur M, Maguire RP. Evaluation and Application of a PET Tracer in Preclinical and Phase 1 Studies to Determine the Brain Biodistribution of Minzasolmin (UCB0599). Mol Imaging Biol. 2024 Apr;26(2):310-321. doi: 10.1007/s11307-023-01878-7. Epub 2023 Dec 18.
- Derived from this study: PubMed 35959805Smit JW, Basile P, Prato MK, Detalle L, Mathy FX, Schmidt A, Lalla M, Germani M, Domange C, Biere AL, Bani M, Carson S, Genius J. Phase 1/1b Studies of UCB0599, an Oral Inhibitor of alpha-Synuclein Misfolding, Including a Randomized Study in Parkinson's Disease. Mov Disord. 2022 Oct;37(10):2045-2056. doi: 10.1002/mds.29170. Epub 2022 Aug 12.
- Individual participant dataSharing planned: No. Due to the small sample size in this trial, IPD cannot be adequately anonymized i.e., there is a reasonable likelihood that individual participants could be re-identified. For this reason, data from this trial cannot be shared.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273 (UCB)
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing UCB0599 in people with Parkinson's Disease, run by UCB Biopharma S.P.R.L. at 6 sites in United States.
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 6 inclusion and 15 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: finished, as of the sponsor's last update on 6 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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 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.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Treatment-Emergent Adverse Avents (TEAEs) from Baseline to End of Study visit (time frame: From Baseline to End of study visit (up to Week 7)). 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 NCT04875962, 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 6 May 2021.