Parkinson's Disease
Conditions
Keywords
Parkinson's disease., UCB0599, Phase 2, Minzasolmin
Brief summary
The purpose of the study is to estimate the pharmacodynamic effects of minzasolmin (UCB0599) on brain pathophysiology in Early-start versus Delayed-start participants originally diagnosed with new onset Parkinson's disease.
Interventions
DRUGMinzasolmin (UCB0599)
Minzasolmin (UCB0599) Pharmaceutical form: Granules in capsules Route of administration: Oral use Participants will receive minzasolmin (UCB0599) in a pre-specified sequence during the Treatment Period.
Sponsors
UCB Biopharma SRL
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
40 Years to 78 Years
Healthy volunteers
No
Inclusion criteria
* Participant completed the Treatment Period of PD0053 (NCT04658186). The Baseline Visit for PD0055 (Visit 2) should be no later than 4 weeks following the end of treatment (EOT) Visit in PD0053 (NCT04658186). Any delay needs to be justified by the Investigator and approved by the Sponsor * A male study participant must agree to use contraception during the Treatment Period and for at least 90 days after the last dose of the IMP and refrain from donating sperm during this period. * A female study participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies: ◦ Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 1 month after the last dose of investigational medicinal product (IMP). The study participant must have a negative urine pregnancy test at Screening (Visit 1), which is to be confirmed negative by urine testing prior to the first dose of IMP at PD0055 Baseline Visit. If oral contraception is used, an additional barrier method will be required during the study as an IMP-related gastrointestinal upset or a drug interaction by cytochrome P450 3A4 (CYP3A4) induction could interfere with efficacy * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the Informed Consent form (ICF) and in this protocol.
Exclusion criteria
* Study participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study * A female study participant who tests positive for pregnancy, plans to get pregnant during the participation in the study, or who is breastfeeding * Study participant had previously participated in PD0055 * Study participant meets any withdrawal criteria in PD0053 (NCT04658186) * Study participants wearing any kind of implantable active device, including cardiac pacemakers, pumps, and implantable cardioverters, will be excluded from using Digital Health Technology, but may participate in the main study * Study participant does not agree to refrain from donating blood or blood products or other body fluids
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Dopamine Transporter Imaging, Measured by Single Photon Emission Computed Tomography (DaT-SPECT), Whole Striatum Specific Binding Ratio up to PD0055 EOT or ET (Corresponding to a Visit Between PD0055 Month 6 and Month 18 Inclusive) | From Baseline (PD0053 Screening Visit) up to PD0055 end of treatment (EOT) or early termination (ET) (corresponding to a visit between PD0055 Month 6 and Month 18 inclusive), up to 36 months post PD0053 Screening | Change from PD0053 Baseline (screening) in mean striatum specific binding ratio (SBR) was assessed by DaT-SPECT using 123I-Ioflupane. Whole striatum was calculated as average of SBR data values for the four following "small" regions: left caudate, right caudate, left putamen, and right putamen. SBR was calculated for each region with the occipital cortex as a reference region. SBR = Average Small region minus Average Occipital region divided by Average Occipital region. Lower SBR indicated worse disease. Data were summarized by mapped visits: a DaT-SPECT within 2 months of PD0055 Screening was mapped to PD0053 Month 18; assessments after Month 23 from PD0053 Baseline were grouped as a single PD0055 EOT/ET visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cumulative Levodopa Equivalent Daily Dose (LEDD) at PD0055 Month 18 | From Baseline (PD0053 Screening Visit) to PD0055 Month 18, up to 36 months post PD0053 Screening | The Cumulative Levodopa Equivalent Daily Dose (LEDD) was calculated for each participant at each visit, based on the dose level and frequency indicated on the concomitant medication form and at the end of study. This was the sum of all the LEDDs taken up to that visit. Any changes in medication (type, dose, or dosing regimen) were accounted for when calculating cumulative doses. |
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From PD0055 Baseline (Day 0) to the Safety Follow-up Visit (PD0055 Month 31) | An adverse event (AE) was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment in PD0055. The percentage of participants data was rounded to one decimal place. |
| Percentage of Participants With Serious TEAEs | From PD0055 Baseline (Day 0) to the Safety Follow-up Visit (PD0055 Month 31) | A SAE was defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation, results in persistent disability or incapacity, is a congenital anomaly or birth defect, and other important medical events which are based on medical or scientific judgement may jeopardise the participant's life, or may require medical or surgical intervention to prevent any of the above. The percentage of participants data was rounded to one decimal place. |
| Percentage of Participants With TEAEs Leading to Withdrawal From the Study | From PD0055 Baseline (Day 0) to the Safety Follow-up Visit (PD0055 Month 31) | Percentage of participants with TEAEs leading to withdrawal from the study are presented. A TEAE was defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment in PD0055. The percentage of participants data was rounded to one decimal place. |
Countries
Canada, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
STUDY_DIRECTORUCB Cares
001 844 599 2273
Participant flow
Recruitment details
The study started to enroll participants in August 2022 and concluded in March 2025.
Pre-assignment details
Participant Flow refers to the Safety Set.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 62.0 years STANDARD_DEVIATION 8.2 |
| Age, Customized 18 to <65 years | 229 Participants |
| Age, Customized 65 to <85 years | 199 Participants |
| Age, Customized >= 85 years | 0 Participants |
| Race/Ethnicity, Customized Asian | 2 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 2 Participants |
| Race/Ethnicity, Customized Missing | 59 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 360 Participants |
| Race/Ethnicity, Customized Other/mixed | 1 Participants |
| Race/Ethnicity, Customized White | 121 Participants |
| Sex: Female, Male Female | 57 Participants |
| Sex: Female, Male Male | 79 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 1 / 153 | 0 / 136 | 0 / 139 |
| other Total, other adverse events | 70 / 153 | 56 / 136 | 42 / 139 |
| serious Total, serious adverse events | 7 / 153 | 9 / 136 | 10 / 139 |
Outcome results
None listed