An extension of the orchestra (PD0053) study to learn how safe minzasolmin is and if it works long-term in participants with Parkinson’s disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UCB Biopharma

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

28 Dec 2022 → 25 Mar 2025

Decision date (initial)

2022-11-29

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

UCB Biopharma SRL, Allée de la Recherche 60, B-1070 Brussels, Belgium

External identifiers

EU CT number

2022-500424-30-00

WHO UTN

U1111-1279-2323

ClinicalTrials.gov

NCT05543252

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Others

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 (PD)

Secondary objectives 1

1. -To estimate the pharmacodynamic effects of minzasolmin (UCB0599) on the need for symptomatic treatment (ST) in Early-start versus Delayedstart participants originally diagnosed with new onset PD -To assess the safety and tolerability of minzasolmin (UCB0599) in participants originally diagnosed with new onset PD

Conditions and MedDRA coding

Parkinson’s disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. - Participant completed the Treatment Period of PD0053. The Baseline Visit for PD0055 (Visit 2) should be no later than 4 weeks following the end of treatment (EOT) Visit in PD0053. 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 PD0055Baseline 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 1

1. - 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 - 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Baseline adjusted Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) whole striatum specific binding ratio (SBR) at PD0055 Month 18

Secondary endpoints 1

1. - Cumulative Levodopa Equivalent Daily Dose; (LEDD) at PD0055 Month 18 - Incidence of treatment-emergent adverse event (TEAEs) - Incidence of serious adverse events (SAEs) - Incidence of TEAEs leading to withdrawal from study

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UCB Biopharma

Sponsor organisation

UCB Biopharma

Address

Researchdreef 60

City

Brussels

Postcode

1070

Country

Belgium

Scientific contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Public contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Third parties 23

Locations

6 EU/EEA countries · 47 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Unexpected events 1 · Art. 53 CTR

Note: SUSARs are reported via EudraVigilance, not CTIS — events shown here are CTIS-public notifications only.

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 44 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

15 submissions — initial application plus substantial / non-substantial modifications