A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Early Parkinson's Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Clinuvel Europe Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

completed 21 Nov 2024

Decision date (initial)

2024-06-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

CLINUVEL EUROPE LIMITED

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

Evaluate the safety of afamelanotide in participants with early PD

Secondary objectives 5

1. Evaluate the changes in α-synuclein in blood in patients with early PD after treatment with afamelanotide
2. Evaluate the changes in inflammation in patients with early PD following treatment with afamelanotide
3. Evaluate the changes in neuromelanin (NM) in participants with early PD after treatment with afamelanotide
4. Evaluate the changes in cognition in participants with early PD following treatment with afamelanotide
5. Evaluate the clinical improvement in participants with early Parkinson’s disease (PD) following treatment with afamelanotide

Conditions and MedDRA coding

Parkinson's Disease

Study design 1 period

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Patient diagnosed with PD less than three years prior to first screening visit and not yet requiring dopaminergic medication nor expected to within three months of enrolment.
2. 40-85 years old

Exclusion criteria 11

1. History of drug abuse, licit or illicit.
2. Heavy alcohol consumption.
3. Current cigarette smokers.
4. Any personal or immediate family history of melanoma or personal history of dysplastic nevus syndrome
5. Known allergy or anaphylaxis to adrenocorticotropic hormone (ACTH) or melanocortins
6. Any evidence of hepatic insufficiency or renal impairment.
7. Female who is pregnant or lactating.
8. Females of child-bearing potential not using highly effective contraceptive measures, for up to three months after the last injection administration.
9. Sexually active men with a partner of child-bearing potential who is not using highly effective contraceptive measures.
10. Participation in any clinical intervention study during the six weeks before the study screening period.
11. Any medical condition or illness in the four weeks before the study screening which may interfere with the study protocol or not suitable for study participation in the opinion of the Investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Assessment of Treatment Emergent Adverse Events including clinically significant changes in laboratory results

Secondary endpoints 5

1. Changes in α-synuclein levels in plasma from baseline to Day 56±2
2. Changes in inflammation biomarker in plasma from baseline to Day 56±2
3. Changes in neuromelanin in the MRI series from screening to Day 56±2
4. Changes in cognition from baseline to Day 56±2
5. Changes in clinical improvement baseline to Day 56±2

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Clinuvel Europe Limited

Sponsor organisation

Clinuvel Europe Limited

Address

Saint Kevins, 10 Earlsfort Terrace 10 Earlsfort Terrace

City

Dublin 2

Postcode

D02 T380

Country

Ireland

Scientific contact point

Organisation

Clinuvel Europe Limited

Contact name

Information

Public contact point

Organisation

Clinuvel Europe Limited

Contact name

Information

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

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 2 files

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

Application history

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