A Study to Evaluate the Pharmacokinetics of Afamelanotide in Patients with Erythropoietic Protoporphyria (EPP).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Clinuvel Europe Limited

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

7 Mar 2024 → 20 Jan 2025

Decision date (initial)

2024-02-20

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

CLINUVEL EUROPE LIMITED

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety

To determine the pharmacokinetics of afamelanotide following administration of afamelanotide to adolescent and adult EPP patients.
To determine the relative comparability of the PK profiles of afamelanotide in adolescent and adult EPP patients.

Secondary objectives 1

1. To determine the safety and tolerability of afamelanotide in adolescent and adult EPP patients.

Conditions and MedDRA coding

Erythropoietic Protoporphyria (EPP)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Fourteen adult EPP adult patients aged between 18 and 70 years (inclusive) and fourteen EPP adolescents aged between 12 and 17 (inclusive).
2. Agree not to use any medications
3. Able to understand and sign the written Informed Consent Form ((and parents or legal representative in case of adolescent patients)
4. Able and willing to follow the Protocol requirements

Exclusion criteria 16

1. Any personal or direct family history of melanoma.
2. Any significant history of allergy and/or sensitivity to any of the contents of study drug product.
3. Any significant history of allergy and/or sensitivity to lignocaine or other local anaesthetics if used.
4. Any significant illness during the four weeks before the study screening period.
5. Any evidence of hepatic or renal impairment.
6. Any contraindication to blood sampling.
7. Female who is pregnant or lactating.
8. Females of child-bearing potential not using adequate contraceptive measures or a life-style excluding pregnancy, for up to three months after the implant administration.
9. Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures.
10. Any factor that may interfere with the skin reflectance measurements.
11. Current cigarette smokers.
12. History of drug abuse, licit or illicit.
13. Regularly drinks more than four standard drinks of alcohol per day.
14. Participation in any clinical trial during the 60 days before the study screening period.
15. Has received afamelanotide in the last 60 days.
16. Has donated 400 mL or more of blood or had significant blood loss during the eight weeks preceding screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Cmax
2. AUC(0-t)

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

Head of Clinical Operations

Public contact point

Organisation

Clinuvel Europe Limited

Contact name

Head of Clinical Operations

Locations

2 EU/EEA countries · 2 investigational sites

By country

Investigational sites

Country notifications

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

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 29 files

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

Application history

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