European randomised clinical trial on mPOX Infection (EPOXI)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

University Medical Center Utrecht

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Virus Diseases [C02]

Trial duration

8 Aug 2024 → 14 Jul 2025

Decision date (initial)

2022-12-20

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

European Commission

External identifiers

EU CT number

2022-501979-10-00

ClinicalTrials.gov

NCT06156566

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

The trial aims to provide evidence about the clinical efficacy, as assessed by time to resolution of cutaneous and mucosal lesions, of a treatment versus control group of patients with proven mPOX.

Secondary objectives 2

1. The trial aims to provide evidence about the clinical efficacy of a treatment versus control in patients with proven mPOX, as assessed by duration of symptoms, complications of infection, clinical severity, and mortality.
2. The trial aims to evaluate the safety of treatment relative to control in patients with proven mPOX.

Conditions and MedDRA coding

Monkeypox

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. PCR/NAAT-confirmed mPOX infection
2. The presence of active skin or mucosal lesion(s)
3. Signed informed consent

Exclusion criteria 4

1. Age <18 years
2. Pregnant and breastfeeding patients are not eligible for inclusion in this study
3. Lack of mental capacity to provide informed consent
4. Trial participation is considered not in the best interest of patient

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Time (days) to complete lesion resolution, counted from start of therapy, assessed at day 28 after randomisation and defined as the first day on which all lesions are completely healed with a new fresh layer of skin. For skin lesions, typically this means the lesion has scabbed, desquamated and new layer of skin formed (if a scar present, this is still defined as complete lesion resolution). For mucosal lesions, the phase of scabbing and desquamation is absent, and healing with new layer of skin

Secondary endpoints 10

1. Time (days) to active lesion resolution, defined as the first day on which all skin lesions are scabbed or desquamated (and mucosal lesions healed), with follow-up up to 28 days after randomisation.
2. Status of the lesions on day 7, 14, 21 and 28 according to an ordinal scale. The ordinal scale is a) all lesions completely resolved (all scabs dropped off and intact skin remains underneath, and all mucosal lesions healed), b) active lesions resolved (all skin lesions scabbed or desquamated, but not fully resolved), c) active lesions persist but no new lesions in last 24 hours, d) new lesion(s) in last 24 hours.
3. Time to resolution of symptoms. Symptoms are assessed by self-assessment and include fatigue, malaise, nausea, vomiting, abdominal pain, anorexia, cough, dysphagia, odynophagia, fever, headache, oral pain, pain with urination, rectal/anal pain. Signs will be evaluated at study visits only, including lymphadenopathy and proctitis, and are not included in the evaluation of symptoms.
4. Occurrence of a negative monkeypox PCR of skin or mucosa swab, assessed for the most active skin or mucosa lesion at days 7, 14 and 28.
5. Persistence of scars and skin discoloration, assessed on day 90 (and day 60 if the visit is live).
6. Change from baseline in quality of life on day 7, 14, 28 and 90 by the Dermatology Quality of Life Index (DQLI)
7. All-cause mortality within 28 days and within 90 days, applicable to all patients
8. Time to complication, all-cause admission to hospital or all-cause death, within 28 days and 90 days, applicable to outpatients only.
9. Frequency of AEs, SAEs and SUSARs for the specific therapeutic, within the first 28 days, but also assessed during the total follow-up (up to day 90)
10. In a subset of patients with pain at lesion site (or proctitis) at baseline: Resolution of pain, by measuring (a) time to resolution of pain assessed by the Numeric Rating Scale (NRS) for pain (Karcioglu 2018), (b) time to cessation of the use of analgesic medication, defined as time to consistently reporting no use of analgesia for monkeypox-related lesions, up to 90 days after randomisation. (c) anal pain on days 7, 14, 28, 60 and 90 assessed by the Health Related Symptom Index

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

University Medical Center Utrecht

Sponsor organisation

University Medical Center Utrecht

Address

Heidelberglaan 100

City

Utrecht

Postcode

3584 CX

Country

Netherlands

Scientific contact point

Organisation

University Medical Center Utrecht

Contact name

Miquel Ekkelenkamp

Public contact point

Organisation

University Medical Center Utrecht

Contact name

Miquel Ekkelenkamp

Third parties 10

Locations

8 EU/EEA countries · 13 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

Corrective measures 1 · Art. 77 CTR

Results and documents

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

Documents 48 files

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

Application history

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