Evaluation of the efficacy and safety of drug treatment in familial polycythemia associated with an EPO gene mutation: a multicentre, open-label, phase I trial

2025-521906-16-00 Protocol ESPOiR Human pharmacology (Phase I) - Other Ongoing, recruitment ended

Start 1 Aug 2025 · Status Ongoing, recruitment ended · 1 EU/EEA countries · 2 sites · Protocol ESPOiR

Overview

Sponsor-declared trial summary

Phase Human pharmacology (Phase I) - Other
Status Ongoing, recruitment ended
Participants planned 4
Countries 1
Sites 2

familial polycythemia associated with an EPO gene mutation

Key facts

Sponsor
Centre Hospitalier Universitaire De Dijon
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Hemic and Lymphatic Diseases [C15]
Trial duration
1 Aug 2025 → ongoing
Decision date (initial)
2025-07-08
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
No

Trial design

CTIS Part I — objectives, methods, condition coding

Conditions and MedDRA coding

familial polycythemia associated with an EPO gene mutation

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Dijon

Sponsor organisation
Centre Hospitalier Universitaire De Dijon
Address
2 Boulevard Mal De Lattre De Tassigny
City
Dijon
Postcode
21000
Country
France

Scientific contact point

Organisation
Centre Hospitalier Universitaire De Dijon
Contact name
Chef de projets

Public contact point

Organisation
Centre Hospitalier Universitaire De Dijon
Contact name
Chef de projets

Locations

1 EU/EEA country · 2 investigational sites

By country

CountryMS statusPlanned subjectsSites
France Ongoing, recruitment ended 4 2
Rest of world 0

Investigational sites

France

2 sites · Ongoing, recruitment ended
Assistance Publique Hopitaux De Paris
Hématologie, 125 Rue De Stalingrad, 93009, Bobigny Cedex
Centre Hospitalier de Macon
Médecine Interne, Boulevard Louis Escande, 71000, Macon

Country notifications

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

Country Trial startTrial end Recruitment startRecruitment end Early termination
France 2025-08-01 2025-08-05 2026-03-12

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-04-30 France Acceptable
2025-07-08
2025-07-08
2 SUBSTANTIAL MODIFICATION SM-1 2026-04-14 France Acceptable
2026-04-30
2026-04-30