Informations générales (source: ClinicalTrials.gov)
An Open-Label, Phase 2 Study to Evaluate Safety, Tolerability & Preliminary Activity of the CTPS1 Inhibitor STP938 in Adult Subjects With High Risk Essential Thrombocythaemia Who Are Resistant to or Intolerant of Hydroxycarbamide Therapy
Interventional
Phase 2
Step Pharma, SAS (Voir sur ClinicalTrials)
juin 2025
septembre 2028
17 juin 2026
The goal of this clinical trial is to learn if the drug STP938 works to treat adults with
high risk essential thrombocythaemia (ET) who are resistant to, or intolerant of,
hydroxycarbamide (also known as hydroxyurea) therapy. The trial will also learn about the
safety of STP938. The main questions the trial aims to answer are:
- Does STP938 control platelet counts
- Does STP938 control platelet counts without inducing unwanted side effects
Participants will:
- Take STP938 every day for up to 12 months.
- Visit the clinic once every week for the first month, then every 2 weeks for
checkups and tests.
- Complete a questionnaire about symptoms once a month.
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CLCC INSTITUT GUSTAVE ROUSSY | Florence PASQUIER | 18/06/2026 15:05:07 | Contacter | ||
| Les établissements hors Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| CHU Nîmes - Nîmes - France | Contact (sur clinicalTrials) | ||||
| Hôpital de la Milétrie, CHU - 86021 - Poitiers - New Aquitaine - France | Contact (sur clinicalTrials) | ||||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| CHU Brest - Brest - France | Contact (sur clinicalTrials) | ||||
| CHU Nantes - Nantes - France | Contact (sur clinicalTrials) | ||||
| CHU Nice - Nice - France | Contact (sur clinicalTrials) | ||||
| Gustave Roussy - Villejuif - France | Contact (sur clinicalTrials) | ||||
| Hôpital Saint-Louis - Paris - France | Contact (sur clinicalTrials) | ||||
| Institut Paoli-Calmettes - Marseille - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
- Adults aged 18 years or older
- A confirmed diagnosis of ET according to World Health Organisation (WHO) or British
Society for Haematology (BSH) criteria.
- Meeting criteria for high risk ET.
- Raised platelet count (>600 x109/L) requiring cytoreductive therapy.
- Neutrophil count >1.0 x109/L.
- Either intolerant of or resistant to hydroxycarbamide having met any one of the
criteria at any point in their disease whilst receiving hydroxycarbamide.
- Adequate hepatic function.
- Adequate blood coagulation parameters.
- Eastern Cooperative Oncology Group (ECOG) status of 0-2.
Key Exclusion Criteria:
- Subjects currently or recently receiving cytoreductive therapy for the treatment of
essential thrombocythaemia (wash out periods apply).
- Subjects who have received any investigational agents for the treatment of essential
thrombocythaemia within 4 weeks before enrolment or less than 5 half-lives since
completion of prior therapy, whichever is shorter.
- Proven or suspected transformation to polycythaemia vera, myelofibrosis,
myelodysplasia, acute myeloid leukaemia or another myeloid neoplasm.
- Known malabsorption syndrome or other condition that may impair absorption of study
medication (e.g. gastrectomy).
- Previous splenectomy.
- Any uncontrolled intercurrent illness.
- Immunocompromised subjects with increased risk of opportunistic infections.
- Known active or chronic hepatitis B or active hepatitis C virus (HCV) infection.
- Pregnant females, breastfeeding females, and women of child bearing age or males not
willing to comply with contraceptive requirements.
- Known hypersensitivity to any of the excipients used in the formulation of the study
drug.
- Corrected QT interval >470 msec based on averaged triplicate electrocardiogram (ECG)
readings at the Screening Visit using the QT interval corrected for heart rate using
Fridericia's method (QTcF).