A Phase 1 Study to Assess STP938 as a Monotherapy in Adults With High Risk Essential Thrombocythaemia
NCT ID: NCT06786234
Last Updated: 2026-01-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1
20 participants
INTERVENTIONAL
2025-06-18
2027-12-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
* 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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia
NCT01214915
A First in HumanTrial to Evaluate The Safety, Tolerability, And Pharmacokinetics Of PF-06755347
NCT03275740
The Tolerability，Safety，PK/PD Study of rhTPO in the Patients With Liver Function Impairment
NCT03673215
Anagrelide Retard in Essential Thrombocythemia
NCT02076815
Anagrelide Retard vs. Placebo: Efficacy and Safety in "At-risk" Patients With Essential Thrombocythaemia
NCT01230775
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
STP938 is a new class of drug that inhibits the enzyme cytidine triphosphate synthase 1 (CTPS1). Inhibition of CTPS1 is a novel way of lowering the platelet count. This study is a phase 1b, open-label, multicentre trial. Participants will receive STP938 capsules every day, in cycles of 28 days, for approximately 12 months. Participants may continue to receive study drug for a longer period, so long as it is controlling the platelet count and not causing side effects. During the study, participants will visit the study site about 26 times (2 times per cycle) over an estimated 12 months. Once the treatment is complete, safety follow-up visit(s) will occur to make sure the participant is not experiencing any adverse effects. The following study procedures will be performed: (a) physical examinations (b) ECGs (c) blood tests, (d) urine tests (e) CT/MRI scans (f) bone marrow biopsies (g) drug administration (h) study drug blood level tests and (i) gene testing.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Phase 1b
Up to 5 dose levels with STP938 administered as oral therapy
STP938
At enrolment all patients will be assigned to a single dose level of STP938 for 4 weeks. After 4 weeks the dose level may be adjusted as needed by the Investigator.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
STP938
At enrolment all patients will be assigned to a single dose level of STP938 for 4 weeks. After 4 weeks the dose level may be adjusted as needed by the Investigator.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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.
Exclusion Criteria
* 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).
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Step Pharma, SAS
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Maureen Higgins
Role: STUDY_DIRECTOR
Step Pharma
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
CHU Brest
Brest, , France
Institut Paoli-Calmettes
Marseille, , France
CHU Nantes
Nantes, , France
CHU Nice
Nice, , France
CHU Nîmes
Nîmes, , France
Hôpital Saint-Louis
Paris, , France
Gustave Roussy
Villejuif, , France
Royal Hallamshire Hospital
Sheffield, South Yorkshire, United Kingdom
University Hospital of Wales
Cardiff, , United Kingdom
Imperial College London / Hammersmith Hospital
London, , United Kingdom
Sarah Cannon Research Institute
London, , United Kingdom
Cancer and Haematology Centre, Churchill Hospital
Oxford, , United Kingdom
University of Southampton Hospital
Southampton, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
STP938-301
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.