Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Se… (NCT07297550) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia
42 participantsStarted 2025-12-15
Plain-language summary
This study is designed as a Phase Ib/II trial. The phase Ib cohort will enroll patients with severe aplastic anemia (SAA) who have failed to respond to intensified immunosuppressive therapy. In contrast, the phase II cohort will include newly diagnosed and treatment-naïve patients with SAA. A Safety Review Committee (SRC), chaired by the principal investigators, will be established to oversee patient safety throughout the study. Suppose the Phase Ib results demonstrate acceptable safety and tolerability. In that case, the data will be submitted to the Ethics Committee for review, and, upon approval, the study will advance to Phase II.
Phase Ib uses a 3+3 dose-escalation design with two cohorts: 150 mg golidocitinib orally every other day (low dose) or once daily (high dose). Phase II is a single-arm trial with Simon's two-stage optimal design.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Phase Ib:
Inclusion Criteria:
* According to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and the Guidelines for the diagnosis and management of adult aplastic anaemia: A British Society for Haematology Guideline (Br J Haematol. 2024; 204: 784-804), the patient is diagnosed with primary acquired AA and meets the diagnostic criteria for SAA.
* Subjects received intensive immunosuppressive therapy combined with a standard-dose TPO receptor agonist for at least 4 months but failed to respond or experienced relapse.
* Unsuitable for or unwilling to undergo allogeneic hematopoietic stem cell transplantation and lacking other superior treatment options.
* Age ≥ 18 years
* ECOG performance status ≤2
* Willing and able to comply with the requirements for this study and written informed consent.
Exclusion Criteria:
* Presence of any other primary or secondary bone marrow failure syndrome, including but not limited to Fanconi anemia, myelodysplastic syndrome (MDS), clonal cytopenia of undetermined significance, or large granular lymphocytic leukemia.
* Bone marrow reticulin fibrosis ≥ grade 2
* Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50% or with evidence of active hemolysis.
* Presence of clonal cytogenetic abnormalities characteristic of MDS, excluding +8, del(20q), and -Y.
* Active infection within 2 weeks before first dosing of the investigational product, including bacterial, viral, or fungal infections (comm…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence and severity of adverse events
Timeframe: Within 12 weeks
2
Percentage of patients with complete hematological response
Timeframe: Within 3 months post treatment
Trial details
NCT IDNCT07297550
SponsorInstitute of Hematology & Blood Diseases Hospital, China