Prospective Single-Arm Clinical Trial of GO Regimen for HLH.
ConditionLymphohistiocytosis, Hemophagocytic
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age14-70
SponsorBeijing Friendship Hospital
About this trial
This study aims to investigate the efficacy and safety of Golidocitinib(GO) monotherapy in the treatment of hemophagocytic lymphohistiocytosis.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Researchers evaluate patients with newly diagnosed or refractory/relapsed HLH disease.
Definite diagnosis of HLH: molecular diagnosis consistent with pHLH, or at least 5 of the 8 criteria in the HLH-2004 diagnostic criteria.
Patients who are currently unsuitable or unable to undergo allo-HSCT.
Researchers estimate that the expected survival period exceeds one month.
Disqualifiers
Heart function above grade II(including II) (NYHA).
Pregnancy or lactating Women and reproductive-age patients who refused to use appropriate contraceptive measures during this trial.
Individuals who are allergic to GO or have a severe allergic constitution.
Active bleeding of the internal organs.
Trial design
Design model
Single group
Treatments tested in this trial
Golidocitinib
DrugAdminister the GO regimen to HLH patients who meet the inclusion criteria, specifically Golicitinib 150 mg once daily.
Treatment groups
28 Participants
are divided into 1 treatment groupGroup A: HLH patientsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Evaluation of treatment response
A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, triglyceride, blood cell count, hemophagocytosis, and level of consciousness (if CNS HLH is present). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; without blood transfusion: Neutrophils \<500/ml must increase by 100% and be \>500/ml; neutrophils 500-2000/ml must increase by 100% and return to normal.; and for patients with ALT \>400 U/L, response was defined as an ALT decrease of at least 50%. No response is defined as not meeting the criteria for partial response.
Time frame
Weeks 2, 4, 6, and 8 after the start of treatmentSecondary outcomes
1
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
An adverse event is defined as an adverse medical condition (including worsening of a pre-existing condition) that occurs in a subject or clinical trial subject after receiving medication, regardless of whether it is causally related to the investigational drug. Serious adverse events are defined as AEs that meet one or more of the following criteria during any study period: death; immediate threat to life; and hospitalization or prolongation of hospitalization.
Time frame
30 days after the last dose is administered.Other outcomes
1
Survival
Patient's HLH-related symptoms and auxiliary examination results
Time frame
1 yearSponsors and contacts
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Source ClinicalTrials.gov