Clinical trial NCT04326348: A Study of TQ05105 Tablets in Subjects With Hemophagocytic Lymphohistiocytosis（HLH）
Official title: A Single-arm, Open-label, Phase I Study of TQ05105 Tablets in Subjects With Hemophagocytic Lymphohistiocytosis.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Hemophagocytic Lymphohistiocytosis
Lead sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2020-03-30
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a phase I study of TQ05105 tablets in subjects with Hemophagocytic Lymphohistiocytosis.TQ05105 is a JAK2 inhibitors and can be used to treat JAK2 target-related diseases. The activation of the JAK/STAT pathway is related to abnormal proliferation, obstruction of apoptosis, and differentiation disorder of leukemia cells which is caused by genetic abnormalities and viral infection.


Eligibility criteria: Inclusion Criteria:

* 1.18 years and older; Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 3; Life expectancy ≥ 3 months.

  2. Diagnosed as new or relapsed HLH according to the HLH-2004 criteria. 3.Has received other treatments for HLH before two weeks ago, surgery before four weeks ago.

  4. Adequate laboratory indicators. 5. No pregnant or breastfeeding women, and a negative pregnancy test. 6. Understood and signed an informed consent form.

Exclusion Criteria:

* 1. Has diagnosed and/or treated additional malignancy within 5 years prior to randomization. With exception of non-melanoma, skin cancer, and carcinoma in situ.

  2. Has participated in other anticancer drug clinical trials within 4 weeks. 3. Has multiple factors affecting oral medication. 4. Has drug abuse history that unable to abstain from or mental disorders. 5. Has active hepatitis B or C, or immunodeficiency. 6. Diagnosed as hepatic failure. 7. Has arteriovenous thrombosis events within 4 weeks. 8. Has active bleeding, or a persistent decrease in hemoglobin. 9. Has received long-term and large-dose glucocorticoids or other immunosuppressive drugs within 28 days before signed the informed consent form.

  10. Has uncontrolled cardiovascular disease. 11. Has uncontrolled hypertension. 12. Has serious systemic infections. 13. According to the judgement of the researchers, there are other factors that subjects are not suitable for the study.

Primary outcomes:
measure: Dose Limiting Toxicity (DLT)
timeFrame: Baseline up to 28 days
measure: Maximal tolerance dose (MTD)
timeFrame: Baseline up to 28 days
Secondary outcomes:
measure: Cmax
timeFrame: Hour 0, 5, 15, 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, 11, 24 hours post-dose on single dose; Hour 0 of day 1,day 3,day5, day 6 on multiple dose and hour0, 5, 15, 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, 11 hours post-dose on multiple dose of day 28.
measure: Tmax
timeFrame: Hour 0, 5, 15, 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, 11, 24 hours post-dose on single dose; Hour 0 of day 1,day 3,day5, day 6 on multiple dose and hour0, 5, 15, 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, 11 hours post-dose on multiple dose of day 28.
measure: AUC0-t
timeFrame: Hour 0, 5, 15, 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, 11, 24 hours post-dose on single dose; Hour 0 of day 1,day 3,day5, day 6 on multiple dose and hour0, 5, 15, 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, 11 hours post-dose on multiple dose of day 28.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Friendship Hospital, Capital Medical University
state: Beijing
Study officials:
References: