Clinical trial NCT04913597: A Study of Switching Avatrombopag and Rh-TPO in ITP
Official title: A Prospective Observational Study of Switching Avatrombopag and Rh-TPO in Chinese Adult Patients With Primary Immune Thrombocytopenia
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Corticosteroid-resistant or Relapsed ITP
Keywords: Immune Thrombocytopenia
Recombinant human thrombopoietin
avatrombopag
switching
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2021-06-04
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: adult ITP patients

Brief summary: Thrombopoietin receptor agonists (TPO-RAs) represent a highly effective and well-tolerated second-line ITP treatment that provides excellent responses.If there is cross-resistance between 2 drugs for the treatment of adult ITP is still unkonwn.The purpose of this study is to investigate the efficacy and safety of switching avatrombopag and rh-TPO in adults with ITP.

Detailed description: Thrombopoietin Receptor Agonists (TPO-RAs) are novel treatments for patients with chronic Primary Immune Thrombocytopenia (ITP). According to the findings of mechanism-based studies, rhTPO competes with endogenous TPO for binding to TPO-R while avatrombopag has an additive effect with endogenous TPO, indicating that the treatment mechanism and side-effect profiles could be somewhat different between these drugs. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. The purpose of this study is to investigate the efficacy and safety of switching avatrombopag and rh-TPO in adults with ITP.This is a non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to avatrombopag or vice versa will be enrolled. The reason for switch will be recorded. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.

Eligibility criteria: Inclusion Criteria:

1.18 years or older 2.Primary ITP 3.Failed initial glucocorticosteroid treatment, 4.Applying rhTPO or Eltrombopag as subsequent treatment 5.Switch from rh-TPO to eltrombopag or vice versa 6.Normal neutrophils 7.Available follow-up at least 6 weeks after switching

Exclusion Criteria:

1. HIV positive status, or active infection of HBV or HCV
2. Suffering from a serious or progressive disease, which, in the investigator's judgment, put the subject at undue risk for participation in this study (i.e. cancer or pre-cancer, immunocompromised, uncontrolled diabetes, epilepsy, severe cardio-cerebrovascular disease(s) (i.e. stroke, idiopathic aortic stenosis, aneurysm, hypertrophic obstructive cardiomyopathy, ischaemic heart disease, tachyarrhythmias, severe heart failure \[classified as NYHA III-IV\], severe lung dysfunctions, etc))
3. History of thrombosis plus two or more risk factors as defined in Caprini thrombosis risk assessment model
4. Lactating or pregnant women, or WOCBP who are unwilling to use highly effective contraceptive measures during the study period
5. Abnormal liver and renal functions: AST or ALT or total bilirubin ≥1.5 × ULN, and/or creatinine ≥176.8 μmol/L
6. Women of childbearing potential (WOCBP) that are pregnant or wish to become pregnant during the prospective phase of the study.
7. Other conditions which the investigator considers inappropriate for enrollment

Primary outcomes:
measure: Initial response after switching
timeFrame: 4 weeks
Secondary outcomes:
measure: Response rate at 12 weeks after switching
timeFrame: 12 weeks
measure: Initial response after switching according to the reasons of switching
timeFrame: 4 weeks
measure: Rate of response at 12 weeks after switching according to the reasons of switching
timeFrame: 12 weeks
measure: Time to response
timeFrame: 4 weeks
measure: Durable response
timeFrame: 24 weeks
measure: Incidence of bleeding events
timeFrame: 24 weeks
measure: Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
timeFrame: 24 weeks
measure: Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)
timeFrame: 24 weeks
measure: Safety assessment
timeFrame: 24 weeks
Other outcomes:
Locations:
Study officials:
affiliation: Peking University People's Hospital
name: Haixia Fu, MD
role: PRINCIPAL_INVESTIGATOR
References:
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