Clinical trial NCT04368598: The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed ITP
Official title: The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed Immune Thrombocytopenia: A Single-arm, Open-label Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: immune thrombocytopenia
acetylcysteine
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2020-04-30
Enrollment: 44
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: A single-arm, open-label study to evaluate the efficacy and safety of Acetylcysteine plus high-dose Dexamethasone in adults newly diagnosed with primary immune thrombocytopenia (ITP).

Detailed description: This is a prospective, open-label, Phase II, single-arm interventional trial performed in a single center in China. The investigators explore the efficacy and safety of Acetylcysteine plus high-dose Dexamethasone in adults newly diagnosed with primary immune thrombocytopenia.

Eligibility criteria: Inclusion Criteria:

1. Clinically confirmed immune thrombocytopenic purpura (ITP) newly diagnosed
2. Platelet count less than 30×10\^9/L on two occasions or Platelets above 30×10\^9/L combined with bleeding manifestation (WHO bleeding scale 2 or above)
3. Subject is ≥ 18 years and ≤80years
4. Subject has signed and dated written informed consent.
5. Fertile patients must use effective contraception during treatment and observational period
6. Negative pregnancy test

Exclusion Criteria:

1. Have an impaired renal function as indicated by a serum creatinine level \> 2.0 mg/dL
2. Have an inadequate liver function as indicated by a total bilirubin level \> 2.0 mg/dL and/or an aspartate aminotransaminase or alanine aminotransferase level \> 3×upper limit of normal
3. Have a New York Heart Classification III or IV heart disease
4. Have a history of severe psychiatric disorder or are unable to comply with study and follow-up procedures
5. Have active hepatitis B or hepatitis C infection
6. Have a HIV infection
7. Have active infection requiring antibiotic therapy within 7 days prior to study entry
8. Are pregnant or lactating women, or plan to become pregnant or impregnated within 12 months of receiving study drug
9. Previous treatment with rituximab
10. Previous splenectomy
11. Had previous or concomitant malignant disease
12. Not willing to participate in the study.
13. Expected survival of \< 2 years
14. Intolerant to murine antibodies
15. Immunosuppressive treatment within the last month
16. Connective tissue disease
17. Autoimmune hemolytic anemia
18. Patients currently involved in another clinical trial with evaluation of drug treatment

Primary outcomes:
measure: Number of patients with durable response after 6 months
timeFrame: 6 months
Secondary outcomes:
measure: Early response
timeFrame: 7 days
measure: Initial response
timeFrame: 1 month
measure: Bleeding
timeFrame: 1 year
measure: DOR
timeFrame: 1 year
measure: Relapse
timeFrame: 1 year
measure: Adverse events
timeFrame: 1 year
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Insititute of Hematology, Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital, Peking University Insititute of Hematology
name: Xiaohui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: