The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed Immune Thrombocytopenia: A Single-arm, Open-label Trial
Trial Snapshot
- Phase
- Phase 2
- Enrollment
- 44
- Locations
- 1
- Primary Endpoint
- Number of patients with durable response after 6 months
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 80 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Clinically confirmed immune thrombocytopenic purpura (ITP) newly diagnosed
- •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)
- •Subject is ≥ 18 years and ≤80years
- •Subject has signed and dated written informed consent.
- •Fertile patients must use effective contraception during treatment and observational period
- •Negative pregnancy test
Exclusion Criteria
- •Have an impaired renal function as indicated by a serum creatinine level > 2.0 mg/dL
- •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
- •Have a New York Heart Classification III or IV heart disease
- •Have a history of severe psychiatric disorder or are unable to comply with study and follow-up procedures
- •Have active hepatitis B or hepatitis C infection
- •Have a HIV infection
- •Have active infection requiring antibiotic therapy within 7 days prior to study entry
- •Are pregnant or lactating women, or plan to become pregnant or impregnated within 12 months of receiving study drug
- •Previous treatment with rituximab
- •Previous splenectomy
- •Had previous or concomitant malignant disease
- •Not willing to participate in the study.
- •Expected survival of < 2 years
- •Intolerant to murine antibodies
- •Immunosuppressive treatment within the last month
- •Connective tissue disease
- •Autoimmune hemolytic anemia
- •Patients currently involved in another clinical trial with evaluation of drug treatment
Arms & Interventions
High-dose Dexamethasone plus Acetylcysteine
For all patients in this arm, DXM was administered intravenously at 40 mg daily for 4 consecutive days and then stopped. If platelet count remained below 30×10^9/L or there were bleeding symptoms by day 10 to 14, an additional 4-day course of DXM (40 mg daily) was given. Besides, all patients received Acetylcysteine orally at 0.4g three times a day for 4 consecutive weeks in the first month.
Intervention: Dexamethasone (Drug)
High-dose Dexamethasone plus Acetylcysteine
For all patients in this arm, DXM was administered intravenously at 40 mg daily for 4 consecutive days and then stopped. If platelet count remained below 30×10^9/L or there were bleeding symptoms by day 10 to 14, an additional 4-day course of DXM (40 mg daily) was given. Besides, all patients received Acetylcysteine orally at 0.4g three times a day for 4 consecutive weeks in the first month.
Intervention: Acetylcysteine (Drug)
Outcomes
Primary Outcomes
Number of patients with durable response after 6 months
Time Frame: 6 months
Number of patients with durable response after 6 months
Secondary Outcomes
- Relapse(1 year)
- Adverse events(1 year)
- Early response(7 days)
- Initial response(1 month)
- Bleeding(1 year)
- DOR(1 year)
Investigators
Xiao Hui Zhang
Vice president of Peking University Institute of Hematology
Peking University People's Hospital