The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed ITP

Sponsor

Peking University People's Hospital (Other)

Overall Status

Unknown status

CT.gov ID

NCT04368598

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

2.1

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenia	Drug: Dexamethasone Drug: Acetylcysteine	Phase 2

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

Anticipated Enrollment :

44 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly-diagnosed Immune Thrombocytopenia: A Single-arm, Open-label Trial

Actual Study Start Date :

Apr 1, 2019

Anticipated Primary Completion Date :

Dec 31, 2020

Anticipated Study Completion Date :

Dec 31, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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.	Drug: Dexamethasone Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated on days 11 to 14 in the case of lack of response by day 10) Other Names: HD-DXM High-dose Dexamethasone Drug: Acetylcysteine Acetylcysteine, po, 400mg tid, for 4 weeks Other Names: YiWeiShi

Arm

Intervention/Treatment

Experimental: 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.

Drug: Dexamethasone

Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated on days 11 to 14 in the case of lack of response by day 10)

Other Names:

HD-DXM

High-dose Dexamethasone

Drug: Acetylcysteine

Acetylcysteine, po, 400mg tid, for 4 weeks

Other Names:

YiWeiShi

Outcome Measures

Primary Outcome Measures

Number of patients with durable response after 6 months [6 months]
Number of patients with durable response after 6 months

Secondary Outcome Measures

Early response [7 days]
Number of patients with early response on day 7
Initial response [1 month]
Number of patients with initial response at 1 month
Bleeding [1 year]
Number of patients with bleeding complication therapy; bleeding score
DOR [1 year]
duration of response
Relapse [1 year]
Number of patients relapse during the observation
Adverse events [1 year]
Number of patients with adverse events

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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