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

Phase 2

• Conditions: Immune Thrombocytopenia
• Interventions: Drug: Dexamethasone; Drug: Acetylcysteine

• Registration Number: NCT04368598
• Lead Sponsor: Peking University People's Hospital

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 Timeline

• Study Start: 2019-04-01
• Status Verified: 2020-04
• Study First Submitted: 2020-04-26
• Study First Submitted QC: 2020-04-28
• Last Update Submitted: 2020-04-28
• Study First Posted: 2020-04-30
• Last Update Posted: 2020-04-30
• Primary Completion: 2020-12-31
• Study Completion: 2020-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 44

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
High-dose Dexamethasone plus Acetylcysteine	Dexamethasone	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.
High-dose Dexamethasone plus Acetylcysteine	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.

Primary Outcome Measures

Name	Time	Method
Number of patients with durable response after 6 months	6 months	Number of patients with durable response after 6 months

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Peking University Insititute of Hematology, Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China