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

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

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).

Study Overview

• Status: Unknown
• Conditions: Immune Thrombocytopenia
• Intervention / Treatment: Drug: Dexamethasone; Drug: Acetylcysteine

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 Type: Interventional
• Enrollment (Anticipated): 44
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xinglin Wang, MD; Phone Number: +8613051522865; Email: wxlbjmu@163.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100044
      • Recruiting
      • Peking University Insititute of Hematology, Peking University People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with durable response after 6 months	Number of patients with durable response after 6 months	6 months

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2019
• Primary Completion (Anticipated): December 31, 2020
• Study Completion (Anticipated): December 31, 2020

Study Registration Dates

• First Submitted: April 26, 2020
• First Submitted That Met QC Criteria: April 28, 2020
• First Posted (Actual): April 30, 2020

Study Record Updates

• Last Update Posted (Actual): April 30, 2020
• Last Update Submitted That Met QC Criteria: April 28, 2020
• Last Verified: April 1, 2020

More Information

Terms related to this study

• Keywords: immune thrombocytopenia; acetylcysteine
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Autonomic Agents; Peripheral Nervous System Agents; Antiviral Agents; Enzyme Inhibitors; Anti-Inflammatory Agents; Antineoplastic Agents; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Protease Inhibitors; Protective Agents; Respiratory System Agents; Antioxidants; Antidotes; Free Radical Scavengers; Expectorants; Dexamethasone; Dexamethasone acetate; BB 1101; Acetylcysteine; N-monoacetylcystine
• Other Study ID Numbers: ITP-PKU008

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No