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

NCT04368598 | Unknown Phase 2

• 1 other identifier: ITP-PKU008
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

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).

Conditions

Immune Thrombocytopenia

Keywords

immune thrombocytopenia; acetylcysteine

Outcome Measures

Primary Outcomes (1)

• Number of patients with durable response after 6 months

  Number of patients with durable response after 6 months

  6 months

Secondary Outcomes (6)

• Early response

  7 days

• Initial response

  1 month

• Bleeding

  1 year

• DOR

  1 year

• Relapse

  1 year

Study Arms (1)

High-dose Dexamethasone plus Acetylcysteine

EXPERIMENTAL

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; Drug: Acetylcysteine

Interventions

Dexamethasone DRUG

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)

Also known as: HD-DXM, High-dose Dexamethasone

High-dose Dexamethasone plus Acetylcysteine

Acetylcysteine DRUG

Acetylcysteine, po, 400mg tid, for 4 weeks

Also known as: YiWeiShi

High-dose Dexamethasone plus Acetylcysteine

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University Insititute of Hematology, Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Dexamethasone; Acetylcysteine

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Xiaohui Zhang, MD

  Peking University People's Hospital, Peking University Insititute of Hematology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaohui Zhang, MD

CONTACT

+8613522338836; zhangxh100@sina.com

Xinglin Wang, MD

CONTACT

+8613051522865; wxlbjmu@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking University Institute of Hematology

Study Record Dates

• First Submitted: April 26, 2020
• First Posted: April 30, 2020
• Study Start: April 1, 2019
• Primary Completion: December 31, 2020
• Study Completion: December 31, 2020
• Last Updated: April 30, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)