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出境医 / 临床实验 / Tacrolimus Treatment for Refractory Autoimmune Cytopenia

Tacrolimus Treatment for Refractory Autoimmune Cytopenia

Study Description
Brief Summary:
Autoimmune cytopenia, including autoimmune hemolytic anemia (AIHA), pure red cell aplasia (PRCA), Evans syndrome (ES), usually has good responses to steroids therapies as first line, but there is a considerable percentage of patients who relapse, become refractory or dependent on steroids to maintain an acceptable level of hemoglobin or platelets. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of tacrolimus for refractory autoimmune cytopenia.

Condition or disease Intervention/treatment Phase
Autoimmune Hemolytic Anemia Pure Red Cell Aplasia Evans Syndrome Drug: Tacrolimus Phase 4

Detailed Description:

Tacrolimus binds FKBP12 with high affinity after entry into cytoplasm, suppresses calcineurin activity and prevents nuclear translocation of transcription factors such as NF-AT that are involved in IL-2 gene transcription. As a result, T-cell activation is inhibited with a subsequent reduction in the production of cytokines that include IL-2, TNF-α, IL-3, IL-4, IFN-γ, IL-6 and IL-10; B-cell activation, class-switching and immunoglobulin production are also attenuated. Reports on the use of tarcrolimus in the treatment of autoimmune cytopenia are anecdotal and describe therapy with a variety of drug dosages.

The purpose of this study is to evaluate the effect of tacrolimus on patients with refractory autoimmune cytopenia, the side-effects will be documented and plasma concentration of tacrolimus will be monitor.

Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Tacrolimus Treatment for Refractory Autoimmune Cytopenia
Actual Study Start Date : May 4, 2019
Estimated Primary Completion Date : August 10, 2020
Estimated Study Completion Date : November 10, 2021
Arms and Interventions
Arm Intervention/treatment
Experimental: Efficiency of tacrolimus on autoimmune cytopenia

A prospective research of the tacrolimus efficiency on refractory autoimmune cytopenia patients. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study.

Medication time should last at least 6 months.

 Drug: Tacrolimus
On refractory autoimmune cytopenia patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study
Other Name: tacrolimus capsule
Outcome Measures
Primary Outcome Measures :
  1. Hemoglobin level [ Time Frame: 6 months ]
    Hemoglobin level in g/L

  2. Platelet count [ Time Frame: 6 months ]
    Platelet count in /L


Secondary Outcome Measures :
  1. Hemoglobin level [ Time Frame: 2 years ]
    Hemoglobin level in g/L

  2. Platelet count [ Time Frame: 2 years ]
    Platelet count in /L


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Refractory autoimmune cytopenia, including autoimmune hemolytic anemia, pure red cell aplasia, Evans syndrome.
  • 18-80 years old.
  • No response or intolerant to first and second line therapies.
  • ECOG Performance Status of 0-2
  • Written informed consent.

Exclusion Criteria:

  • Other diseases which might cause hematological abnormalities.
  • Response and well tolerate to first or second line therapy.
  • Patients who are under 18-year-old or over 80-year-old.
  • Pregnant or lactating.
  • Patients unwilling to or unable to comply with the protocol.
Contacts and Locations

Contacts
Layout table for location contacts
Contact: Ruoxi Zhang, M.D. +86 18510064839 rx_zh15@163.com

Locations
Layout table for location information
China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Ruoxi Zhang, MD    +86 18510064839    rx_zh15@163.com   
Sponsors and Collaborators
Peking Union Medical College Hospital
Investigators
Layout table for investigator information
Principal Investigator: Bing Hang, PhD Peking Union Medical College Hospital
Tracking Information
First Submitted Date  ICMJE April 16, 2019
First Posted Date  ICMJE April 17, 2019
Last Update Posted Date December 3, 2019
Actual Study Start Date  ICMJE May 4, 2019
Estimated Primary Completion Date August 10, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
  • Hemoglobin level [ Time Frame: 6 months ]
    Hemoglobin level in g/L
  • Platelet count [ Time Frame: 6 months ]
    Platelet count in /L
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
  • Hemoglobin level [ Time Frame: 2 years ]
    Hemoglobin level in g/L
  • Platelet count [ Time Frame: 2 years ]
    Platelet count in /L
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Tacrolimus Treatment for Refractory Autoimmune Cytopenia
Official Title  ICMJE Tacrolimus Treatment for Refractory Autoimmune Cytopenia
Brief Summary Autoimmune cytopenia, including autoimmune hemolytic anemia (AIHA), pure red cell aplasia (PRCA), Evans syndrome (ES), usually has good responses to steroids therapies as first line, but there is a considerable percentage of patients who relapse, become refractory or dependent on steroids to maintain an acceptable level of hemoglobin or platelets. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of tacrolimus for refractory autoimmune cytopenia.
Detailed Description

Tacrolimus binds FKBP12 with high affinity after entry into cytoplasm, suppresses calcineurin activity and prevents nuclear translocation of transcription factors such as NF-AT that are involved in IL-2 gene transcription. As a result, T-cell activation is inhibited with a subsequent reduction in the production of cytokines that include IL-2, TNF-α, IL-3, IL-4, IFN-γ, IL-6 and IL-10; B-cell activation, class-switching and immunoglobulin production are also attenuated. Reports on the use of tarcrolimus in the treatment of autoimmune cytopenia are anecdotal and describe therapy with a variety of drug dosages.

The purpose of this study is to evaluate the effect of tacrolimus on patients with refractory autoimmune cytopenia, the side-effects will be documented and plasma concentration of tacrolimus will be monitor.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Autoimmune Hemolytic Anemia
  • Pure Red Cell Aplasia
  • Evans Syndrome
Intervention  ICMJE Drug: Tacrolimus
On refractory autoimmune cytopenia patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study
Other Name: tacrolimus capsule
Study Arms  ICMJE Experimental: Efficiency of tacrolimus on autoimmune cytopenia

A prospective research of the tacrolimus efficiency on refractory autoimmune cytopenia patients. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study.

Medication time should last at least 6 months.

Intervention: Drug: Tacrolimus
Publications *
  • Li Y, Feng X. Efficacy and safety of tacrolimus in systemic lupus erythematosus patients with refractory thrombocytopenia: a retrospective study. Lupus. 2018 Jan;27(1):60-65. doi: 10.1177/0961203317711011. Epub 2017 May 31.
  • Tabchi S, Hanna C, Kourie HR, Aftimos P, El Osta L, Ghosn M. Successful treatment of Evans syndrome with Tacrolimus. Invest New Drugs. 2015 Feb;33(1):254-6. doi: 10.1007/s10637-014-0155-9. Epub 2014 Sep 12.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 16, 2019) 		80
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 10, 2021
Estimated Primary Completion Date August 10, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Refractory autoimmune cytopenia, including autoimmune hemolytic anemia, pure red cell aplasia, Evans syndrome.
  • 18-80 years old.
  • No response or intolerant to first and second line therapies.
  • ECOG Performance Status of 0-2
  • Written informed consent.

Exclusion Criteria:

  • Other diseases which might cause hematological abnormalities.
  • Response and well tolerate to first or second line therapy.
  • Patients who are under 18-year-old or over 80-year-old.
  • Pregnant or lactating.
  • Patients unwilling to or unable to comply with the protocol.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ruoxi Zhang, M.D. +86 18510064839 rx_zh15@163.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03918265
Other Study ID Numbers  ICMJE tacrolimus-2
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Peking Union Medical College Hospital
Study Sponsor  ICMJE Peking Union Medical College Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Bing Hang, PhD Peking Union Medical College Hospital
PRS Account Peking Union Medical College Hospital
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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