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Tacrolimus Treatment for Refractory Autoimmune Cytopenia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03918265
Recruitment Status : Not yet recruiting
First Posted : April 17, 2019
Last Update Posted : April 17, 2019
Information provided by (Responsible Party):
Peking Union Medical College Hospital

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Tacrolimus Treatment for Refractory Autoimmune Cytopenia
Estimated Study Start Date : April 10, 2019
Estimated Primary Completion Date : October 10, 2019
Estimated Study Completion Date : April 10, 2021

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

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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03918265

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Contact: Ruoxi Zhang, M.D. +86 18510064839

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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Ruoxi Zhang, MD    +86 18510064839   
Sponsors and Collaborators
Peking Union Medical College Hospital
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Principal Investigator: Bing Hang, PhD Peking Union Medical College Hospital

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Responsible Party: Peking Union Medical College Hospital Identifier: NCT03918265     History of Changes
Other Study ID Numbers: tacrolimus-2
First Posted: April 17, 2019    Key Record Dates
Last Update Posted: April 17, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Peking Union Medical College Hospital:
Refractory autoimmune cytopenia
Prospective study
Additional relevant MeSH terms:
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Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Red-Cell Aplasia, Pure
Hematologic Diseases
Autoimmune Diseases
Immune System Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action