Autoimmune Hemolytic AnemiaPhase 4NCT03918265
Tacrolimus Treatment for Refractory Autoimmune Cytopenia
A Phase 4 study testing Tacrolimus in people with Autoimmune Hemolytic Anemia and Pure Red Cell Aplasia, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Autoimmune Hemolytic Anemia, Pure Red Cell Aplasia, Evans Syndrome
- What is being tested?
- Tacrolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteChina
- Study dates
- 4 May 2019to 10 Nov 2021
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Ruoxi Zhang, MD, +86 18510064839, rx_zh15@163.com
About this study
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.
Read the 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.
- Being tested
- Tacrolimus
- Keywords
- Tacrolimus, Refractory autoimmune cytopenia, Prospective study
- Medical subject headings
- Anemia, Hemolytic, Autoimmune, Red-Cell Aplasia, Pure, Evans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TacrolimusDrug
Also called tacrolimus capsule
On refractory autoimmune cytopenia patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study
| Study group | Role | What they receive |
|---|---|---|
| Efficiency of tacrolimus on autoimmune cytopenia | Experimental | 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. Receives: Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin level
Hemoglobin level in g/L
Measured: 6 months
Platelet count
Platelet count in /L
Measured: 6 months
Secondary outcomes (2)
Hemoglobin level
Hemoglobin level in g/L
Measured: 2 years
Platelet count
Platelet count in /L
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2019
- Study start4 May 2019
- Primary completion10 Aug 2020 (estimated)
- Study completion10 Nov 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Bing Hang, PhD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03918265
- Sponsor's protocol ID
- tacrolimus-2
- Status last verified
- Jun 2019
- First submitted
- 16 Apr 2019
- Last updated
- 3 Dec 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 28566017Tabchi 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. No abstract available.
PubMed 25209619
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tacrolimus Treatment for Refractory Autoimmune Cytopenia
- ClinicalTrials.gov ID
- NCT03918265
- Acronym / sponsor ID
- tacrolimus-2
- Keywords
- Tacrolimus, Refractory autoimmune cytopenia, Prospective study
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 4 May 2019 (actual)
- Primary completion
- 10 Aug 2020 (estimated)
- Study completion
- 10 Nov 2021 (estimated)
- First posted
- 17 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Efficiency of tacrolimus on autoimmune cytopenia | Experimental | Drug: TacrolimusA 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. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TacrolimusAlso: tacrolimus capsuleOn refractory autoimmune cytopenia patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study | Drug | Efficiency of tacrolimus on autoimmune cytopenia |
Primary outcome
Hemoglobin level
Time frame: 6 months
Hemoglobin level in g/L
Platelet count
Time frame: 6 months
Platelet count in /L
2 secondary outcomes
Hemoglobin level
Time frame: 2 years
Platelet count
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2019.
- Background: PubMed 28566017Li 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.
- Background: PubMed 25209619Tabchi 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. No abstract available.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bing Hang, PhD principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tacrolimus in people with Autoimmune Hemolytic Anemia and Pure Red Cell Aplasia, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ruoxi Zhang, M.D. (+86 18510064839, rx_zh15@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin level (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03918265, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Dec 2019.