NCT03364764

Brief Summary

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. 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 sirolimus for refractory PRCA.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
64

participants targeted

Target at P25-P50 for phase_4

Timeline
Completed

Started Feb 2018

Typical duration for phase_4

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 21, 2017

Completed
16 days until next milestone

First Posted

Study publicly available on registry

December 7, 2017

Completed
2 months until next milestone

Study Start

First participant enrolled

February 1, 2018

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2019

Completed
2.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2021

Completed
Last Updated

July 6, 2021

Status Verified

June 1, 2020

Enrollment Period

1 year

First QC Date

November 21, 2017

Last Update Submit

July 1, 2021

Conditions

Pure Red Cell Aplasia

Keywords

sirolimusrefractory pure red cell aplasiaprospective study

Outcome Measures

Primary Outcomes (1)

  • Hemoglobin level

    Hemoglobin level in g/L

    6 months

Secondary Outcomes (1)

  • Hemoglobin level

    2 years

Study Arms (1)

efficiency of sirolimus on PRCA

EXPERIMENTAL

A prospective research of the sirolimus efficiency on refractory PRCA patients On refractory PRCA patients, sirolimus was tried. Dosage: 2mg QD for the first day, then 1 mg QD. Medication time should last at least 6 months.

Drug: Sirolimus

Interventions

SirolimusDRUG

On refractory PRCA patients, sirolimus was tried. Dosage: 2mg QD for the first day, then 1 mg QD. Medication time should last at least 6 months.

Also known as: Sirolimus Tablets
efficiency of sirolimus on PRCA

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Refractory pure red cell aplasia.
  • Excluding other diseases which might cause hematological abnormalities.
  • No response or intolerant to first and second line therapies.
  • years old.
  • All subjects must: agree not to donate blood or be counseled about pregnancy precautions and risks of fetal exposure.
  • Written informed consent.

You may not qualify if:

  • NOT refractory pure red cell anemia.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Related Publications (4)

  • Bride KL, Vincent T, Smith-Whitley K, Lambert MP, Bleesing JJ, Seif AE, Manno CS, Casper J, Grupp SA, Teachey DT. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. Blood. 2016 Jan 7;127(1):17-28. doi: 10.1182/blood-2015-07-657981. Epub 2015 Oct 26.

    PMID: 26504182RESULT

  • Li J, Wang Z, Dai L, Cao L, Su J, Zhu M, Yu Z, Bai X, Ruan C. Effects of rapamycin combined with low dose prednisone in patients with chronic immune thrombocytopenia. Clin Dev Immunol. 2013;2013:548085. doi: 10.1155/2013/548085. Epub 2013 Dec 2.

    PMID: 24363761RESULT

  • Means RT Jr. Pure red cell aplasia. Blood. 2016 Nov 24;128(21):2504-2509. doi: 10.1182/blood-2016-05-717140.

    PMID: 27881371RESULT

  • Teachey DT, Greiner R, Seif A, Attiyeh E, Bleesing J, Choi J, Manno C, Rappaport E, Schwabe D, Sheen C, Sullivan KE, Zhuang H, Wechsler DS, Grupp SA. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. Br J Haematol. 2009 Apr;145(1):101-6. doi: 10.1111/j.1365-2141.2009.07595.x. Epub 2009 Feb 4.

    PMID: 19208097RESULT

MeSH Terms

Conditions

Red-Cell Aplasia, Pure

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Bing B Han, PhD.

    Peking Union Medical College Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

November 21, 2017

First Posted

December 7, 2017

Study Start

February 1, 2018

Primary Completion

February 1, 2019

Study Completion

May 1, 2021

Last Updated

July 6, 2021

Record last verified: 2020-06

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)