NCT03688191

Brief Summary

We have designed a single-arm, open-label study of sirolimus in Chinese patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital (PUMCH). Patients received oral sirolimus at a starting dose of 2 mg per day for 3 days and a sequential dose of 1mg per day, with dose adjusted according to tolerance and to maintain a therapeutic range of 6-15 ng/mL. Patients were treated with sirolimus for more than 6 months. Safety outcomes and the efficacy endpoints were assessed in all patients who received at least one dose of treatment.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
20

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Sep 2017

Typical duration for phase_4

Geographic Reach
1 country

1 active site

Status
unknown

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

Study Start

First participant enrolled

September 21, 2017

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

July 30, 2018

Completed
2 months until next milestone

First Posted

Study publicly available on registry

September 28, 2018

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2018

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2019

Completed
Last Updated

April 17, 2019

Status Verified

December 1, 2018

Enrollment Period

1.3 years

First QC Date

July 30, 2018

Last Update Submit

April 16, 2019

Conditions

Connective Tissue DiseasesThrombocytopenia

Keywords

Sirolimusconnective tissue diseasethrombocytopenia

Outcome Measures

Primary Outcomes (1)

  • Number of participants with complete remission of platelet count

    Complete remission of platelet count means the platelet count over 100X10\^9/L. every participants will take CBC as their screening times.

    6 months

Secondary Outcomes (1)

  • Number of participants with partial remission of platelet count

    6 months

Other Outcomes (1)

  • Bleeding Events with treatment-related adverse events

    6 months

Study Arms (1)

Study Group

EXPERIMENTAL

participants who received sirolimus treatment

Drug: Sirolimus

Interventions

SirolimusDRUG

Patients will receive oral sirolimus at a starting dose of 2 mg per day for 3 days and a sequential dose of 1mg per day, with dose adjusted according to tolerance and to maintain a therapeutic range of 6-15 ng/mL.

Study Group

Eligibility Criteria

Age16 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital.

You may not qualify if:

  • new diagnosed CTD-IT patients

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100005, China

Location

Related Publications (1)

  • Wu C, Wang Q, Xu D, Li M, Zeng X. Sirolimus for patients with connective tissue disease-related refractory thrombocytopenia: a single-arm, open-label clinical trial. Rheumatology (Oxford). 2021 Jun 18;60(6):2629-2634. doi: 10.1093/rheumatology/keaa645.

    PMID: 33200217DERIVED

MeSH Terms

Conditions

Connective Tissue DiseasesThrombocytopenia

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Skin and Connective Tissue DiseasesBlood Platelet DisordersHematologic DiseasesHemic and Lymphatic DiseasesCytopenia

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Xiaofeng Peng, Professor

    Peking Union Medical College Hospital

    STUDY CHAIR

  • Chanyuan WU, MD

    Peking Union Medical College Hospital

    PRINCIPAL INVESTIGATOR

  • Mengtao LI, Professor

    Peking Union Medical College Hospital

    STUDY DIRECTOR

Study Design

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

Study Record Dates

First Submitted

July 30, 2018

First Posted

September 28, 2018

Study Start

September 21, 2017

Primary Completion

December 31, 2018

Study Completion

September 30, 2019

Last Updated

April 17, 2019

Record last verified: 2018-12

Locations

CN(1)