NCT05746689

Brief Summary

gG4-related disease (IgG4-RD) is a newly recognized systemic autoimmune disease that can involve the pan-creatobiliary tract, retroperitoneum/aorta, head and neck region, and salivary glands, et al. Glucocorticoids are the first-line agents for the treatment of IgG4-RD, however, in order to maintain long-term disease stability and avoid disease relapse, glucocorticoids maintenance therapy should last for a long period, which may induce various glucocorticoid-associated adverse reactions. Sirolimus plays dual roles in inhibiting lymphocyte activation and fibroblast proliferation. It is inferred from its mechanism that sirolimus is a good potential treatment option for IgG4-RD. Therefore, we conducted this single-arm clinical trial on patients with IgG4-RD to determine the efficacy and safety of sirolimus.

Trial Health

65
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Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for not_applicable

Timeline
32mo left

Started Mar 2023

Longer than P75 for not_applicable

Status
not yet recruiting

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress55%
Mar 2023Dec 2028

First Submitted

Initial submission to the registry

February 13, 2023

Completed
15 days until next milestone

First Posted

Study publicly available on registry

February 28, 2023

Completed
1 day until next milestone

Study Start

First participant enrolled

March 1, 2023

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2026

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2028

Last Updated

February 28, 2023

Status Verified

February 1, 2023

Enrollment Period

3.8 years

First QC Date

February 13, 2023

Last Update Submit

February 25, 2023

Conditions

IgG4-related Disease

Keywords

IgG4-related diseasesirolimusmTOR

Outcome Measures

Primary Outcomes (1)

  • PrRelapse rate

    For patients who achieve disease response at 12 weeks, recurrence is defined as increases in the IgG4-RD RI ≥2 and/or the need for the reinstitution of treatment.

    through study completion, an average of 1 year

Secondary Outcomes (3)

  • Disease response rate at 12 weeks

    12 weeks of treatment

  • Remission rate at 48 weeks

    48 weeks of treatment

  • Improvement of patient's global assessment (PGA)

    48 weeks of treatment

Study Arms (1)

glucocorticoid and sirolimus combination therapy

EXPERIMENTAL

Prednisone acetate 0.8mg/Kg/d (maximum dose 60mg/d), reduced by 5mg every 14 days, reduced by 2.5mg every 2 weeks after 30mg/d until discontinuation. At the same time, treatment for prevention or control of osteoporosis was given. Sirolimus: 2mg/day for the first three days and 1mg/day thereafter. The plasma drug concentration was monitored at 14 days, 12 weeks, and 48 weeks of medication to maintain a plasma drug concentration of 4-15 ug/L.

Drug: Sirolimus

Interventions

SirolimusDRUG

Sirolimus The efficacy is evaluated at 12 weeks, and treatment will be adjusted according to the control of disease and adverse effects.For experimental group, if a patient is assessed as treatment failure (TS), the patient should be withdrawn from the study and receive rescue treatment. Whereas, a patient would be transferred to the control group if he/ she cann't stand the side effects of sirolimus but not serious adverse event (SAE).

Also known as: Prednisone
glucocorticoid and sirolimus combination therapy

Eligibility Criteria

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

You may qualify if:

  • Patients diagnosed with IgG4-RD according to the 2011 Comprehensive Diagnostic Criteria for IgG4-RD;
  • Status classified as active disease based on an IgG4-RD Responder Index (RI) ≥2 at screening.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Immunoglobulin G4-Related Disease

Interventions

SirolimusPrednisone

Condition Hierarchy (Ancestors)

Autoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic ChemicalsPregnadienediolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic Compounds

Study Officials

  • Yuying Wang

    Peking University International Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yuying WANG, Master

CONTACT

8615210976309wangyuying028@126.com

Hui Gao, Doctor

CONTACT

8613811833264gh841017@126.com

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigato

Study Record Dates

First Submitted

February 13, 2023

First Posted

February 28, 2023

Study Start

March 1, 2023

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2028

Last Updated

February 28, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share