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RIC alloBMT With Post-transplant Cyclophosphamide for Refractory Systemic Sclerosis

NCT05298358 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-02-04

No results posted yet for this study

Summary

This is a Phase I, single arm, open label, single center pilot study to assess a reduced-intensity conditioning regimen, bone marrow transplantation with high dose cyclophosphamide (PTCy) in recipients with refractory systemic sclerosis. This study expects to enroll 15 donor/recipient pairs for a total of 30 participants.

The primary objective of this study is to assess the safety of using a reduced intensity condition (RIC) preparative regimen bone marrow transplant (BMT) with post-transplant cyclophosphamide for graft vs host disease (GVHD) prophylaxis as treatment for patients with scleroderma. Safety events are grade III-IV GVHD and treatment related mortality within 1 year.

Eligibility includes patients \>18 years who are eligible for transplantation according to the BMT Policy Manual, meet the 2013 ACR/EULAR Criteria for Systemic Sclerosis and display active diffuse cutaneous disease.

The trial also includes analyses of the effects of BMT on skeletal and cardiac muscle using systemic scleroderma serum biomarkers of CK, aldolase, and troponin as well as periodic monitoring of circulating scleroderma auto-antibody titers, autoreactive T cells, and flow cytometric signatures over the one-year study period to correlate with response.

Conditions

Interventions

BIOLOGICAL

RIC alloBMT w PTCy in refractory SSc

The preparative regimen: * Thymoglobulin 0.5 mg/kg IV day -9 * Thymoglobulin 2 mg/kg IV Days -8, -7 * Cyclophosphamide (Cy) 14.5 mg/kg IV Days -6,-5 * Fludarabine 30 mg/m2 IV Days -6, -5, -4, -3,-2 * Total body irradiation (TBI) 400cGy * Day 0- infusion of unmanipulated bone marrow The GVHD prophylaxis regimen: * Cy 50 mg/kg IV Days 3, 4 * Tacrolimus (FK-506) (IV or oral (po)) beginning Day 5 with dose adjusted to maintain a trough level of 5-15 ng/mL. Sirolimus may be substituted for tacrolimus. * Mycophenolate mofetil (MMF) 15 mg/kg po TID, maximum dose 1 g po TID Day 5 - Day 35 * Filgrastim (G-CSF) 5 µg/kg SQ daily beginning Day 5 until absolute neutrophil (ANC) is greater than 1000/µl over at least 2 days. Supportive care: * Mesna- 10 mg/kg/ IV Day 3, 4 * Patients will receive infection prophylaxis and treatment according to institutional guidelines.

Sponsors & Collaborators

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    lead OTHER

Principal Investigators

  • Cole Sterling, MD · Johns Hopkins University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2022-11-18
Primary Completion
2024-11-13
Completion
2024-11-21
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05298358 on ClinicalTrials.gov