Last reviewed · How we verify

NCT05298358

RIC alloBMT With Post-transplant Cyclophosphamide for Refractory Systemic Sclerosis

Terminated Phase 1 Last updated 4 February 2025
What this trial tests

Phase 1 trial testing RIC alloBMT w PTCy in refractory SSc in Systemic Sclerosis in 1 participant. Terminated before completion.

Timeline
18 November 2022
Primary endpoint
13 November 2024
21 November 2024

Quick facts

Lead sponsorSidney Kimmel Comprehensive Cancer Center at Johns Hopkins
PhasePhase 1
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment1
Start date18 November 2022
Primary completion13 November 2024
Estimated completion21 November 2024
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — full company profile →

Who can join

Adults 18 to 65, any sex, with Systemic Sclerosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

Verify or expand the search:

Other recruiting trials for Systemic Sclerosis

Currently open trials in the same condition.

Other Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05298358.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing