Clinical Trials

Brief Summary: This observational study is being done to understand why people with scleroderma can develop pulmonary arterial hypertension (high blood pressure in the lungs, abbreviated PAH) and a weak heart muscle (heart failure). The study will also help the investigators understand why people with PAH from an unknown cause (called idiopathic PAH, or IPAH) can also develop a weakened heart muscle. The response of the right side of the heart or right ventricle (RV) to standard PAH therapy in scleroderma-associated PAH and in IPAH will be assessed. Blood and tissue samples will be collected from research participants during participants’ normal standard of care procedures. People with scleroderma-associated PAH or idiopathic cause (IPAH) who need a right heart catheterization may join this study.

Contacts:
Johns Hopkins Baltimore, Maryland 21287
Paul Hassoun, MD   410-614-6311   phassou1@jhmi.edu
Dezeray Cephas Dutton   410 502 4362   dcephas1@jhu.edu

Brief Summary: This is a Phase II randomized, double-blind, placebo-controlled trial of sildenafil in men and women with Scleroderma with mildly elevated pulmonary pressures (SSc-MEP) to determine whether sildenafil may be an effective treatment for SSc-MEP.

Contacts:
Johns Hopkins Baltimore, Maryland 21287
Stephen Mathai, MD  410.614.6311  smathai4@jhmi.edu
Dezeray Dutton  443.507.8222  dcephas1@jhu.edu

Brief Summary:  The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of RO7303509 treatment in participants with systemic sclerosis (SSc) during a multiple-ascending-dose (MAD) portion of the trial. In the MAD phase, increasing doses of study drug will be tested sequentially. For each dose tested, the MAD stage will consist of a treatment period of 12 weeks followed by either a safety follow-up period of 13 weeks or continued treatment in an optional open-label safety extension (OSE) stage of 52 weeks to assess the long-term safety. All patients in the OSE stage will receive RO7303509 and no patient will receive placebo.

Contacts:
Medstar Georgetown University Hospital, Washington, District of Columbia
2000 Reference Study ID Number: GA43360

https://forpatients.roche.com/
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com

Brief Summary:  The purpose of this phase 2 multicenter, randomized, double-blind, placebo-controlled, study is to assess the safety and efficacy of ifetroban in patients with diffuse cutaneous systemic SSc (dcSSc) or SSc-associated pulmonary arterial hypertension (SSc-PAH).

Contact:
Johns Hopkins University, Baltimore, Maryland 21224
Gwen Leatherman, RN, MS, CCRP   410-550-8582
Principal Investigator: Laura Hummer, MD

Brief 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.

Contacts:
Johns Hopkins University Baltimore, Maryland 21287
Cole Sterling, MD    410-955-8893    csterli4@jhmi.edu
Laura Ackley, RN    410-502-0969    lackley1@jhmi.edu

Platform Clinical Study for Conquering Scleroderma (CONQUEST)
ClinicalTrials.gov ID NCT06195072
Sponsor Scleroderma Research Foundation, Inc.
Information provided by Scleroderma Research Foundation, Inc. (Responsible Party)

Brief Summary

The goal of this clinical trial is to test efficacy of different investigational products (IPs) compared with placebo on the change from baseline to the end of the treatment period at Week 52 in lung capacity in participants with Interstitial Lung Disease Secondary to Systemic Sclerosis.

Official Title

Platform Clinical Study for Conquering Scleroderma: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2b Platform Clinical Study to Evaluate the Safety and Efficacy of Investigational Products in Participants With Interstitial Lung Disease Secondary to Systemic Sclerosis

Intervention / Treatment

• Drug: Amlitelimab
• Drug: BI 1015550
• Drug: Placebo

Other Study ID Numbers SRF 201

Study Contact
Name: Kelly Oliver
Phone Number: (415) 260 -5330
Email: kelly@sclerodermaresearch.org

Washington, District of Columbia
Georgetown University Medical Center – Department of Rheumatology
Name: Virginia Steen Principal Investigator
Phone Number: 202-444-6210
Email: steenv@georgetown.edu

A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease (BLISS chILD)
ClinicalTrials.gov ID NCT05878717
Sponsor GlaxoSmithKline
Information provided by GlaxoSmithKline (Responsible Party)

Brief Summary

This study investigates the efficacy and safety of belimumab compared to placebo, in addition to standard therapy, for the treatment of participants with systemic sclerosis associated interstitial lung disease (SSc-ILD). The study will evaluate the effect of belimumab treatment on lung function as well as on extra-pulmonary disease manifestations, including skin thickening and general symptoms, such as fatigue, that impact quality of life (QoL).

Study Contact
Washington, District of Columbia
Georgetown University Medical Center – Department of Rheumatology
Name: Virginia Steen Principal Investigator
Phone Number: 202-444-6210
Email: steenv@georgetown.edu

RecruitingGSK Investigational Site
Contact: US GSK Clinical Trials Call Center
Phone Number: 877-379-3718
Email: GSKClinicalSupportHD@gsk.com