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Bone Marrow Transplantation

RIC alloBMT + Cyclophosphamide for Scleroderma

Phase 1

Recruiting

Led By Cole Sterling, MD

Research Sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male patients meeting contraceptive requirements

Patients with active diffuse cutaneous disease with specific clinical indicators

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

Study Summary

This trialstudies safety of reduced intensity BMT w/ cyclophosphamide to treat scleroderma. Eligibility includes adults with active diffuse cutaneous disease. Effects on skeletal & cardiac muscles will also be monitored.

Who is the study for?

This trial is for adults aged 18-65 with active diffuse cutaneous systemic sclerosis who haven't improved with first-line therapy. They must meet the 2013 ACR/EULAR criteria, have a Karnofsky performance score over 70%, and proper organ function. Pregnant individuals, those with certain infections or uncontrolled diseases like diabetes, recent major surgery or live vaccines are excluded.Check my eligibility

What is being tested?

The study tests a reduced-intensity bone marrow transplant followed by high dose cyclophosphamide in patients with refractory systemic sclerosis to prevent graft vs host disease. It's an open-label Phase I trial enrolling 15 pairs to assess safety and monitor effects on muscle using biomarkers over one year.See study design

What are the potential side effects?

Potential side effects include severe graft vs host disease (a serious condition where the donor cells attack the recipient's body) and treatment-related mortality within one year of receiving the transplant.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a male and follow the required contraceptive measures.

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I have widespread skin disease with noticeable symptoms.

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I am eligible for a bone marrow transplant according to the policy.

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I am following the required birth control measures.

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My heart, liver, kidneys, and lungs are working well.

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I have moderate-to-severe systemic sclerosis.

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I am mostly independent and can care for myself.

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My first cancer treatment did not work.

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I am between 18 and 65 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Acute Graft vs Host disease (GVHD) incidence

Bone Transplantation

Disease relapse or progression incidence

+1 more

Secondary outcome measures

Event free survival- CTCAE v5 Grade 3 or higher toxicities

Event free survival- cardiomyopathy

Event free survival- donor cell engraftment

+9 more

Other outcome measures

Additional exploratory objective- autoreactive T cells

Additional exploratory objective- flow cytometric analysis of HLA-A, B or DR biomarkers on lymphocytes

Additional exploratory objective- scleroderma autoantibody titers

+3 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: RIC- alloBMT with high PTCy in SScExperimental Treatment1 Intervention

Days -9 Thymoglobulin 0.5 mg/kg IV Days -8,-7 Thymoglobulin 2 mg/kg IV daily Days -6, -5 Fludarabine 30 mg/M2 IV Cyclophosphamide 14.5 mg/kg IV Days -4, -3-2 Fludarabine 30 mg/M2 IV Day -1 TBI 400 cGy Day 0 Infuse unmanipulated marrow; begin antimicrobial prophylaxis. Days 3, 4 Cy 50 mg/kg IV and Mesna 40 mg/kg IV Day 5 FK-506 oral and MMF oral and G-CSF Day 30 Assess peripheral blood chimerism Day 35 Discontinue MMF Day 60 Assess peripheral blood chimerism Day 180 Discontinue FK-506 Evaluate disease Assess Chimerism in peripheral blood 1 yr. Evaluate disease by peripheral blood chimerism

0 / 9Eligibility criteria met

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsLead Sponsor

557 Previous Clinical Trials

32,870 Total Patients Enrolled

Cole Sterling, MDPrincipal InvestigatorJohns Hopkins University

Douglas Gladstone, MDPrincipal InvestigatorJohns Hopkins University

2 Previous Clinical Trials

72 Total Patients Enrolled

Media Library

Cyclophosphamide (Bone Marrow Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT05298358 — Phase 1

Systemic Sclerosis Research Study Groups: RIC- alloBMT with high PTCy in SSc

Systemic Sclerosis Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT05298358 — Phase 1

Cyclophosphamide (Bone Marrow Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05298358 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

To whom is this clinical research accessible?

"This medical trial requires that applicants possess progressive systemic sclerosis and are between 18 to 65 years of age. At the moment, roughly 30 people need to be recruited for participation."

Answered by AI

Does this clinical research endeavor accept participants aged 20 and above?

"This clinical trial is only open to individuals aged 18-65. Those under 18 have 47 trials available, whereas those over 65 can participate in 353 different medical studies."

Answered by AI

How many participants is the trial accommodating at this time?

"Affirmative. Clinicaltrials.gov's data implies that this medical trial is recruiting participants at present; the study was originally listed on November 18th 2022 and updated most recently on January 9th 2023. A total of 30 patients are required for one site to complete the research."

Answered by AI

Has the FDA granted approval for RIC-alloBMT with high PTCy in SSc?

"Given that this is a first-phase trial, with limited clinical evidence to support safety and efficacy of RIC-alloBMT with high PTCy in SSc, our team at Power has rated it as 1 on the risk scale."

Answered by AI

Are recruitment efforts ongoing for this research project?

"Affirmative. The information on clinicaltrials.gov implies that the recruitment phase of this trial is currently underway, having first been posted on November 18th 2022 and most recently edited January 9th 2023. A total of 30 participants are needed from a single research centre."

Answered by AI

What are the hoped-for outcomes of this research endeavor?

"This medical trial, running for about 12 months in total, is examining how much Disease relapse or progression occurs. Secondary goals include Event free survival- increased mRSS scoring in diffuse scleroderma patients (number of participants initially diagnosed with this disease whose mRSS score increases by 5 points), Event free survival- transplant related death (how many participants die due to the transplant) and Event free survival- pulmonary function assessment (those who suffer a 10% decrease in FVC or both 5-10% drop plus 15% reduction in DLCO)."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

RIC alloBMT With Post-transplant Cyclophosphamide for Refractory Systemic Sclerosis

2022. "RIC alloBMT With Post-transplant Cyclophosphamide for Refractory Systemic Sclerosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05298358.

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