Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis (NCT03630211) | Clinical Trial Compass
RecruitingPhase 2
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
United States8 participantsStarted 2018-07-31
Plain-language summary
The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.
Who can participate
Age range
8 Years – 60 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patient, parent, or legal guardian must have given written informed consent. For patients ≥ 168 years of age who are developmentally able, assent or affirmation will be obtained.
. Age 8-24, inclusive, at time of consent.
. Diagnosed with Systemic Sclerosis (SSc) at the age of ≤19.
. Failure to respond, specifically no improvement or progression of disease, to at least 2 disease-modifying antirheumatic drugs (DMARDS) within 12 months of consent with any of the following conditions:
. Progression of skin thickening over the past 6 months or Modified Rodnan skin score (mRSS) ≥ 20
. Progression of ILD within 18 months prior to consent. Progression to be determined by either of the following:
. Myositis - CPK \> 2x upper limit of normal or MRI consistent with myositis
. Childhood Myositis Assessment Score \< 30
Exclusion criteria
. FVC \<35%, determined by pulmonary function tests for those able to complete spirometry adequately (per investigator's determination)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
High Dose Immunoablative therapy-Safety
Timeframe: Up to 36 months post HSCT
2
Death
Timeframe: Post Transplant through study completion, an average of 36 months
3
Respiratory Failure
Timeframe: Post Transplant through study completion, an average of 36 months
4
Renal Failure
Timeframe: Post Transplant through study completion, an average of 36 months
5
The occurrence of cardiomyopathy
Timeframe: Post Transplant through study completion, an average of 36 months
6
Treatment-related mortality (TRM)
Timeframe: Mobilization through study completion, an average of 36 months
7
High Dose Immunoablative therapy-Treatment Effect
Timeframe: up to 36 months post HSCT (hematopoietic stem cell transplantation)