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 range8 Years – 60 Years
SexALL
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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)
✕. O2 sat \<92% at rest in room air
✕. Estimated CrCl \<40 mL/min,using Cockcroft-Gault formula based on actual body weight.
✕. Active, untreated SSc renal crisis at the time of consent.
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)