Background/Purpose:  Current medical therapy to improve hand function has had very limited benefit in patients with systemic sclerosis (SSc). Cells present within a person’s own adipose tissue (Adipose Derived Regenerative Cells [ADRCs]) have the potential to improve tissue injury in scleroderma by modulating inflammation, stimulating angiogenesis, improving vasomotor reactivity, and stimulating local repair cells.

Methods:   The STAR Trial is a phase III, pivotal, prospective, randomized (1:1), placebo-controlled, multi-center trial to assess safety and efficacy of subcutaneous administration of ADRCs into fingers of patients with hand dysfunction due to scleroderma. Key inclusion criteria include a classification of SSc, based on 2013 ACR/ EULAR classification criteria and a baseline Cochin Hand Function Scale (CHFS) score ≥20 units (suggesting at least moderate hand impairment). All enrolled patients (ADRC and placebo) undergo fat harvest through small volume liposuction (~200 to 400 mL). Lipoaspirate is processed in the Celution^® System (Cytori Therapeutics, San Diego) to isolate and concentrate ADRCs, which contain CD34+ multipotent cells, pericytes, endothelial cells, and other cellular constituents. Patients then receive subcutaneous administration of 1 mL test substance (ADRC [4 million cells per finger] or matching placebo [Lactated Ringers containing patient’s own blood to visually match the placebo to ADRCs]) into all fingers. Placebo patients may cross-over to open label ADRCs at the end of the trial. Key endpoints include: CHFS, Raynaud’s Condition Score (RCS), Scleroderma Health Assessment Questionnaire (SHAQ), global assessments, Hand Mobility in Scleroderma (HAMIS) test, digital ulcer counts/time to new ulcer, modified Rodnan Score (hands only), grip/pinch strength, and analgesic use. Follow up visits are scheduled for weeks 1, 4, 12, 24, 36, 48 weeks.

Results: Trial enrollment has been completed (n=88) with baseline blinded data available from up to 86 patients. Patient demographics from the cohort indicated a mean age 53.2±10.6 years, female 87%, 77% Caucasian, and %limited/diffuse 42/58. Baseline Cochin score was 42.2±14.2 (0-60) and the baseline RCS was 3.9±2.1 (0-10); 73% of patients had a history of digital ulcers with 39% reported with digital ulcer(s) at baseline. Patients experienced expected adverse events such as liposuction related discomfort and ecchymosis; however, no peri-procedure complications or serious adverse events occurred. No cell related adverse events have been reported. All patients were discharged the same day following fat harvest, cell processing and cell injection. Mean adipose tissue harvest 293±50 mL, ADRC yield 120.6±66 x 10⁶ cells, ADRC/gm adipose tissue 3.97±1.82 x 10⁵, ADRC viability 89.1±2.7%. Data continues to be collected in the follow-up period.

Conclusion: The STAR trial demonstrates same day fat harvest, cell processing and subcutaneous injection of ADRCs to all fingers in patients with scleroderma is feasible and can be performed safely.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/feasibility-of-same-day-adipose-tissue-harvest-cell-processing-and-subcutaneous-delivery-of-adipose-derived-regenerative-cells-into-fingers-of-scleroderma-patients-within-a-randomized-double-blind-cl/