Scleroderma Treatment With Celution Processed Adipose Derived Regenerative Cells (STAR) (STAR)

Scleroderma Treatment With Celution Processed Adipose Derived Regenerative Cells (STAR): A Randomized, Double-Blind, Placebo-Controlled Trial With Incomplete Crossover

The primary objective of this study is to assess the safety and efficacy of the Celution Device in the processing of an autologous graft consisting of adipose derived regenerative cells (ADRCs) in the treatment of hand dysfunction due to scleroderma.

Study Overview

• Status: Completed
• Conditions: Systemic Sclerosis; Scleroderma; Raynaud Phenomena; Raynaud's Disease
• Intervention / Treatment: Other: Placebo; Device: Celution Device

Detailed Description

The STAR Trial is a prospective, randomized, multi-center device trial intended to assess safety and efficacy of subcutaneous administration of Celution processed ADRCs into fingers of patients with hand dysfunction due to scleroderma.

Following informed consent and screening evaluations, eligible subjects will undergo pre-operative testing. Subjects will then undergo fat harvest through small volume liposuction under local anesthesia. Lipoaspirate will be processed in the Celution System to isolate and concentrate ADRCs for immediate subcutaneous administration under local anesthesia. Subjects will be randomly assigned to receive ADRCs (40 million cells) or a visually-matched placebo in a 1:1 ratio. All subjects will receive subcutaneous administration of test substance (ADRC or placebo) into all fingers of both hands. Following completion of all 48 week visits and database lock, placebo treated subjects will be offered treatment with their ADRCs cells should they continue to qualify according to the inclusion/exclusion criteria and should they wish to be treated with ADRCs and agree to the fat harvest procedure.

• Study Type: Interventional
• Enrollment (Actual): 88
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85253
      • Arizona Arthritis and Rheumatology Research, PLLC
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Baptist Health Center for Clinical Research
  • California
    • Los Angeles, California, United States, 90095
      • UCLA David Geffen School of Medicine
    • Los Angeles, California, United States, 90045
      • Arthritis Associates of Southern California
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
      • Georgetown University Hospital
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Kansas
    • Wichita, Kansas, United States, 67207
      • Heartland Research Associates
  • Kentucky
    • Lexington, Kentucky, United States, 40509
      • Central Kentucky Research Associates, Inc.
  • Maryland
    • Baltimore, Maryland, United States, 21224
      • Johns Hopkins University
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Boston University Medical Center
  • Michigan
    • Grand Rapids, Michigan, United States, 49546
      • West Michigan Rheumatology, PLLC
  • Missouri
    • Kansas City, Missouri, United States, 64114
      • Center for Pharmaceutical Research
  • New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Robert Wood Johnson Medical School
  • New York
    • New York, New York, United States, 10021
      • Hospital for Special Surgery
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15261
      • University of Pittsburgh
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas Houston Medical School

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

1. Males or females ≥ 18 and ≤ 70 years of age
2. Diagnosis of diffuse cutaneous scleroderma (duration > 5 years) or limited cutaneous scleroderma.
3. Cochin score ≥ 20 units
4. Ability to safely undergo liposuction
5. Symptoms consistent with Raynaud's Phemomena
6. Compliant with standard preventative recommendations

Key Exclusion Criteria:

1. Body Mass Index < 18 kg/m2
2. Active infection in any finger during screening period or infection in any finger requiring antibiotics in the 30 days prior to the Screening Visit
3. Active infection at the potential site(s) of fat harvest during the screening period
4. Contractures of any finger or ulceration at point of injection precluding completion of injection procedure
5. Amputation of any finger proximal to the proximal interphalangeal joint or any amputation in more than one finger
6. Diagnosis of Rheumatoid Arthritis
7. Inflammatory arthritis of the hand, including significant osteoarthritis, that is not due to scleroderma or significant acute inflammation in the hand that is due to scleroderma, as per the investigator's clinical judgment
8. Oral cyclophosphamide exceeding 2 mg/kg/day, any intravenous cyclophosphamide, methotrexate exceeding 25 mg/week, mycophenolate mofetil exceeding 3 gm/day, hydoxychloroquine exceeding 7 mg/kg/day or azathioprine exceeding 300 mg/day or any other immunosuppressive medication in the 90 days prior to the Screening Visit

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Adipose Derived Regenerative Cells; Adipose Derived Regenerative Cells (ADRCs) processed by the Celution Device 40,000,000 ADRCs administered in 2 injections per digit on both hands.	Device: Celution Device; ADRCs prepared using the investigational Celution Device
PLACEBO_COMPARATOR: Placebo; Sterile Lactated Ringers Solution mixed with small amount of study subject's own freshly drawn blood and administered in 2 injections per digit on both hands.	Other: Placebo; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Cochin score	24 Weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Cochin score	48 Weeks
Raynaud's Condition Score	24 Weeks
Scleroderma Health Assessment Questionnaire (SHAQ)	24 Weeks

Other Outcome Measures

Outcome Measure	Time Frame
Cochin Score	up to 48 Weeks
Raynaud's Condition Score	up to 48 Weeks
Scleroderma Health Assessment Questionnaire (SHAQ)	up to 48 Weeks
Physician and Patient Global Assessment	up to 48 Weeks
Hand Mobility in Scleroderma (HAMIS)	up to 48 Weeks
Digital ulcer count	up to 48 Weeks
Modified Rodnan Score	up to 48 Weeks
Grip strength and pinch strength	up to 48 Weeks
Finger circumference (with hand volume)	up to 48 Weeks
1st corner distance and sum of 2nd, 3rd and 4th corner distances	up to 48 Weeks
The EuroQOL five dimensions questionnaire (EQ-5D)	up to 48 Weeks
Adverse events, Serious Adverse Events	up to 48 Weeks

Collaborators and Investigators

• Sponsor: Cytori Therapeutics
• Investigators: Study Director: Steven Kesten, MD, Cytori Therapeutics

Publications and helpful links

General Publications

• Khanna D, Caldron P, Martin RW, Kafaja S, Spiera R, Shahouri S, Shah A, Hsu V, Ervin J, Simms R, Domsic RT, Steen V, Hummers LK, Derk C, Mayes M, Chatterjee S, Varga J, Kesten S, Fraser JK, Furst DE. Adipose-Derived Regenerative Cell Transplantation for the Treatment of Hand Dysfunction in Systemic Sclerosis: A Randomized Clinical Trial. Arthritis Rheumatol. 2022 Aug;74(8):1399-1408. doi: 10.1002/art.42133. Epub 2022 Jun 27.

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 15, 2015
• Primary Completion (ACTUAL): September 19, 2017
• Study Completion (ACTUAL): May 22, 2018

Study Registration Dates

• First Submitted: March 12, 2015
• First Submitted That Met QC Criteria: March 23, 2015
• First Posted (ESTIMATE): March 24, 2015

Study Record Updates

• Last Update Posted (ACTUAL): June 4, 2018
• Last Update Submitted That Met QC Criteria: June 1, 2018
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Connective Tissue Diseases; Peripheral Vascular Diseases; Scleroderma, Systemic; Scleroderma, Diffuse; Scleroderma, Localized; Raynaud Disease
• Other Study ID Numbers: STAR trial

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO