Clinical trial NCT02396238: Scleroderma Treatment With Celution Processed Adipose Derived Regenerative Cells (STAR)
Official title: Scleroderma Treatment With Celution Processed Adipose Derived Regenerative Cells (STAR): A Randomized, Double-Blind, Placebo-Controlled Trial With Incomplete Crossover
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Scleroderma
Systemic Sclerosis
Raynaud Phenomena
Raynaud's Disease
Lead sponsor: Cytori Therapeutics
Sponsor class: INDUSTRY
First posted: 2015-03-24
Enrollment: 88
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Cochin score
timeFrame: 24 Weeks
Secondary outcomes:
measure: Cochin score
timeFrame: 48 Weeks
measure: Raynaud's Condition Score
timeFrame: 24 Weeks
measure: Scleroderma Health Assessment Questionnaire (SHAQ)
timeFrame: 24 Weeks
Other outcomes:
measure: Cochin Score
timeFrame: up to 48 Weeks
measure: Raynaud's Condition Score
timeFrame: up to 48 Weeks
measure: Scleroderma Health Assessment Questionnaire (SHAQ)
timeFrame: up to 48 Weeks
measure: Physician and Patient Global Assessment
timeFrame: up to 48 Weeks
measure: Hand Mobility in Scleroderma (HAMIS)
timeFrame: up to 48 Weeks
measure: Digital ulcer count
timeFrame: up to 48 Weeks
measure: Modified Rodnan Score
timeFrame: up to 48 Weeks
measure: Grip strength and pinch strength
timeFrame: up to 48 Weeks
measure: Finger circumference (with hand volume)
timeFrame: up to 48 Weeks
measure: 1st corner distance and sum of 2nd, 3rd and 4th corner distances
timeFrame: up to 48 Weeks
measure: The EuroQOL five dimensions questionnaire (EQ-5D)
timeFrame: up to 48 Weeks
measure: Adverse events, Serious Adverse Events
timeFrame: up to 48 Weeks
Locations:
city: Phoenix
country: United States
facility: Arizona Arthritis and Rheumatology Research, PLLC
state: Arizona
city: Little Rock
country: United States
facility: Baptist Health Center for Clinical Research
state: Arkansas
city: Los Angeles
country: United States
facility: Arthritis Associates of Southern California
state: California
city: Los Angeles
country: United States
facility: UCLA David Geffen School of Medicine
state: California
city: Washington
country: United States
facility: Georgetown University Hospital
state: District of Columbia
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Wichita
country: United States
facility: Heartland Research Associates
state: Kansas
city: Lexington
country: United States
facility: Central Kentucky Research Associates, Inc.
state: Kentucky
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston University Medical Center
state: Massachusetts
city: Grand Rapids
country: United States
facility: West Michigan Rheumatology, PLLC
state: Michigan
city: Kansas City
country: United States
facility: Center For Pharmaceutical Research
state: Missouri
city: New Brunswick
country: United States
facility: Robert Wood Johnson Medical School
state: New Jersey
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: University of Texas Houston Medical School
state: Texas
Study officials:
affiliation: Cytori Therapeutics
name: Steven Kesten, MD
role: STUDY_DIRECTOR
References:
citation: 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.