Injection of Autologous Adipose-derived Stromal Vascular Fraction in the Finger of Systemic Sclerosis Patients

Safety, Tolerability and Potential Efficacy of Injection of Autologous Adipose-derived Stromal Vascular Fraction in the Finger of Systemic Sclerosis Patients

This study outlines the safety of the autologous SVF cells injection in the hands of patients with SSc. Preliminary assessments at 6 months will suggest potential efficacy needing confirmation in a randomised placebo-controlled trial on a larger population

Study Overview

• Status: Completed
• Conditions: Systemic Sclerosis
• Intervention / Treatment: Procedure: SVF injection

Detailed Description

In patients with systemic sclerosis(scleroderma, SSc), impaired hand function greatly contributes to disability and reduced quality of life, and is insufficiently relived by currently available therapies. Adipose tissue-derived stromal vascular fraction (SVF) is increasingly recognized as an easily accessible source of regenerative cells with therapeutic potential in ischaemic or autoimmune disease. The investigators aimed to measure for the first time the safety, tolerability, and potential efficacy of autologous SVF cells local injections in patients with SSc with hand disability.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• age over 18 year old
• Cochin Hand Function Scale (CHFS) > 20/90

Exclusion Criteria:

• new vasodilators or immunosuppressive therapy for SSc in the 3 months prior to enrolment
• new vasodilators or immunosuppressive therapy for SSc during the 6-month follow-up
• clinical or radiological signs of digital infection
• positive status for HIV
• positive status for hepatitis B or C
• positive status for human T-cell leukemia virus 1-2
• positive status for syphilis
• pregnancy
• BMI less than 17kg/m2

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: SVF injection; SVF was obtained from lipoaspirates, using an automated processing system, and subsequently injected into the subcutaneous tissue of each finger in contact with neurovascular pedicles	Procedure: SVF injection; SVF is increasingly recognised as an easily accessible source of regenerative cells with therapeutic potential in ischaemic or autoimmune disease. We aimed to measure for the first time the safety, tolerability and potential efficacy of autologous SVF cells local injections in patients with systemic sclerosis

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Raynaud's Condition Scale	The severity of Raynaud's phenomenon was assessed using the Raynaud's condition scale (ranging 0 to 10, with higher scores representing more severe Raynaud's phenomenon)	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Hand Visual Analogue Scale	Hand pain was assessed using the Hand visual analogue scale (ranging 0 to 10, with higher scores representing more severe pain)	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Colchin Hand Function Scale	The Colchin hand function scale (CHFS, ranging 0 to 90, with higher scores representing more deteriorated hand function) scores	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Kapandji Score (Ranging 0 to 10) - Dominant Hand	Kapandji score (ranging 0 to 10) is validated measures in past studies as outcomes for hand disability. The Kapandji score is a tool useful for assessing the opposition of the thumb, based on where on their hand the patient is able to touch with the tip of their thumb. A score of 0 indicates no opposition, a score of 10 indicates maximal opposition.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Kapandji Score (Non-dominant Hand)	Kapandji score (ranging 0 to 10) is validated measures in past studies as outcomes for hand disability. The Kapandji score is a tool useful for assessing the opposition of the thumb, based on where on their hand the patient is able to touch with the tip of their thumb. A score of 0 indicates no opposition, a score of 10 indicates maximal opposition.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Disease-related QOL (Quality of Life) : EuroQol-5 Dimensions Time Trade-off (EQ-5D TTO)	Disease-related QOL (quality of life) was measured by EuroQol-5 dimensions time trade-off (EQ-5D TTO, ranging 0 to 1, with higher values representing better QOL) values	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Disease-related QOL (Quality of Life) : EuroQol Visual Analog Scale (EQ VAS)	Disease-related QOL (quality of life) : EuroQol visual analog scale (EQ VAS, ranging 0 to 100, with higher scores representing better QOL)	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Disease-related QOL (Quality of Life) : Health Assessment Questionnaire (HAQ)	Disease-related QOL (quality of life) : health assessment questionnaire (HAQ, ranging 0 to 3, with higher scores meaning lower QOL)	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
The Degree of Hand Edema (Right)	The finger circumferences of the second to the fifth fingers measured in the middle portion of each proximal phalanx were used to assess the degree of hand edema	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
The Degree of Hand Edema (Left)	The finger circumferences of the second to the fifth fingers measured in the middle portion of each proximal phalanx were used to assess the degree of hand edema	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes of Nailfold Capillary Microscopic Findings (Irregularly Enlarged Capillaries)	In order to assess the severity of microangiopathies in both fingers, a semiquantitative scoring system for nailfold capillary microscopic findings was adopted. A total of six parameters (irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array and capillary ramifications) were measured using the semiquantitative manner on the scale ranging 0 to 3, indicating more severe as scores were higher. Each parameter was scored for four fingers of both hands, except thumbs, and the median score value of each parameter in both hands was finally presented.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Changes of Nailfold Capillary Microscopic Findings (Giant Capillaries)	In order to assess the severity of microangiopathies in both fingers, a semiquantitative scoring system for nailfold capillary microscopic findings was adopted. A total of six parameters (irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array and capillary ramifications) were measured using the semiquantitative manner on the scale ranging 0 to 3, indicating more severe as scores were higher. Each parameter was scored for four fingers of both hands, except thumbs, and the median score value of each parameter in both hands was finally presented.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Changes of Nailfold Capillary Microscopic Findings (Hemorrhages)	In order to assess the severity of microangiopathies in both fingers, a semiquantitative scoring system for nailfold capillary microscopic findings was adopted. A total of six parameters (irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array and capillary ramifications) were measured using the semiquantitative manner on the scale ranging 0 to 3, indicating more severe as scores were higher. Each parameter was scored for four fingers of both hands, except thumbs, and the median score value of each parameter in both hands was finally presented.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Changes of Nailfold Capillary Microscopic Findings (Loss of Capillaries)	In order to assess the severity of microangiopathies in both fingers, a semiquantitative scoring system for nailfold capillary microscopic findings was adopted. A total of six parameters (irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array and capillary ramifications) were measured using the semiquantitative manner on the scale ranging 0 to 3, indicating more severe as scores were higher. Each parameter was scored for four fingers of both hands, except thumbs, and the median score value of each parameter in both hands was finally presented.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Changes of Nailfold Capillary Microscopic Findings (Disorganization of the Vascular Array)	In order to assess the severity of microangiopathies in both fingers, a semiquantitative scoring system for nailfold capillary microscopic findings was adopted. A total of six parameters (irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array and capillary ramifications) were measured using the semiquantitative manner on the scale ranging 0 to 3, indicating more severe as scores were higher. Each parameter was scored for four fingers of both hands, except thumbs, and the median score value of each parameter in both hands was finally presented.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks
Changes of Nailfold Capillary Microscopic Findings (Capillary Ramifications)	In order to assess the severity of microangiopathies in both fingers, a semiquantitative scoring system for nailfold capillary microscopic findings was adopted. A total of six parameters (irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array and capillary ramifications) were measured using the semiquantitative manner on the scale ranging 0 to 3, indicating more severe as scores were higher. Each parameter was scored for four fingers of both hands, except thumbs, and the median score value of each parameter in both hands was finally presented.	Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks

Collaborators and Investigators

• Sponsor: Suk-Ho Moon
• Investigators: Study Director: Suk-Ho Moon, MD, PhD, Seoul St. Mary's Hospital; Principal Investigator: Seung-Ki Kwok, Seoul St. Mary's Hospital

Publications and helpful links

General Publications

• Park Y, Lee YJ, Koh JH, Lee J, Min HK, Kim MY, Kim KJ, Lee SJ, Rhie JW, Kim WU, Park SH, Moon SH, Kwok SK. Clinical Efficacy and Safety of Injection of Stromal Vascular Fraction Derived from Autologous Adipose Tissues in Systemic Sclerosis Patients with Hand Disability: A Proof-Of-Concept Trial. J Clin Med. 2020 Sep 19;9(9):3023. doi: 10.3390/jcm9093023.

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 1, 2018
• Primary Completion (ACTUAL): December 30, 2019
• Study Completion (ACTUAL): December 30, 2019

Study Registration Dates

• First Submitted: November 9, 2016
• First Submitted That Met QC Criteria: February 21, 2017
• First Posted (ACTUAL): February 23, 2017

Study Record Updates

• Last Update Posted (ACTUAL): October 8, 2020
• Last Update Submitted That Met QC Criteria: October 3, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Keywords: mesenchymal stem cells; stromal vascular fraction; hand disability; phase I trial
• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Connective Tissue Diseases; Sclerosis; Scleroderma, Systemic; Scleroderma, Diffuse
• Other Study ID Numbers: SVF treatment in SSc

Plan for Individual participant data (IPD)

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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No