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Critical Limb IschemiaPhase 1/2NCT04661644

Efficacy and Safety of Mesenchymal Stem Cell Clusters in Patients with Critical Limb Ischemia

A Phase 1/2 study testing Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^7 cells/1 mL/vial) and Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^8 cells/1 mL/vial) in people with Critical Limb Ischemia, run by S.Biomedics Co., Ltd. at 1 site in South Korea.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This clinical trial is designed as a Phase 1/2a clinical trial targeting patients with critical limb ischemia. The trial is composed of Phase 1 to assess the tolerability and safety and Phase 2a to assess the safety and efficacy of the investigational product(A cluster of adipose-derived mesenchymal stem cells (3D-A)… Read more about this study

Who is it for?
Ages 19 years and older
Conditions the study is for
Critical Limb Ischemia
What is being tested?
Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^7 cells/1 mL/vial) and Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^8 cells/1 mL/vial)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteSouth Korea
Study dates
4 Nov 2020to 1 Jul 2024
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 19 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adults aged 19 or older
  • A person diagnosed with critical limb ischemia due to peripheral artery stenosis or obstructive occlusive disease (Rutherford category 4, 5, 6)
  • Patients with critical limb ischemia where symptoms do not improve even after medication treatment for more than 3 months[1]
  • Persons who voluntarily agreed to participate in this clinical trial

Who cannot join

  • Persons whose life expectancy is less than 6 months
  • Patients who underwent surgery and interventional procedures (percutaneous vascular intervention, vascular reconstruction, etc.) in the same disease within 3 months of screening
  • Patients in need of interventional procedure or surgery
  • Patients with a history of administration of other cell therapy products
  • Persons who have received systemic immunosuppression treatment within 3 months of screening
  • Persons with a history of a malignant tumor within 5 years of screening (However, non-metastatic basal cell skin carcinoma, squamous cell carcinoma of the skin or carcinoma in situ of the cervix that does not recur for at least 1 year before the registration are excluded)
Show all 18Show fewer
  • Persons with a hematologic disease with major bleeding or bleeding predisposition within 3 months of screening
  • Women who are pregnant, breastfeeding or planning to become pregnant during the clinical trial period or women of childbearing potential who do not use medically acceptable birth control
  • Medically acceptable contraception:
  • Medicine: Oral contraceptives, skin patches or progestin medications (Transplant or injection)
  • Diaphragm: Condoms, diaphragms, intrauterine devices (IUDs), vaginal suppositories
  • Abstinence: Absolute abstinence (However, periodic abstinence (e.g., calendar method, ovulation method, sympto-thermal method) and control are not considered acceptable contraceptive methods.)
  • Persons who participated in other clinical trials within 3 months of screening
  • Persons who were administered prohibited concomitant medications related to this clinical trial
  • Persons with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels more than twice the normal upper limit at the time of screening
  • Persons identified with estimated glomerular filtration rate (eGFR) levels < 30 mL/min/1.73 m2 at the time of screening
  • Persons with a history of allergies or hypersensitivity to the investigational product or its components
  • Persons who are judged to be inadequate to participate in clinical trials by other investigators

Trial locations and contacts

1 site in 1 country.

South Korea1 site
  • Samsung Medical Center

    Seoul, Seoul, 06351

Other trials in South Korea

About this study

Official title: A Phase 1/2a Clinical Trial to Evaluate the Efficacy and Safety of Allogenic Adipose Tissue-derived Mesenchymal Stem Cell Clusters in Patients with Critical Limb Ischemia

This clinical trial is designed as a Phase 1/2a clinical trial targeting patients with critical limb ischemia. The trial is composed of Phase 1 to assess the tolerability and safety and Phase 2a to assess the safety and efficacy of the investigational product(A cluster of adipose-derived mesenchymal stem cells (3D-A) (cluster of adipose- derived mesenchymal stem cells)) and proceeds in that order.

Read the detailed description

This clinical trial is designed as a Phase 1/2a clinical trial targeting patients with critical limb ischemia. The trial is composed of Phase 1 to assess the tolerability and safety and Phase 2a to assess the safety and efficacy of the investigational product.

Phase 1: Open-label, 3 + 3 design, single-center This phase is conducted in a 3 + 3 design by dose level to evaluate the tolerability of the investigational product.

If the subject voluntarily agrees signs the informed consent formin writing to participate in this clinical trial, the subjects who had necessary check-ups and tests and meet the inclusion criteria after subject conformity assessment are registered in the clinical trial.The subjects receive the investigational product once and are checked for adverse events through day 1 and then discharged. The safety and efficacy assessments are conducted through visits at week 4, week 12, and week 24 after administration.

Subjects are evaluated for adverse events up to week 4 after the administration of the investigational product by registering 3 subjects each at the dosing phase. This evaluation is conducted according to the number of persons subjects who developed Grade 3 or higher adverse drug reactions (ADRs) according to CTCAE Version_5.0 in which a causal relation with the investigational product cannot be ruled out.

If the maximum tolerated dose (MTD) is determined with the test group 2 (high dose) in the Phase 1 clinical trial, the Phase 2a clinical trial will be conducted with the two groups, test group 2 and test group 1.

However, if adverse events occur in 1 of 6 subjects, the test group 2 will be decided considered as the maximum tolerated dose (MTD), or but only the test group 1 will proceed to Phase 2a.

If adverse events occur in 2 or more subjects out of 6 subjects, a dose smaller than test group 1 (low dose) is determined to be the maximum tolerated dose. The Phase 2a clinical trial will not proceed and the clinical trial will end.

Phase 2a: Open-label, sequential assignment, single-center If tolerability and safety are secured 4 weeks after administration of the investigational product of Phase 1, Phase 2a is conducted.

Only those subjects meeting the final inclusion criteria are sequentially assigned, admitted by the same procedure, and administered the investigational product once. Subjects are checked for adverse events through day 1 and discharged. Safety and efficacy assessments are performed at week 4, week 12, and week 24 after administration. All subjects are tested with the same schedule, excluding their doses.

The subjects enrolled in Phase 1 are analyzed for efficacy through visits at week 12 and week 24 so that all subjects (Phase 1 and Phase 2a) are assessed and analyzed for efficacy.

Being tested
Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^7 cells/1 mL/vial), Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^8 cells/1 mL/vial)
Keywords
Adipose-derived mesenchymal stem cells, Critical limb ischemia, Stem cells, Ischemia, S.Biomedics
Medical subject headings
Chronic Limb-Threatening Ischemia, Ischemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^7 cells/1 mL/vial)Biological

    Dose: 1 x 10^7 cells/1 mL/vial (1,000 clusters of adipose-derived mesenchymal stem cells)

  • Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^8 cells/1 mL/vial)Biological

    Dose: 1 x 10^8 cells/1 mL/vial (10,000 clusters of adipose-derived mesenchymal stem cells)

Study groups in NCT04661644
Study groupRoleWhat they receive
Test Group 1 (Low Dose)Experimental- Administration Method: 1 mL is withdrawn from 1 vial of clusters of adipose-derived mesenchymal stem cells, composed of adipose-derived mesenchymal stem cells and diluted with 20 mL of saline, which is divided into 1 cc pre-filled syringes and administered in 20 divided doses to 15-20 parts of the muscle in divided doses along the working areacourse of the tibial artery and peroneal artery below the knee joint (above the lower ischemic area) of the subject (above the lower ischemic area) of the subjects under general anesthesia or spinal anesthesia so that the entire diluted solution is administered.
Receives: Biological: Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^7 cells/1 mL/vial)
Test Group 2 (High Dose)Experimental- Administration Method: 1 mL is withdrawn from 1 vial of clusters of adipose-derived mesenchymal stem cells, composed of adipose-derived mesenchymal stem cells and diluted with 20 mL of saline, then administered to 15-20 parts of the muscle along the working areacourse of the tibial artery and peroneal artery below the knee joint (above the lower ischemic area) of the subject (above the lower ischemic area) of the subjects under general anesthesia or spinal anesthesia.
Receives: Biological: Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^8 cells/1 mL/vial)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Changes in ischemic pain

    Changes in 10 cm VAS (Visual analog score) measurement shall be recorded, and statistically analyzed (mean, median, max, min, STD, average). The degree of change shall be compared between the test groups using two sample t-test or Wilcoxon's rank sum test)

    Measured: The degrees of changes in VAS measurement on week 4, week 12, week 24, compared to the baseline.

  2. Changes in pain-free walking distance

    Changes in maximum walking distance on a treadmill shall be measured, and statistically analyzed(mean, median, max, min, STD, average). The degree of change shall be compared between the test groups using two sample t-test or Wilcoxon's rank sum test)

    Measured: The degrees of changes in pain-free walking distance on week 4, week 12, week 24, compared to the baseline.

  3. Changes in TBI (Toe Brachial Index)

    Changes in TBI (Toe Brachial Index) shall be recorded, and statistically analyzed (mean, median, max, min, STD, average). The degree of change shall be compared between the test groups using two sample t-test or Wilcoxon's rank sum test)

    Measured: The degrees of changes in TBI on week 4, week 12, week 24, compared to the baseline.

  4. Changes in ABI (Ankle Brachial Index)

    Changes in ABI (Ankle Brachial Index) shall be recorded, and statistically analyzed (mean, median, max, min, STD, average). The degree of change shall be compared between the test groups using two sample t-test or Wilcoxon's rank sum test)

    Measured: The degrees of changes in ABI on week 4, week 12, week 24, compared to the baseline.

  5. Change in size of the Ulcer

    The size of the largest ulcer shall be measured and statistically analyzed (mean, median, max, min, STD, average). The degree of change shall be compared between the test groups using two sample t-test or Wilcoxon's rank sum test)

    Measured: The degrees of changes in Ulcer size on week 4, week 12, week 24, compared to the baseline.

  6. Determination of maximum tolerable dose according to DLT occurrence

    Occurrence of adverse drug reactions that are definitely related to the administrated drug are monitored. Maximum tolerable dose is determined when ADR with grade 3 or higher with accordance to CTCAE version 5.0 has occurred to at least one of the three subjects.

    Measured: Occurrence of ADR is monitored throughout the baseline to week 4 during phase 1.

  7. Incidence of abnormal laboratory tests results

    The following laboratory tests are conducted. In case the results are abnormal, it shall be recorded as a adverse event or excluded from study (screening).

    Hematologic Test: WBC, RBC, Hemoglobin, Hematocrit, Platelets count, Blast, Promyelocyte, Metamyelocyte, Neutrophil (Seg, Band), Eosinophil, Basophil, Lymphocyte, Monocyte, Atypical Lymphocyte, Immature cell, Plasma cell, Nucleated RBC, Abnormal lymphoid cell Blood Chemistry Test: Total protein, Albumin, Globulin, Total Bilirubin, AST, ALT, ALP, BUN, Creatinine, Glucose, Uric Acid, Estimated GFR, Ca, P, CRP, CPK Urine Test: Specific gravity, pH, Glucose, Albumin, Bilirubin, Urobilinogen, Ketone, Blood, Nitrite, Leukocyte Esterase

    Measured: Laboratory tests are performed on screening(-4~-2 week), baseline, 1 day after the baseline, and on week 4, week 12, week 24, compared to the baseline.

  8. Incidence of abnormal blood pressure

    Systolic/Diastolic blood pressure is measured after relaxing in sitted position for 5 minutes. In case the results are clinically abnormal, it shall be recorded as an adverse event or excluded from study (screening).

    Measured: Blood pressure is measured throughout the study (on screening visit(4~2 week before the baseline), base line, a day after the baseline, week 4, 12, 24 compared to the baseline.)

  9. Incidence of abnormal temperature

    Temperature is measured via eardrum after relaxing in sitted position for 5 minutes. In case the results are clinically abnormal, it shall be recorded as an adverse event.

    Measured: Temperature is measured throughout the study (on screening visit(4~2 week before the baseline), base line, a day after the baseline, week 4, 12, 24 compared to the baseline.)

  10. Incidence of abnormal physical condition

    Appearance, skin, head, neck, heart, stomach, urology system, reproductive system, limbs, musculoskeletal system, nervous system, lymphatic glands and etc are examined. In case any clinically abnormal condition has been monitored, it shall be recorded as an adverse event.

    Measured: Examined on every visits, throughout the study (on screening visit(4~2 week before the baseline), base line, a day after the baseline, week 4, 12, 24 compared to the baseline.)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Dec 2020
  2. Study start4 Nov 2020
  3. Primary completion22 Apr 2024
  4. Study completion1 Jul 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
Phase 1 is conducted to evaluate the tolerability of the IP Subjects receive the IP once and are checked for adverse events. Subjects are evaluated for adverse events after the administration of the IP by registering 3 subjects each at the dosing phase. If the maximum tolerated dose (MTD) is determined with the test group 2 (high dose) in the Phase 1, the Phase 2a clinical trial will be conducted. However, if 1 adverse events occur, the test group 2 will be considered as the maximum tolerated dose (MTD), or but only the test group 1 will proceed to Phase 2a. If 2 adverse events occur, a dose smaller than test group 1 (low dose) is determined to be the maximum tolerated dose. The Phase 2a clinical trial will not proceed and the clinical trial will end. If tolerability and safety are secured, subjects meeting the final inclusion criteria are assigned to Phase 2a, admitted by the same procedure, and administered the IP once. Safety and efficacy assessments are performed.
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04661644
Sponsor's protocol ID
SB-AC-001
Status last verified
Aug 2024
First submitted
20 Nov 2020
Last updated
30 Aug 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Isner JM, Baumgartner I, Rauh G, Schainfeld R, Blair R, Manor O, Razvi S, Symes JF. Treatment of thromboangiitis obliterans (Buerger's disease) by intramuscular gene transfer of vascular endothelial growth factor: preliminary clinical results. J Vasc Surg. 1998 Dec;28(6):964-73; discussion 73-5. doi: 10.1016/s0741-5214(98)70022-9.

    PubMed 9845647
  • Bura A, Planat-Benard V, Bourin P, Silvestre JS, Gross F, Grolleau JL, Saint-Lebese B, Peyrafitte JA, Fleury S, Gadelorge M, Taurand M, Dupuis-Coronas S, Leobon B, Casteilla L. Phase I trial: the use of autologous cultured adipose-derived stroma/stem cells to treat patients with non-revascularizable critical limb ischemia. Cytotherapy. 2014 Feb;16(2):245-57. doi: 10.1016/j.jcyt.2013.11.011.

    PubMed 24438903
  • Powell RJ, Goodney P, Mendelsohn FO, Moen EK, Annex BH; HGF-0205 Trial Investigators. Safety and efficacy of patient specific intramuscular injection of HGF plasmid gene therapy on limb perfusion and wound healing in patients with ischemic lower extremity ulceration: results of the HGF-0205 trial. J Vasc Surg. 2010 Dec;52(6):1525-30. doi: 10.1016/j.jvs.2010.07.044.

    PubMed 21146749
  • Lee HC, An SG, Lee HW, Park JS, Cha KS, Hong TJ, Park JH, Lee SY, Kim SP, Kim YD, Chung SW, Bae YC, Shin YB, Kim JI, Jung JS. Safety and effect of adipose tissue-derived stem cell implantation in patients with critical limb ischemia: a pilot study. Circ J. 2012;76(7):1750-60. doi: 10.1253/circj.cj-11-1135. Epub 2012 Apr 12.

    PubMed 22498564
  • Murphy MP, Lawson JH, Rapp BM, Dalsing MC, Klein J, Wilson MG, Hutchins GD, March KL. Autologous bone marrow mononuclear cell therapy is safe and promotes amputation-free survival in patients with critical limb ischemia. J Vasc Surg. 2011 Jun;53(6):1565-74.e1. doi: 10.1016/j.jvs.2011.01.074. Epub 2011 Apr 22.

    PubMed 21514773
  • Lasala GP, Silva JA, Minguell JJ. Therapeutic angiogenesis in patients with severe limb ischemia by transplantation of a combination stem cell product. J Thorac Cardiovasc Surg. 2012 Aug;144(2):377-82. doi: 10.1016/j.jtcvs.2011.08.053. Epub 2011 Nov 12.

    PubMed 22079876
  • Kawamoto A, Katayama M, Handa N, Kinoshita M, Takano H, Horii M, Sadamoto K, Yokoyama A, Yamanaka T, Onodera R, Kuroda A, Baba R, Kaneko Y, Tsukie T, Kurimoto Y, Okada Y, Kihara Y, Morioka S, Fukushima M, Asahara T. Intramuscular transplantation of G-CSF-mobilized CD34(+) cells in patients with critical limb ischemia: a phase I/IIa, multicenter, single-blinded, dose-escalation clinical trial. Stem Cells. 2009 Nov;27(11):2857-64. doi: 10.1002/stem.207.

    PubMed 19711453
  • Ruiz-Salmeron R, de la Cuesta-Diaz A, Constantino-Bermejo M, Perez-Camacho I, Marcos-Sanchez F, Hmadcha A, Soria B. Angiographic demonstration of neoangiogenesis after intra-arterial infusion of autologous bone marrow mononuclear cells in diabetic patients with critical limb ischemia. Cell Transplant. 2011;20(10):1629-39. doi: 10.3727/096368910X0177.

    PubMed 22289660
  • Benoit E, O'Donnell TF, Patel AN. Safety and efficacy of autologous cell therapy in critical limb ischemia: a systematic review. Cell Transplant. 2013;22(3):545-62. doi: 10.3727/096368912X636777. Epub 2012 Mar 28.

    PubMed 22490340
  • Cacione DG, do Carmo Novaes F, Moreno DH. Stem cell therapy for treatment of thromboangiitis obliterans (Buerger's disease). Cochrane Database Syst Rev. 2018 Oct 31;10(10):CD012794. doi: 10.1002/14651858.CD012794.pub2.

    PubMed 30378681
  • Criqui MH, Fronek A, Barrett-Connor E, Klauber MR, Gabriel S, Goodman D. The prevalence of peripheral arterial disease in a defined population. Circulation. 1985 Mar;71(3):510-5. doi: 10.1161/01.cir.71.3.510.

    PubMed 3156006
  • Selvin E, Erlinger TP. Prevalence of and risk factors for peripheral arterial disease in the United States: results from the National Health and Nutrition Examination Survey, 1999-2000. Circulation. 2004 Aug 10;110(6):738-43. doi: 10.1161/01.CIR.0000137913.26087.F0. Epub 2004 Jul 19.

    PubMed 15262830
  • Hirsch AT, Criqui MH, Treat-Jacobson D, Regensteiner JG, Creager MA, Olin JW, Krook SH, Hunninghake DB, Comerota AJ, Walsh ME, McDermott MM, Hiatt WR. Peripheral arterial disease detection, awareness, and treatment in primary care. JAMA. 2001 Sep 19;286(11):1317-24. doi: 10.1001/jama.286.11.1317.

    PubMed 11560536
  • Kinlay S. Management of Critical Limb Ischemia. Circ Cardiovasc Interv. 2016 Feb;9(2):e001946. doi: 10.1161/CIRCINTERVENTIONS.115.001946.

    PubMed 26858079
  • Dohmen A, Eder S, Euringer W, Zeller T, Beyersdorf F. Chronic critical limb ischemia. Dtsch Arztebl Int. 2012 Feb;109(6):95-101. doi: 10.3238/arztebl.2012.0095. Epub 2012 Feb 10.

    PubMed 22396707

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^7 cells/1 mL/vial) and Clusters of adipose-derived mesenchymal stem cells (Dose: 1 x 10^8 cells/1 mL/vial) in people with Critical Limb Ischemia, run by S.Biomedics Co., Ltd. at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Changes in ischemic pain (time frame: The degrees of changes in VAS measurement on week 4, week 12, week 24, compared to the baseline.). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04661644, which also lists 38 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Aug 2024.

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