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NCT01733186

Evaluation of Safety and Exploratory Efficacy of CARTISTEM®, a Cell Therapy Product for Articular Cartilage Defects

Completed Phase 1, PHASE2 Results posted Last updated 29 January 2026
What this trial tests

Phase 1, PHASE2 trial testing CARTISTEM® in Degeneration Articular Cartilage Knee in 12 participants. Completed in 29 August 2017.

Timeline
7 January 2013
Primary endpoint
2 June 2017
29 August 2017

Quick facts

Lead sponsorMedipost, Inc.
PhasePhase 1, PHASE2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment12
Start date7 January 2013
Primary completion2 June 2017
Estimated completion29 August 2017
Sites2 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Medipost, Inc. — full company profile →

Who can join

18 and older, any sex, with Degeneration Articular Cartilage Knee. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

IKDC Score Primary · 12 months

Subjects underwent a self-assessment of knee function using the IKDC (International Knee Documentation Committee Assessment)subjective knee evaluation before and after the administration of the IP. The assessment is designed to detect improvement or deterioration in symptoms, function, and sports activities due to knee impairment. The possible score ranges from 0 to 100, where 100 = no limitation with daily or sporting activities and the absence of symptoms. The IKDC assessment was performed before IP administration and at Months 12 after IP administration in an exploratory fashion. The IKDC

GroupValue95% CI
Dose A Cohort32.0± 27.7
Dose B Cohort25.5± 18.9
VAS Secondary · 24 months

Subjects underwent a self-assessment of joint pain using the 100-mm VAS (Visual Analogue Scale) before and at Months 12 and 24 after IP (investigational product) administration. Higher values represent worse joint pain. Use of NSAIDs and any other analgesics had to be discontinued for 48 hours and 24 hours, respectively, prior to the 100 mm VAS evaluation. The outcome was presented with the post-operative change amount at Months 12 and 24 from the baseline. 100-mm VAS scores range from 0 to 100. Higher values represent worse joint pain.

12 MONTHS
GroupValue95% CI
Dose A Cohort-23.8± 22.9
Dose B Cohort-12.5± 31.5
24 MONTHS
GroupValue95% CI
Dose A Cohort-31.7± 18.0
Dose B Cohort-11.3± 30.2
Lysholm Score Secondary · 24 months

The Lysholm knee scoring scale is a self-assessment that rates the severity of common complaints related to knee problems, such as pain, swelling, abnormal sensations, and ability to squat or climb stairs. The possible score ranges from 0 to 100, where 100 = no symptoms or disability. Scores are categorized as excellent (95 to 100), good (84 to 94), fair (65 to 83), and poor (≤64). Subjects underwent a self-assessment of knee function using the Lysholm score before IP administration and at Months 12 and 24 after IP administration. The outcome was presented with the post-operative change amount

12 MONTHS
GroupValue95% CI
Dose A Cohort26.7± 20.5
Dose B Cohort22.5± 18.3
24 MONTHS
GroupValue95% CI
Dose A Cohort32.0± 20.9
Dose B Cohort10.2± 13.0
KOOS Score Secondary · 24 months

The Knee Injury and Osteoarthritis Outcome score (KOOS) knee survey is a self assessment of knee function and knee-related quality of life (QOL). Subjects responded to each question regarding knee-related symptoms, swelling, pain, impaired function, or changes in QOL with 1 of 5 possible answers that range from "never/not at all" to "always/extremely." The possible score for each parameter ranges from 0 to 100, where 0=extreme problems and 100=no problems. Subjects completed the KOOS knee survey before IP (investigational product) administration and at Month 24 after IP administration. The out

ADL (24 MONTHS)
GroupValue95% CI
Dose A Cohort28.2± 25.7
Dose B Cohort12.3± 19.2
PAIN (24 MONTHS)
GroupValue95% CI
Dose A Cohort23.6± 24.3
Dose B Cohort15.8± 13.1
QOL (24 MONTHS)
GroupValue95% CI
Dose A Cohort50.0± 36.4
Dose B Cohort19.8± 24.2
SPORTS (24 MONTHS)
GroupValue95% CI
Dose A Cohort39.2± 37.2
Dose B Cohort36.7± 31.9
SYMPTOM (24 MONTHS)
GroupValue95% CI
Dose A Cohort24.4± 19.9
Dose B Cohort8.3± 12.3
IKDC Score Secondary · 24 months

Subjects underwent a self-assessment of knee function using the IKDC subjective knee evaluation before and after the administration of the IP. The assessment is designed to detect improvement or deterioration in symptoms, function, and sports activities due to knee impairment. The possible score ranges from 0 to 100, where 100 = no limitation with daily or sporting activities and the absence of symptoms. The IKDC assessment was performed before IP administration and at Month 24 after IP administration in an exploratory fashion. The outcome was presented with the post-operative change amount at

GroupValue95% CI
Dose A Cohort41.2± 25.8
Dose B Cohort27.5± 20.9

Adverse events — posted to ClinicalTrials.gov

Time frame: 24 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Dose A Cohort
Serious: 0/6 (0%)
Deaths: 0/6
Dose B Cohort
Serious: 0/6 (0%)
Deaths: 0/6
Other adverse events (21 terms — click to expand)

ReactionSystemDose A CohortDose B Cohort
JOINT RANGE OF MOTION DECREASEDMusculoskeletal and connective tissue disorders
arthralgiaMusculoskeletal and connective tissue disorders
muscle atrophyMusculoskeletal and connective tissue disorders
joint effusionMusculoskeletal and connective tissue disorders
joint swellingMusculoskeletal and connective tissue disorders
prehypertensionVascular disorders
bone marrow oedemaBlood and lymphatic system disorders
joint crepitationMusculoskeletal and connective tissue disorders
sinusitisInfections and infestations
procedural painInjury, poisoning and procedural complications
soft tissue injuryInjury, poisoning and procedural complications
blood creatine phosphokinase increasedInvestigations
blood pressure increasedInvestigations
nuclear magnetic resonance imaging abnormalInvestigations
chondromalaciaMusculoskeletal and connective tissue disorders
chondropathyMusculoskeletal and connective tissue disorders
exostosisMusculoskeletal and connective tissue disorders
haemarthrosisMusculoskeletal and connective tissue disorders
joint lockMusculoskeletal and connective tissue disorders
plica syndromeMusculoskeletal and connective tissue disorders
tendonitisMusculoskeletal and connective tissue disorders

Data from ClinicalTrials.gov NCT01733186 adverse events section.

Sponsor's own description

The purpose of this study is to determine whether CARTISTEM, a cell therapeutic product, is safe and effective in the treatment of articular cartilage defects of the knee as a result of ageing, trauma, or degenerative diseases.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. The use of mesenchymal stem cells for cartilage repair and regeneration: a systematic review.
    Goldberg A, Mitchell K, Soans J, Kim L, et al · · 2017 · cited 159× · PMID 28279182 · DOI 10.1186/s13018-017-0534-y
  2. Engineering Stem and Stromal Cell Therapies for Musculoskeletal Tissue Repair.
    Loebel C, Burdick JA. · · 2018 · cited 105× · PMID 29429944 · DOI 10.1016/j.stem.2018.01.014
  3. Culture-expanded mesenchymal stromal cell therapy: does it work in knee osteoarthritis? A pathway to clinical success.
    Copp G, Robb KP, Viswanathan S. · · 2023 · cited 83× · PMID 37095295 · DOI 10.1038/s41423-023-01020-1
  4. Current perspectives in stem cell research for knee cartilage repair.
    Orth P, Rey-Rico A, Venkatesan JK, Madry H, et al · · 2014 · cited 80× · PMID 24520197 · DOI 10.2147/sccaa.s42880
  5. Mesenchymal stem cell therapy for osteoarthritis: current perspectives.
    Wyles CC, Houdek MT, Behfar A, Sierra RJ. · · 2015 · cited 71× · PMID 26357483 · DOI 10.2147/sccaa.s68073
  6. Autologous, allogeneic, induced pluripotent stem cell or a combination stem cell therapy? Where are we headed in cartilage repair and why: a concise review.
    Vonk LA, de Windt TS, Slaper-Cortenbach IC, Saris DB. · · 2015 · cited 56× · PMID 25976213 · DOI 10.1186/s13287-015-0086-1
  7. Good manufacturing practice-compliant isolation and culture of human umbilical cord blood-derived mesenchymal stem cells.
    Pham PV, Vu NB, Pham VM, Truong NH, et al · · 2014 · cited 43× · PMID 24565047 · DOI 10.1186/1479-5876-12-56
  8. Cell therapy for cartilage repair.
    Hulme CH, Perry J, McCarthy HS, Wright KT, et al · · 2021 · cited 35× · PMID 34423830 · DOI 10.1042/etls20210015

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