E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The Achilles tendon is the largest tendon in the body and it plays an important role in the biomechanics of the lower extremity.The disease to be studied in the trial is Achilles tendinopathy (AT). Achilles Tendinopathy a degenerative process that leads to pain and dysfunction in middle-aged individuals and sports participants. |
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E.1.1.1 | Medical condition in easily understood language |
Pain of the Achilles Tendon |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000435 |
E.1.2 | Term | Achilles tendon injury |
E.1.2 | System Organ Class | 100000004863 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective is to determine safety by showing that there are no serious adverse reactions to autologous stem cells injected into tendon. |
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E.2.2 | Secondary objectives of the trial |
To show that autologous bone marrow derived, culture expanded stem cells might show clinical efficacy in showing improved clinical outcomes (as measured by clinical scores) and to gain experience in using Ultrasound Tissue Characterisation (UTC) and an assessment of its usefulness as an outcome measurement. As this study is a pilot/feasibility it is not adequately powered to show statistical significance for these variables. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Aged equal to or over 18 and equal to or less than 70 (both males and females) • Participants with chronic midportion AT (as defined by pain in region of AT and tender swelling in mid portion of AT (no tenderness over bony attachment to heel) with symptoms for longer than 6 months who have failed conservative treatment (at least a full course of physiotherapy) and for whom surgery is being considered •Able to provide written informed consent •Females of childbearing age and potential must be willing to use two forms of effective contraception from the time of consent to 6 months post-injection. |
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E.4 | Principal exclusion criteria |
• Previous bony surgery (e.g. reconstructive pelvic osteotomy) at or in proximity to the bone marrow harvest site • Pregnancy or lactation • Current use of steroids, anti-TNF drugs, methotrexate, or ciprofloxacin (or use within 4 weeks of assessment for eligibility) • Positive for HBV, HCV, HIV 1 and 2, syphilis and HTLV • Previous AT surgery on the tendon to receive MSC implantation • Inflammatory arthritis • Known or suspected underlying haematological malignancy • Other active malignancy in the past 3 years • Bovine or antibiotic allergy |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary safety outcome is the incidence rate of Serious adverse Reactions (SARs). This will be expressed as the proportion of participants experiencing a SAR at any time over the 24 week follow-up period. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 0(visit 3), 24 hrs (visit 4), 6weeks (visit 5), 12 weeks (visit 6), 24 weeks (visit 7) |
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E.5.2 | Secondary end point(s) |
To show that autologous bone marrow derived, culture expanded stem cells might show clinical efficacy in showing improved clinical outcomes (as measured by clinical scores) and to gain experience in using Ultrasound Tissue Characterisation (UTC) and an assessment of its usefulness as an outcome measurement. As this study is a pilot/feasibility it is not adequately powered to show statistical significance for these variables. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
baseline, 6 weeks (visit 5), 12 weeks (visit 6), 24 weeks (visit 7) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 1 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 1 |