Autologous muscle derived stem cell therapy

Unassigned

New Medicines

Urinary stress incontinence in women

Information

Advanced therapy medicinal product (ATMP)
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Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials

Category

Autologous stem cell-based therapy derived from skeletal muscle biopsies
UK prevalence rates of urinary incontinence are between 5-69% in women
Urinary stress incontinence in women
Transurethral injection

Trial or other data

Apr 20 · PIII CELLEBRATE study is recruiting. Trial sites in the UK now include St. James ´ University Hospital in Leeds as well as Derriford Hospital in Plymouth. Primary outcome is the number of leaks due to stress incontinence episodes over 12 months; collection of these data is due to complete Sep 22 [8].
Dec 19 · UK trial sites; NCT03104517 (Plymouth) [7]
Dec 19 · NCT01893138 due Oct 19, NCT03104517 due Sept 22 [7]
Dec 18 · NCT01893138 data now due July 19; NCT03104517 now due Sep 2022. NCT01382602 has reported results; Percentage of Participants With Response (Based on Stress IEF, or In-office Pad Weight Test, or 24-hour Pad Weight Test) at 12 Months was 82 (95% CI 73 to 90) for intervention vs. 90 (78 to 97) for placebo [6].
Sep 17 · PIII study (NCT01893138) has finished recruiting and collection of primary outcome data is now expected Dec 18. PIII CELLEBRATE study (NCT03104517) is due to complete collection of primary outcome data in Aug 19 [5].
Jul 17 · NCT01382602 is complete but no results are available. Another PIII study is due to start NCT03104517 with a primary completion date of 2019 [4]
Nov 15 · NCT01382602 is no longer recruiting patients but remains active. Data collection for primary outcomes completed in Jan 16 and the study is due for completion in Jan 17 [2].
Oct 15 · NCT01893138 is active and recruiting. Timelines are slightly set back with primary outcome data expected in Dec 17, the study is expected to complete in Dec 2018 [2].
Feb 14 · Two PIII trials evaluating efficacy in reducing stress leaks and safety against placebo are ongoing (NCT01893138 & NCT01382602). Each study will enrol 267 women with SUI of ≥6 months duration. Inclusion criteria include previously attempted conservative treatment; ≤2 episodes of awakening to void during normal sleeping hours; no symptoms of pure urge, or mixed urinary incontinence where urge incontinence is the predominant factor. Women will be randomised to AMDC-USR 150 (±20%) x 106 cells or placebo, both via transurethral injection at baseline, 1, 3, 6, 12 and 24 months. The primary outcome is ≥50% reduction in stress leaks at one year. Studies are expected to complete in 2H 2017 [1].

Evidence based evaluations

Faecal incontinence

Information

Advanced therapy medicinal product (ATMP)
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Development and Regulatory status

Phase II Clinical Trials
None
None

Category

Autologous stem cell-based therapy derived from skeletal muscle biopsies
Faecal incontinence is the inability to control or the involuntary discharge of stool or gas. It is estimated to affect 8-9% of the population. Middle-aged women and nursing home residents are more commonly affected. Advancing age, diarrhoea, urinary incontinence and multiple childbirths, particularly vaginal delivery with sphincter tear, are independent risk factors [1].
Faecal incontinence
Intramuscular

Trial or other data

Jul 19 · PI/II trial (NCT01600755) is recruiting [2].
May 12 · PI/II trial to investigate the safety and feasibility of autologous muscle derived stem cell therapy injection into the anal sphincter for treatment of patients with faecal incontinence starts (NCT01600755). 50 adults will be recruited in Canada and the UK (Royal London Hospital). Primary outcome is incidence of treatment-related adverse events; secondary outcome measures are frequency of incontinent episodes, incontinence score, sphincter pressure and faecal incontinence quality of life scale (FIQL). The trial is due to complete May 20 [2].