Anchored muscle cells for incontinence

Plain English summary of protocol

Background and study aims
Faecal incontinence (FI) is a public health problem with a big impact on people’s quality of life. Women who have given birth form a significant proportion of this patient group, as tears to the perineum during childbirth can lead to faecal incontinence. This study will test the safety of a cell-based therapy in women with post-obstetric faecal incontinence.

Who can participate?
Female patients aged 18 years and over with chronic faecal incontinence and a history of obstetric anal sphincter injury.

What does the study involve?
Surgeons will take a muscle biopsy from the patient's chest. This biopsy will be processed to grow muscle cells, which are combined with small porous carrier particles called microcarriers. The combination cell-microcarrier product will be injected into the patient’s external anal sphincter, leading to muscle regeneration.
All the patients will receive the same treatment and all the patients and their doctors are aware of the treatment. The researchers will assess the number of FI episodes, safety and other patient-reported outcomes. Data collected during the patient’s initial visits will be compared with data collected 12 months after treatment to assess how effective treatment is.
Patients will take part in 11 visits.

What are the possible benefits and risks of participating?
The treatment used is an experimental product so it has not yet been authorised/marketed for use by the general public. As this study is a ‘first-in-human’ usage, there is no data to show its safety.
The researchers have reviewed all previous studies of ASMDC (cells without microcarriers). In these studies, which involved a total of 615 patients (in 14 studies), the risks of treatment with cells alone were small. The risks were almost entirely related to the biopsy and injection procedures:
Pain or skin irritation: approx. 1 in 10 (10%)
Minor bruising: approx. 1 in 20 (5%)
Superficial infection: <1 in 20 (<5%)
Muscle weakness at biopsy site: <1 in 100 (<1%)
Bleeding requiring surgical intervention: 1 reported in 615
Complications of routine anaesthesia: No occurrences. Typical risk is <1 in 1000.
Since PLGA microcarriers are a newer treatment, we don’t have studies like this on their safety in treating faecal incontinence. The study team has also done tests in laboratories to check the risks of treatment, and the results have been acceptable. There are also several commercially available products which have been approved for clinical use which use microcarriers in products that are injected into the body.
Muscle biopsy: It is inevitable that some pain will be experienced during the procedure due to the injection of the local anaesthetic and possibly the biopsy itself. There may also be some pain after the procedure. Potential risks include bruising, haematoma (a clot of blood), infection, delayed wound healing and a small amount of scarring. Participants may be provided with simple analgesics as needed.
Product injection: Potential side effects include bleeding, infection, inflammatory response or irritation, pain or discomfort, worsening incontinence, diarrhoea, constipation, blockage of the intestine or faecal retention. There is also a small risk of allergic response to the contents of the product (cells or components of the final formulation), and infection. This is expected to be minimal because the product is made from the participant’s own cells.
Because this study involves an experimental treatment, not all risks and outcomes can be predicted. There may be some risks that the study doctors are not currently aware of. It is possible that some of these unknown risks could be permanent, serious, or life-threatening.
Participant safety will be monitored throughout the trial. Oversight committees (and where appropriate, regulators) will be kept informed of trial safety. Stopping rules will trigger study stop if accruing data indicates a safety concern.
Patients will be provided with a safety card containing contact details of the study team, which they can use to ask questions about the study, or if they are experiencing pain or discomfort and want advice.
There is a chance of product delivery failure. This could happen for a number of reasons, including but not limited to delays to the courier, problems with temperature control, or issues when making the product, which could mean the trial treatment isn’t safe to use. Should the risk occur, where time and resources allow, participants will be offered a second round of muscle biopsy and treatment.
Participants may find trial involvement or questionnaire/ diary completion burdensome. Clinical trial involvement could affect participants’ insurance cover.
This research study may have unknown risks for a pregnancy, and nursing infants. People who are breastfeeding/ pregnant/ planning to become pregnant cannot participate in the study. Pregnancy testing will be performed, and the use of effective contraception methods will be required for people of child-bearing potential. Should patients become pregnant during the trial, they will be withdrawn from the trial or monitored (according to whether the treatment has yet been administered).
Administered drugs: A small chance of reaction or allergy to antibiotics and other drugs used.
Endoanal ultrasound, anorectal manometry, sigmoidoscopy: Small chance of soreness or minor bleeding afterwards and a negligible (<1/10,000) possibility of perforation or damage of the intestinal wall.
Blood testing: Risk of bleeding, discomfort, light-headedness, pain, bruising, and rarely, an infection at the puncture site.

Where is the study run from?
University College London (UK)

When is the study starting and how long is it expected to run for?
September 2025 to August 2027

Who is funding the study?
Horizon 2020

Who is the main contact?
Dr Rumana Jalil-Rahman, cctu.amelie@ucl.ac.uk

Contact information

Study information

Eligibility

Locations

Countries of recruitment

• United Kingdom

Study participating centre

Results and Publications

Study outputs

Editorial Notes

03/10/2025: Internal review.
22/02/2025: ISRCTN received notification of combined HRA/MHRA approval for this trial on 22/02/2025.
12/11/2024: Study's existence confirmed by the HRA.