Iltamiocel for Bowel Incontinence

(DigniFI Trial)

Not currently recruiting at 28 trial locations

Overseen By Melissa DeBoer

Age: 18+

Sex: Female

Trial Phase: Phase 3

Sponsor: Cook MyoSite

Disqualifiers: Pregnancy, Cancer, Inflammatory bowel disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new injection called iltamiocel to determine its effectiveness in reducing bowel accidents in women who have experienced this issue for at least a year due to childbirth injuries. Half of the participants will receive the iltamiocel injection, while the other half will receive a placebo (a shot with no active medicine). This trial targets adult women who have tried treatments like diet changes or exercises but still experience frequent bowel accidents. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, since participants must have failed conservative treatments like antidiarrheal medications for at least 6 months, it seems you may continue with those treatments.

Is there any evidence suggesting that iltamiocel is likely to be safe for humans?

Research has shown that iltamiocel, a treatment made from muscle cells, is safe. In earlier studies, people who received iltamiocel experienced symptom improvements without major safety concerns. The treatment involves injecting these cells to aid bowel control. Studies have found that patients tolerate this treatment well, with no significant reports of serious side effects. This suggests that iltamiocel is a promising option for those dealing with bowel incontinence, especially women with childbirth-related injuries. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for bowel incontinence, which often include medications and lifestyle changes, Iltamiocel offers a novel approach by using advanced cell therapy. Researchers are excited because Iltamiocel involves the use of regenerative cells that target the underlying causes of incontinence, potentially restoring normal function rather than just managing symptoms. This innovative mechanism could lead to more effective and lasting relief for patients, setting it apart from conventional options.

What evidence suggests that iltamiocel might be an effective treatment for bowel incontinence?

Research has shown that iltamiocel, a type of cell therapy, could help treat fecal incontinence, especially in women with childbirth-related injuries. In this trial, participants may receive iltamiocel, which studies have found to greatly reduce incontinence episodes, improving patients' quality of life. The treatment uses muscle cells to strengthen weak tissues in the anal area. Early results suggest it is safe and effective when other treatments have failed. These findings highlight iltamiocel as a promising option for those with ongoing fecal incontinence. ^{1 2 3 4 6}

Who Is on the Research Team?

Ron Jankowski, PhD

Principal Investigator

Cook Myosite, Inc.

Are You a Good Fit for This Trial?

This trial is for adult women over 18 with chronic fecal incontinence due to childbirth injuries. They must have tried other treatments like diet changes or pelvic exercises for at least 6 months without success and score ≥9 on a specific incontinence scale.

Inclusion Criteria

I have tried treatments like diet changes and medications for my condition for over 6 months without success.

You have had a serious tear during childbirth.

You have a Cleveland Clinic Florida Incontinence Score (CCFIS) of 9 or higher at the beginning of the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single injection of iltamiocel or placebo

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants who received placebo may opt to receive an injection with their cells

Not specified

What Are the Treatments Tested in This Trial?

Interventions

• Iltamiocel
• Placebo

Trial Overview

The study tests the effectiveness of a single injection of Iltamiocel (a cell therapy) versus a placebo in reducing episodes of bowel leakage. Participants are randomly divided into two groups, one receiving Iltamiocel and the other a placebo.

How Is the Trial Designed?

Group I: Iltamiocel Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Cook MyoSite

Lead Sponsor

Trials

14

Recruited

1,100+

Published Research Related to This Trial

In a phase I clinical trial involving 10 patients with intrinsic sphincter deficiency (ISD), periurethral implantation of myofibres containing muscle precursor cells (MPCs) significantly increased intraurethral pressure and improved continence, with four out of five women experiencing notable improvements over 12 months.

The procedure was found to be safe, with no serious side effects reported, indicating that myofibre implantation could be a promising one-step treatment for urinary incontinence due to ISD, although further research is needed to enhance its effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Periurethral skeletal myofibre implantation in patients with urinary incontinence and intrinsic sphincter deficiency: a phase I clinical trial. Yiou, R., Hogrel, JY., Loche, CM., et al.[2015]

A multicenter, placebo-controlled study showed that injecting high doses of autologous skeletal muscle-derived cells significantly improved fecal incontinence (FI) symptoms, particularly in patients with a shorter duration of FI and higher baseline incontinence episode frequency.

The treatment was safe, with no unexpected adverse events reported, and the benefits were sustained or increased over 12 months in the high cell count group, suggesting potential for this therapy in managing FI.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Skeletal Muscle-Derived Cell Implantation for the Treatment of Fecal Incontinence: A Randomized, Placebo-Controlled Study. Frudinger, A., Gauruder-Burmester, A., Graf, W., et al.[2023]

Iltamiocel, a cellular therapy using autologous muscle-derived cells, was found to be safe with no serious adverse events reported in a study of 48 adults with fecal incontinence.

At 12 months post-treatment, participants experienced a significant reduction in fecal incontinence episodes and improved quality of life, with over half of the participants showing a ≥50% reduction in episodes and nearly a quarter achieving complete continence.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Iltamiocel Cellular Therapy for the Treatment of Fecal Incontinence. Results of a Phase 1/2 Study. Knowles, CH., Canestrari, E., Jankowski, RJ., et al.[2023]

The study found that autologous muscle-derived cells for urinary sphincter repair (AMDC-USR) were safe and well-tolerated, with no serious adverse events reported among female subjects with stress urinary incontinence.

Although the primary efficacy results were inconclusive due to a high placebo response rate (90%), post hoc analyses indicated that stricter endpoints might reveal a potential treatment effect, suggesting the need for refined trial designs in future studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A double-blind, randomized, placebo-controlled clinical trial evaluating the safety and efficacy of autologous muscle derived cells in female subjects with stress urinary incontinence. Jankowski, RJ., Tu, LM., Carlson, C., et al.[2021]

In a clinical trial involving 39 patients (average age 60.6 years), ultrasound-guided injection of autologous skeletal muscle-derived cells (SMDC) into the external anal sphincter significantly reduced weekly incontinence episodes by at least 50% in 80.6% of males and 78.4% of females after 12 months.

The procedure was well tolerated with no serious adverse events reported, suggesting it is a safe and effective treatment for fecal incontinence due to external anal sphincter damage or atrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Skeletal muscle-derived cell implantation for the treatment of sphincter-related faecal incontinence. Frudinger, A., Marksteiner, R., Pfeifer, J., et al.[2019]

In a study of 58 participants, both autologous muscle-derived cell (AMDC) injections and placebo injections led to significant increases in urethral sphincter volumes and reductions in stress incontinence episode frequency (IEF) after 12 months, but there were no significant differences between the two groups.

Factors such as longer urethral length and larger external sphincter volume at baseline were associated with better responses to AMDC injections, suggesting these may be important predictors of treatment efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of the effects of autologous muscle-derived cell injections on urethral sphincter morphometry using 3D/4D ultrasound. Ismail, S., Morin, M., Tu, LM.[2021]

The first clinical trial using autologous muscle-derived stem cells (AMDC) for treating underactive bladder (UAB) showed no adverse events, indicating that the treatment is safe.

After one year, while the patient still required self-catheterization, there was a notable reduction in maximum bladder capacity, suggesting that AMDC injections may improve bladder function in UAB patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intradetrusor injection of adult muscle-derived cells for the treatment of underactive bladder: pilot study. Levanovich, PE., Diokno, A., Hasenau, DL., et al.[2018]

Skeletal muscle-derived mononuclear cells (SMDMCs) were successfully incorporated into the urethra of female rats, promoting recovery of the damaged urethral sphincter after stress urinary incontinence surgery.

The SMDMC treatment led to a significant reduction in connective tissue deposition and an increase in muscle content compared to the saline control group, indicating potential efficacy for this novel cell-based therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Periurethral muscle-derived mononuclear cell injection improves urethral sphincter restoration in rats. Turco, MP., de Souza, AB., de Campos Sousa, I., et al.[2018]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/37144409/

Safety and Efficacy of Iltamiocel Cellular Therapy for the ...

The administration of iltamiocel cellular therapy is safe. Iltamiocel shows promise for significantly improving fecal incontinence symptoms and quality of life.

2.

ics.org

ics.org/2022/abstract/397

an analysis of efficacy in women with obstetric injury

We examined the efficacy and safety of iltamiocel, a cellular therapy investigational product comprised of autologous muscle derived cells (AMDC), as a ...

3.

advances.massgeneral.org

advances.massgeneral.org/digestive-health/article.aspx?id=1599

Evaluating Cell Therapy for Chronic Fecal Incontinence ...

A clinical trial across 25 sites worldwide is assessing the effectiveness of cell therapy to reinforce weakened tissue associated with fecal ...

4.

cookmyosite.com

cookmyosite.com/clinical-research

Clinical Research | Leaders in Regenerative Medicine

This study evaluates the efficacy and safety of a single injection of iltamiocel compared to a placebo in the reduction of fecal incontinence episode frequency ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04976153

NCT04976153 | Skeletal Muscle-derived Cell Implantation ...

Patient has symptoms of urge fecal incontinence with a disease duration of at least 6 months and did not improve sufficiently by conservative treatment ...

6.

researchgate.net

researchgate.net/publication/370552413_Safety_and_Efficacy_of_Iltamiocel_Cellular_Therapy_for_the_Treatment_of_Fecal_Incontinence_Results_of_a_Phase_12_Study

Safety and Efficacy of Iltamiocel Cellular Therapy for the ...

Autologous skeletal muscle-derived cells to treat obstetric anal incontinence resulted in sustained improvement in incontinence episodes ...