FI-3, Fidelia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Innovacell GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

20 Apr 2022 → ongoing

Decision date (initial)

2025-01-30

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-518243-38-00

EudraCT number

2021-001376-42

ClinicalTrials.gov

NCT04976153

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

The primary objective of this study is the final assessment of clinical efficacy and clinical safety of ICEF15 therapy for patients with fecal incontinence due to external anal sphincter dysfunction caused by its disruption and/or weakness.

Secondary objectives 1

1. The assessment of treatment efficacy during the observation period, the assessment of FI condition improvement, and the assessment of the improvement in the patients’ “Quality of Life”.

Conditions and MedDRA coding

Fecal incontinence in female and male patients

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

Swedish Medical Products Agency, European Medicines Agency, European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Part 1 General: Patients of ≥18 years of age
2. Patients willing and able to comply with the study procedures
3. Patients who are mentally competent and able to understand all study requirements
4. Patients must agree to read and sign the Informed Consent (IC) form prior to any studyrelated procedures
5. Female patients of childbearing potential willing to use appropriate methods of contraception (see Section 8.4.6.3). Women considered of childbearing potential shall only be included in the study after a confirmed menstrual period and a negative pregnancy test.
6. Part 2 Related to fecal incontinence: At Screening (V1), the patient has symptoms of urge fecal incontinence with a disease duration of at least 6 months but equal to or less than 10 years, and did not improve sufficiently by conservative treatment performed for at least 3 months
7. Urge fecal incontinence episodes exceeding ”traces” (as defined by the patient’s diary) that occur more than twice a week
8. Maximal incremental voluntary squeeze pressure (increase to resting pressure) on anal manometry of 100 mmHg or less in women and 150 mmHg or less in men
9. Ultrasound of the anal canal showing intact external anal sphincter or a maximal overall extent of external anal sphincter injury and tear of 180 degrees

Exclusion criteria 29

1. Part 1 General: Patients who, according to the clinical judgment of the investigator, are not suitable for this study
2. Patients deprived of their liberty by a judicial or administrative decision, patients admitted to a hospital, social institution or who are under a measure of legal protection, patients hospitalized without consent or who are in an emergency situation
3. Patients who are currently participating or have participated in another clinical study (testing a medical device or drug) within 30 days prior to screening Visit 1 in this study or have previously participated in this study
4. Patients dependent from the sponsor, CRO, or the investigator (e.g. employees, relatives, etc.)
5. Female patients who are pregnant, lactating, or intending pregnancy in the study and female patients of childbearing potential who are not willing to use appropriate methods of contraception (see Section 8.4.6.3) up to Visit 8. or who have a positive pregnancy test (only to be performed in women of childbearing potential)
6. Part 2 Related to fecal incontinence: Patients for whom the investigator determines that FI has a different cause than external anal sphincter dysfunction. This evaluation is based on the current medical history, surgical history physical examination, anoscopy / rectoscopy, anorectal manometry, and anal canal ultrasonography at the screening visit (V1) As specific exclusion: • Patients with chronic diarrhea or liquid stool that my cause fecal incontinence • Patients with rectal fecal impaction that may be the cause of overflow fecal incontinence • Patients who may have passive fecal incontinence due to rectal hyposensitivity and lack of urge to defecate • Patients in whom the cause of fecal incontinence is considered to be or cannot be excluded to be neuropathy or myelopathy • Patients with Goligher class III internal hemorrhoids (patients with Goligher class I and class II internal hemorrhoids are not excluded as they are considered not clinically involved in fecal incontinence)
7. Patients with global fragmentation of the external anal sphincter as assessed by anal canal ultrasound
8. Patients who underwent any anorectal surgery within 6 months before screening visit
9. Patients who underwent a total of two or more external anal sphincter-related surgeries (only one repair after an obstetric injury after vaginal birth is allowed)
10. Patients who currently have anal fistulas or fissures or have recurrent anal fistulas or fissures
11. Patients with poorly controlled chronic constipation including obstructed defecation syndrome
12. Part 3 Biopsy/Implantation-specific: Patients with indications against a surgery under anesthesia
13. Part 4 Cell therapy-specific: Patients with a malignant disease not in remission for 5 years or more
14. Patients who have undergone radiation therapy of the bowel and pelvis
15. Patients who have undergone chemotherapy within the last 5 years prior to study enrolment and/or chemotherapy related neuropathy of the bowel and pelvis
16. Patients with compromised immune system and/or rheumatic disease, and patients under immunosuppressive therapy
17. Patients with a diagnosis of chronic inflammatory bowel disease (e.g. Crohn’s disease, Colitis Ulcerosa)
18. Patients suffering from a disease which has not been resolved within 4 weeks prior to screening including fever and/or diarrhea of unknown reasons
19. Patients with known hypersensitivity to any component of the product (autologous cells, Ringer’s lactate, human serum albumin, DMSO, bovine proteins, fibroblast growth factor (FGF), gentamicin)
20. Patients diagnosed with human immunodeficiency virus (HIV), acute or chronic Hepatitis- C-virus (HCV), acute or chronic Hepatitis-B-virus (HBV), active Syphilis or Human Tlymphotropic virus (HTLV) (HTLV tested upon risk assessment by investigator)
21. Patients diagnosed with any kind of skeletal muscle-disease and/or neuronal disorders
22. Part 5 Related to electrical stimulation: Patients with severe myocardial disorders, irregular pulse or a pacemaker
23. Patients with implantations of metal components in the electrical stimulation treatment area
24. Defects of the skin and mucous membranes (injuries, ulcerations) and acute inflammation of the mucous membranes, skin or subcutaneous tissue in the treatment area
25. Metal implants in the field of treatment
26. Bleeding in the treatment area
27. Known mental illnesses, cognitive disorders
28. Part 6 Other: Patients with uncontrolled diabetes mellitus type I or II, or suffering from diabetic peripheral neuropathic pain
29. Patients with clinically relevant abnormal laboratory values judged by the responsible investigator as relevant for the study treatment

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Changes in frequency of incontinence episodes as measured by the bowel diary records prior to Visit 8 (target day 457) compared to the baseline period from diary records prior to implantation Visit 3 (target day 92), in both treatment groups.

Secondary endpoints 1

1. Frequency of response measured in both treatment arms as a reduction of the frequency of incontinence episodes by ≥ 50% under treatment. Derived from diary records prior to Visit 8 (target day 457) compared to the baseline period from diary records prior to implantation Visit 3 (target day 92).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Innovacell GmbH

Sponsor organisation

Innovacell GmbH

Address

Mitterweg 24, Hoetting Hoetting

City

Innsbruck

Postcode

6020

Country

Austria

Scientific contact point

Organisation

Innovacell GmbH

Contact name

Melanie Amort-Achmüller

Public contact point

Organisation

Innovacell GmbH

Contact name

Melanie Amort-Achmüller

Third parties 3

Locations

10 EU/EEA countries · 27 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 281 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

17 submissions — initial application plus substantial / non-substantial modifications