Studies
| Study First Submitted Date | 2021-01-21 |
| Study First Posted Date | 2021-01-28 |
| Last Update Posted Date | 2023-08-03 |
| Start Month Year | June 2024 |
| Primary Completion Month Year | August 2026 |
| Verification Month Year | August 2023 |
| Verification Date | 2023-08-31 |
| Last Update Posted Date | 2023-08-03 |
Detailed Descriptions
| Sequence: | 20724120 |
| Description | Epispadias is the mildest form of exstrophy-epispadias complex (EEC); a congenital malformation disorder involving the midline abdominal and genitourinary structures. Patients with epispadias have a defect in the urethral sphincter resulting in urinary incontinence. This trial investigates the injection of autologous primary human muscle stem cells into the urethral sphincter with the aim of repairing the defect and restore the anatomic ability for continence. Eligible participants will undergo muscle biopsy during a routine cystoscopy. Acquired muscle tissue is used for the isolation and expansion of muscle stem cells ex-vivo. Muscle stem cells are injected into the urethral sphincter under visual control using cystoscopy. Participants are assessed for safety and efficacy for a minimum of 12 months post-intervention. |
Facilities
| Sequence: | 200133391 | Sequence: | 200133392 |
| Name | Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital | Name | Pediatric Urology, Department for Urology University of Ulm |
| City | Regensburg | City | Ulm |
| Zip | 93049 | Zip | 89075 |
| Country | Germany | Country | Germany |
Facility Contacts
| Sequence: | 28111158 | Sequence: | 28111159 |
| Facility Id | 200133391 | Facility Id | 200133392 |
| Contact Type | primary | Contact Type | primary |
| Name | Wolfgang H Rösch, Prof. Dr. | Name | Anne K Ebert, Prof. Dr. |
| wolfgang.roesch@barmherzige-regensburg.de | Anne-Karoline.Ebert@uniklinik-ulm.de | ||
| Phone | +49 (0) 941 369 5453 | Phone | +49 (0) 731 500 58000 |
Facility Investigators
| Sequence: | 18334946 | Sequence: | 18334947 |
| Facility Id | 200133391 | Facility Id | 200133392 |
| Role | Sub-Investigator | Role | Principal Investigator |
| Name | Wolfgang H Rösch, Prof. Dr. | Name | Anne K Ebert, Prof. Dr. |
Conditions
| Sequence: | 52177774 | Sequence: | 52177775 |
| Name | Urinary Incontinence | Name | Epispadias, Male |
| Downcase Name | urinary incontinence | Downcase Name | epispadias, male |
Id Information
| Sequence: | 40163270 |
| Id Source | org_study_id |
| Id Value | MUST |
Countries
| Sequence: | 42574352 |
| Name | Germany |
| Removed | False |
Design Groups
| Sequence: | 55600479 | Sequence: | 55600480 |
| Group Type | Experimental | Group Type | Placebo Comparator |
| Title | Verum Group | Title | Placebo group |
| Description | Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy. | Description | Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy. |
Interventions
| Sequence: | 52491865 | Sequence: | 52491866 |
| Intervention Type | Biological | Intervention Type | Other |
| Name | Primary human muscle stem cells (Satori-01) | Name | Placebo |
| Description | Primary human muscle stem cells are isolated from patient's muscle tissue and expanded ex-vivo. They are injected into the urethral sphincter region as a one-time autologous treatment. | Description | Placebo is the injection solution without muscle stem cells. |
Keywords
| Sequence: | 79876999 | Sequence: | 79877000 | Sequence: | 79877001 | Sequence: | 79877002 |
| Name | Isolated epispadias | Name | Urinary incontinence | Name | Muscle stem cell therapy | Name | Autologous cell therapy |
| Downcase Name | isolated epispadias | Downcase Name | urinary incontinence | Downcase Name | muscle stem cell therapy | Downcase Name | autologous cell therapy |
Design Outcomes
| Sequence: | 177405322 | Sequence: | 177405323 |
| Outcome Type | primary | Outcome Type | primary |
| Measure | Prevalence of Intervention-related Adverse Events | Measure | Change in Leak Point Pressure (LPP) |
| Time Frame | Upto 12 months post-intervention | Time Frame | Six months post-intervention |
| Description | Characterization of type, incidence, severity, and duration of adverse events | Description | Change in LPP is calculated from baseline measurement |
Browse Conditions
| Sequence: | 193511392 | Sequence: | 193511379 | Sequence: | 193511380 | Sequence: | 193511381 | Sequence: | 193511382 | Sequence: | 193511383 | Sequence: | 193511384 | Sequence: | 193511385 | Sequence: | 193511386 | Sequence: | 193511387 | Sequence: | 193511388 | Sequence: | 193511389 | Sequence: | 193511390 | Sequence: | 193511391 | Sequence: | 193511393 | Sequence: | 193511394 | Sequence: | 193511395 |
| Mesh Term | Mental Disorders | Mesh Term | Urinary Incontinence | Mesh Term | Enuresis | Mesh Term | Epispadias | Mesh Term | Urination Disorders | Mesh Term | Urologic Diseases | Mesh Term | Female Urogenital Diseases | Mesh Term | Female Urogenital Diseases and Pregnancy Complications | Mesh Term | Urogenital Diseases | Mesh Term | Male Urogenital Diseases | Mesh Term | Lower Urinary Tract Symptoms | Mesh Term | Urological Manifestations | Mesh Term | Behavioral Symptoms | Mesh Term | Elimination Disorders | Mesh Term | Urogenital Abnormalities | Mesh Term | Urethral Diseases | Mesh Term | Congenital Abnormalities |
| Downcase Mesh Term | mental disorders | Downcase Mesh Term | urinary incontinence | Downcase Mesh Term | enuresis | Downcase Mesh Term | epispadias | Downcase Mesh Term | urination disorders | Downcase Mesh Term | urologic diseases | Downcase Mesh Term | female urogenital diseases | Downcase Mesh Term | female urogenital diseases and pregnancy complications | Downcase Mesh Term | urogenital diseases | Downcase Mesh Term | male urogenital diseases | Downcase Mesh Term | lower urinary tract symptoms | Downcase Mesh Term | urological manifestations | Downcase Mesh Term | behavioral symptoms | Downcase Mesh Term | elimination disorders | Downcase Mesh Term | urogenital abnormalities | Downcase Mesh Term | urethral diseases | Downcase Mesh Term | congenital abnormalities |
| Mesh Type | mesh-ancestor | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48325036 | Sequence: | 48325037 |
| Agency Class | OTHER | Agency Class | OTHER_GOV |
| Lead Or Collaborator | lead | Lead Or Collaborator | collaborator |
| Name | Simone Spuler, MD | Name | German Federal Ministry of Education and Research |
Overall Officials
| Sequence: | 29288535 |
| Role | Principal Investigator |
| Name | Anne K Ebert, Prof. Dr. |
| Affiliation | Pediatric Urology, Department for Urology University of Ulm, Germany |
Central Contacts
| Sequence: | 12010752 |
| Contact Type | primary |
| Name | Simone Spuler, Prof. Dr. |
| Phone | +49 (0) 30 450 540 501 |
| simone.spuler@charite.de | |
| Role | Contact |
Design Group Interventions
| Sequence: | 68158127 | Sequence: | 68158128 |
| Design Group Id | 55600479 | Design Group Id | 55600480 |
| Intervention Id | 52491865 | Intervention Id | 52491866 |
Eligibilities
| Sequence: | 30769031 |
| Gender | Male |
| Minimum Age | 3 Years |
| Maximum Age | 17 Years |
| Healthy Volunteers | No |
| Criteria | Inclusion Criteria: Male Isolated epispadias Aged ≥ 3 years Urinary incontinence as defined according to the International Children´s Continence Society Informed consent Exclusion Criteria: Acute or chronic inflammatory local or systemic disease Coagulation Disorder Previous adverse reaction to anesthesia Congenital heart defect, cardiac arrhythmia |
| Adult | False |
| Child | True |
| Older Adult | False |
Calculated Values
| Sequence: | 253933000 |
| Number Of Facilities | 2 |
| Registered In Calendar Year | 2021 |
| Were Results Reported | False |
| Has Us Facility | False |
| Has Single Facility | False |
| Minimum Age Num | 3 |
| Maximum Age Num | 17 |
| Minimum Age Unit | Years |
| Maximum Age Unit | Years |
| Number Of Primary Outcomes To Measure | 2 |
Designs
| Sequence: | 30515190 |
| Allocation | Randomized |
| Intervention Model | Parallel Assignment |
| Observational Model | |
| Primary Purpose | Treatment |
| Time Perspective | |
| Masking | Quadruple |
| Intervention Model Description | This is a randomized placebo-controlled clinical trial |
| Subject Masked | True |
| Caregiver Masked | True |
| Investigator Masked | True |
| Outcomes Assessor Masked | True |
Responsible Parties
| Sequence: | 28881494 |
| Responsible Party Type | Sponsor-Investigator |
| Name | Simone Spuler, MD |
| Title | Principal investigator |
| Affiliation | Charite University, Berlin, Germany |