Urinary IncontinencePhase 1/2MUSTNCT04729582
Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients
A Phase 1/2 study testing Primary human muscle stem cells (Satori-01) in people with Urinary Incontinence and Epispadias, Male, run by Simone Spuler, MD at 2 sites in Germany.
Trial summary
The aim of this trial is to evaluate the safety and efficacy of an autologous muscle stem cell therapy in the treatment of congenital urinary incontinence in isolated epispadias. Read more about this study
- Who is it for?
- Ages 3 to 17 years, men only
- Conditions the study is for
- Urinary Incontinence, Epispadias, Male
- What is being tested?
- Primary human muscle stem cells (Satori-01)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 2 sitesGermany
- Study dates
- Jun 2024to Oct 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male
- Isolated epispadias
- Aged ≥ 3 years
- Urinary incontinence as defined according to the International Children´s Continence Society
- Informed consent
Who cannot join
- Acute or chronic inflammatory local or systemic disease
- Coagulation Disorder
- Previous adverse reaction to anesthesia
- Congenital heart defect, cardiac arrhythmia
Trial locations and contacts
2 sites in 1 country.
Germany2 sites
Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
Regensburg, 93049
Wolfgang H Rösch, Prof. Dr., +49 (0) 941 369 5453, wolfgang.roesch@barmherzige-regensburg.de
Wolfgang H Rösch, Prof. Dr.
Pediatric Urology, Department for Urology University of Ulm
Ulm, 89075
Anne K Ebert, Prof. Dr., +49 (0) 731 500 58000, Anne-Karoline.Ebert@uniklinik-ulm.de
Anne K Ebert, Prof. Dr.
About this study
Official title: Combined Phase 1 and 1/2a Clinical Trial Evaluating the Safety and Efficacy of an Autologous Muscle Stem Cell Therapy in the Treatment of Urinary Incontinence in Isolated Epispadias
The aim of this trial is to evaluate the safety and efficacy of an autologous muscle stem cell therapy in the treatment of congenital urinary incontinence in isolated epispadias.
Read the detailed 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.
- Conditions
- Urinary Incontinence, Epispadias, Male
- Being tested
- Primary human muscle stem cells (Satori-01), Placebo
- Keywords
- Isolated epispadias, Urinary incontinence, Muscle stem cell therapy, Autologous cell therapy
- Medical subject headings
- Urinary Incontinence, Epispadias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Primary human muscle stem cells (Satori-01)Biological
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.
PlaceboOther
Placebo is the injection solution without muscle stem cells.
| Study group | Role | What they receive |
|---|---|---|
| Verum Group | Experimental | Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy. Receives: Biological: Primary human muscle stem cells (Satori-01) |
| Placebo group | Placebo comparator | Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of Intervention-related Adverse Events
Characterization of type, incidence, severity, and duration of adverse events
Measured: Upto 12 months post-intervention
Change in Leak Point Pressure (LPP)
Change in LPP is calculated from baseline measurement
Measured: Six months post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2021
- Study startJun 2024 (estimated)
- Primary completionAug 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urinary IncontinenceStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a randomized placebo-controlled clinical trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Simone Spuler, MD
- Sponsor type
- University, hospital or other
- Collaborators
- German Federal Ministry of Education and Research
- Responsible party
- Sponsor-investigator, Simone Spuler, MD, Charite University, Berlin, Germany
- Investigators
- Anne K Ebert, Prof. Dr., Principal investigator, Pediatric Urology, Department for Urology University of Ulm, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04729582
- Sponsor's protocol ID
- MUST
- Acronym
- MUST
- Status last verified
- Aug 2023
- First submitted
- 21 Jan 2021
- Last updated
- 3 Aug 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Combined Phase 1 and 1/2a Clinical Trial Evaluating the Safety and Efficacy of an Autologous Muscle Stem Cell Therapy in the Treatment of Urinary Incontinence in Isolated Epispadias
- ClinicalTrials.gov ID
- NCT04729582
- Acronym / sponsor ID
- MUST / MUST
- Conditions
- Urinary Incontinence, Epispadias, Male
- Keywords
- Isolated epispadias, Urinary incontinence, Muscle stem cell therapy, Autologous cell therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Simone Spuler, MDUniversity, hospital or other
- Collaborators
- German Federal Ministry of Education and Research
- Study start
- Jun 2024 (estimated)
- Primary completion
- Aug 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 28 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this trial is to evaluate the safety and efficacy of an autologous muscle stem cell therapy in the treatment of congenital urinary incontinence in isolated epispadias.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This is a randomized placebo-controlled clinical trial
| Arm | Type | Receives |
|---|---|---|
| Verum Group | Experimental | Biological: Primary human muscle stem cells (Satori-01)Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy. |
| Placebo group | Placebo comparator | Other: PlaceboParticipants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy. |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Male
- Isolated epispadias
- Aged ≥ 3 years
- Urinary incontinence as defined according to the International Children´s Continence Society
- Informed consent
Key exclusion criteria (4 listed)
- Acute or chronic inflammatory local or systemic disease
- Coagulation Disorder
- Previous adverse reaction to anesthesia
- Congenital heart defect, cardiac arrhythmia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Primary human muscle stem cells (Satori-01)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. | Biological | Verum Group |
| PlaceboPlacebo is the injection solution without muscle stem cells. | Other | Placebo group |
Primary outcome
Prevalence of Intervention-related Adverse Events
Time frame: Upto 12 months post-intervention
Characterization of type, incidence, severity, and duration of adverse events
Change in Leak Point Pressure (LPP)
Time frame: Six months post-intervention
Change in LPP is calculated from baseline measurement
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anne K Ebert, Prof. Dr. principal investigator
Pediatric Urology, Department for Urology University of Ulm, Germany
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Primary human muscle stem cells (Satori-01) in people with Urinary Incontinence and Epispadias, Male, run by Simone Spuler, MD at 2 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Simone Spuler, Prof. Dr. (+49 (0) 30 450 540 501, simone.spuler@charite.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevalence of Intervention-related Adverse Events (time frame: Upto 12 months post-intervention). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2023.