- ICH GCP
- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT05054790
Cistoplastica di aumento con un costrutto neovescicale autologo
Uno studio di fase 2 sulla cistoplastica additiva utilizzando un costrutto neo-vescicale autologo in soggetti con vescica contratta fibrotica
The purpose of this research study is to find out if autologous "neo-bladder" construct for the treatment of fibrotic and/or contracted bladder can improve bladder compliance and be safe long term. The neo-bladder is like a reservoir or pouch that will be surgically attached to the bladder to assist with urine collection.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
This is a prospective, open-label, single-group clinical study. All subjects will have non-neurogenic, fibrotic contracted bladder that is refractory to medical treatment and require augmentation cystoplasty for preventing long-term sequelae (i.e., kidney failure) that result from persistently high intravesical pressure. Subjects will undergo an open full thickness bladder biopsy from which autologous urinary bladder smooth muscle and urothelial cells will be procured and expanded ex vivo. After approximately 5 - 7 weeks, expanded cells will be seeded on a biodegradable scaffold to produce the neo-bladder construct that will be surgically implanted onto an opened native bladder during an augmentation cystoplasty. Subjects will be followed for 36 months.
Tipo di studio
Iscrizione (Stimato)
Fase
- Fase 2
Contatti e Sedi
Contatto studio
- Nome: Mary-Clare Day, RN, BSN
- Numero di telefono: 336-713-1343
- Email: mday@wakehealth.edu
Luoghi di studio
-
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North Carolina
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Winston-Salem, North Carolina, Stati Uniti, 27157
- Atrium Health Wake Forest Baptist
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Contatto:
- Mary-Clare Day, RN, BSN
- Numero di telefono: 336-713-1343
- Email: mday@wakehealth.edu
-
Investigatore principale:
- Robert J Evans, MD
-
-
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
Inclusion Criteria:
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Subjects with small contracted and/or fibrotic non-neurogenic bladder.
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Small bladder <150cc capacity
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Willing and able to give signed informed consent in English.
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Ability of subject to be successfully trained in clean intermittent catheterization and bladder cycling
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Failure to respond to maximum approve dose of medical therapy (e.g. anticholinergics) or failure to tolerate /contraindication to such agents
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Medical need for bladder augmentation, as defined by the presence of:
- Decreased and inadequate bladder compliance with a bladder pressure ≥40 cmH20. OR
- New-onset of upper urinary tract changes (hydronephrosis, vesicoureteral reflux) in the last 12 months
Exclusion:
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Neurogenic bladder
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Recent (< 12 months) urological or intraperitoneal surgery or device implantation; recent (<6 month neurologic surgery-brain or spine)
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Any prior bladder augmentation procedure
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Known or suspected limitation to obtaining omentum for implantation procedure (e.g., extensive intraperitoneal adhesions)
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Any contraindication to general anesthesia
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Any contraindication or known anaphylactic or severe systemic reaction to either human blood products or materials of bovine origin
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Human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV), active hepatitis A (HAV) or other known active infection
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Subjects with active tuberculosis (TB) requiring treatment in the past 3 years. Subjects with a current positive (≥5 mm induration for high-risk subjects; otherwise ≥10 mm of induration) purified protein derivative (PPD) test are excluded unless they have completed a full course of treatment for latent TB and have a negative chest x-ray film at enrollment.
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Subjects known to be colonized with either methicillin-resistant staphylococcus aureus (MRSA) or vancomycin-resistant Enterococcus (VRE), or gentamicin-resistant organisms.
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Receipt of blood or blood products for transfusion during the 3 months prior to biopsy
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Immunocompromised subjects or subjects receiving immunosuppressive agents (inhaled corticosteroids and chronic low-dose corticosteroids [≤0.25 mg/kg prednisone or equivalent per day] are permitted).
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Brief pulsed corticosteroids for intermittent symptoms (e.g., asthma) are permitted.
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Known history of hypersensitivity to aminoglycosides or fluoroquinolones.
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History of true allergy to iodine or iodinated x-ray contrast agents
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Use of any investigational product within 3 months
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Prior participation in the study
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Any history of alcohol and/or any drug abuse
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Current incarceration for any reason
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Unwillingness, inability, or unlikely compliance with study related procedures
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Any circumstance in which the investigator deems participation in the study is not in the subject's best interest
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Subjects with an Alanine Aminotransferase (ALT) or aspartate aminotransferase (AST) value >3 times the upper limit of normal
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Subjects with an albumin value <3.0 g/dL
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Subjects with a history of an anaphylactic or a severe systemic reaction to the biodegradable polymers glycolic acid (PGA) and lactic-co-glycolic acid (PLGA)
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Subjects with acute or chronic abdominal skin infections and/or acute or chronic inflammatory bowel disease
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Subjects with uncontrolled diabetes, unstable cardiac and/or pulmonary disorders, or bleeding disorders
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Subjects who have received Botulinum Toxin A injections into the bladder within the previous 12 months.
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Female subjects who are pregnant, planning to get pregnant or lactating/breast-feeding.
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Female subjects of child-bearing potential unwilling to practice an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy) for duration of the study.
- If a patient has had a hysterectomy, a bilateral oophorectomy, documented ovarian failure or is older than age 51.5 years and has not had a menstrual period in more than 12 months, they will be considered to be non-childbearing. This will be ascertained from patient interview/self-report, medical records (if necessary) and the investigator's judgment. "Spontaneous menopause," occurs in the United States at a mean age of 51.5 years.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: Autologous "Neo-Bladder" Construct
All subjects enrolled will have non-neurogenic, fibrotic contracted bladder that is refractory to medical treatment and require augmentation cystoplasty for preventing long-term sequelae (i.e., kidney failure) that result from persistently high intravesical pressure. |
Subjects will undergo an open full thickness bladder biopsy from which autologous urinary bladder smooth muscle and urothelial cells will be procured and expanded ex vivo. After approximately 5 - 7 weeks, expanded cells will be seeded on a biodegradable scaffold to produce the neo-bladder construct that will be surgically implanted onto an opened native bladder during an augmentation cystoplasty. |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Bladder Compliance Average
Lasso di tempo: Month 12
|
Average bladder capacity as found on Urodynamics results |
Month 12
|
|
Average Number of 24 Hour Voids
Lasso di tempo: Month 6
|
Per voiding diary |
Month 6
|
|
Average Number of 24 Hour Voids
Lasso di tempo: Month 9
|
Per voiding diary |
Month 9
|
|
Average Number of 24 Hour Voids
Lasso di tempo: Month 12
|
Per voiding diary |
Month 12
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Change in Bladder Capacity
Lasso di tempo: From Baseline through Month 12
|
Shown through urodynamics study |
From Baseline through Month 12
|
|
End filling pressure
Lasso di tempo: Baseline, Month 6, Month 9 and Month 12
|
At age-related nomogram-derived bladder capacity |
Baseline, Month 6, Month 9 and Month 12
|
|
Urogenital Distress Inventory-6 Question (UDI-6)
Lasso di tempo: Month 6, Month 9 and Month 12
|
Evaluation of urogenital function from self-reported questionnaire. Score ranges from 0-18 with higher score denoting worsening in urogenital function. |
Month 6, Month 9 and Month 12
|
|
Urogenital Distress Inventory-7 Question (UDI-7)
Lasso di tempo: Month 6, Month 9 and Month 12
|
Evaluation of urogenital function from self-reported questionnaire. Score ranges from 0-21 with higher score denoting worsening in urogenital function. |
Month 6, Month 9 and Month 12
|
|
Genitourinary Pain Index (GUPI)
Lasso di tempo: Month 6, Month 9 and Month 12
|
Evaluation of pain from self-reported questionnaire. Score ranges from 0-45 with a higher score denoting worse symptoms. |
Month 6, Month 9 and Month 12
|
|
Quality of Life (QOL) Questionnaire
Lasso di tempo: Month 6, Month 9 and Month 12
|
Self-reported quality of life evaluation. Score ranges from 0-6 with highest score denoting the worst feelings about quality of life. |
Month 6, Month 9 and Month 12
|
|
American Urological Association Symptom Score (AUASS)
Lasso di tempo: Month 6, Month 9 and Month 12
|
Self-reported urological symptom assesment. Score ranges from 1-35 with score of 1-7 denoting mild symptoms, 8-19 denoting moderate symptoms and 20-35 denoting severe symptoms. |
Month 6, Month 9 and Month 12
|
Collaboratori e investigatori
Investigatori
- Investigatore principale: Robert J. Evans, MD, Atrium Health Wake Forest Baptist
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Inizio studio (Stimato)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Altri numeri di identificazione dello studio
- IRB00076977
Piano per i dati dei singoli partecipanti (IPD)
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Informazioni su farmaci e dispositivi, documenti di studio
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