Intravesical Instillation of Liposome Encapsulated Botulinum Toxin A (Lipotoxin) in Treatment of Interstitial Cystitis
Condition: Interstitial Cystitis
MeSH terms: Cystitis; Cystitis, Interstitial
Study type: Interventional
Phase: Phase 2

Summary: 
      To evaluate the efficacy and safety of intravesical instillation of Lipotoxin for the
      treatment of IC/BPS
    

Detailed description: 
      Liposome has been proven able to carry botulinum toxin protein across the cell membrane and
      effect on urothelial receptors in human overactive bladder. However, the therapeutic duration
      is limited to 1 month. Intravesical BOTOX injection in patients with interstitial cystitis
      (IC) can effectively decrease pain, improve bladder capacity and decrease frequency. However,
      the need of cystoscopic injection limits its wide application.

      A total of 100 eligible women with non-ulcer IC will be enrolled to receive intravesical
      instillation of Lipotoxin containing 80mg liposomes and 200U BOTOX (treatment group), 200U
      BOTOX in normal saline (N/S) (active control group) or normal saline (placebo control group)
      single treatment. At least 90 evaluable patients will be included for the final analysis.

      All patients should have IC symptoms for at least 6 months, and proven to have grade 2
      diffused glomerulations after cystoscopic hydrodistention (HD) within recent 1 year without
      Hunner's lesion. Patients should not have UTI in recent 12 months, no urinary tract stone.
      Patients should have been proven free of detrusor overactivity or bladder outlet obstruction.
      Patients should not receive intravesical hyaluronic acid treatment in recent 6 months, or
      intravesical Botox injection in recent 12 months. Intravesical instillation of Lipotoxin at
      OPD and the patient should hold the solution for 2 hours to allow bladder distention.
      Retreatment with Lipotoxin at 3 months if patient reports ineffective.

      Primary end-point is the change of the O'Leary-Sant symptom score (including ICSI and ICPI)
      from baseline to 1 month after treatment. Secondary endpoints include VAS, daily frequency,
      nocturia and FBC as record in 3-day voiding diary, Qmax, voided volume, PVR and global
      response assessment (GRA). Four visits are required at baseline screening (before first
      treatment), treatment (V1), 2 weeks (V2), 4 weeks (V3, primary end-point) and 12 weeks (V4).
    

Intervention name(s): Liposome encapsulated BoNT-A; BOTOX 200U in normal saline; Normal saline
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Liposome encapsulated BoNT-A ( mixed BOTOX 200U/10ml in Liposome 80mg/40ml) in single intravesical instillation; BOTOX 200U in normal saline (BoNT-A/NS) 50ml in single intravesical instillation; Normal saline (N/S) 50ml in single intravesical instillation
Other intervention name(s): Liposome; onabotulinumtoxinA 200U; onabotulinumtoxinA 200U; Saline
Intervention MeSH terms: Botulinum Toxins, Type A; abobotulinumtoxinA
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: This is a double-blind, randomized, placebo-controlled physician initiated study enrolled patients with refractory IC/BPS. Patients were assigned to intravesical instillation of lipotoxin (onabotulinumtoxinA 200 U with 80mg sphingomyelin), onabotulinumtoxinA 200 U in normal saline, or normal saline.
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: IC/PBS; VAS; Botulinum Toxin A; Lipotoxin