Early termination of a trial of mycophenolate mofetil for treatment of interstitial cystitis/painful bladder syndrome: lessons learned.
OBJECTIVE
We evaluated the efficacy and tolerability of mycophenolate mofetil in patients with treatment refractory interstitial cystitis/painful bladder syndrome.


METHODS
A total of 210 patients with interstitial cystitis/painful bladder syndrome were to be randomized into a multicenter, placebo controlled trial using a 2:1 randomization. Participants in whom at least 3 interstitial cystitis/painful bladder syndrome specific treatments had failed and who had at least moderately severe symptoms were enrolled in a 12-week treatment study. The primary study end point was the global response assessment. Secondary end points were general and disease specific symptom questionnaires, and voiding diaries.


RESULTS
Only 58 subjects were randomized before a black box warning regarding mycophenolate mofetil safety was issued by the manufacturer in October 2007. The trial was halted, and interim analysis was performed and presented to an independent data and safety monitoring board. Six of the 39 subjects (15%) randomized at study cessation were considered responders for mycophenolate mofetil compared to 3 of 19 controls (16%, p=0.67). Secondary outcome measures reflected more improvement in controls.


CONCLUSIONS
In a randomized, placebo controlled trial that was prematurely halted mycophenolate mofetil showed efficacy similar to that of placebo to treat symptoms of refractory interstitial cystitis/painful bladder syndrome. The results of this limited study cannot be used to confirm or refute the hypothesis that immunosuppressive therapy may be beneficial to at least a subgroup of patients with interstitial cystitis/painful bladder syndrome. Despite study termination lessons can be gleaned to inform future investigations.
Department of Urology, University of Washington, Seattle, Washington 98195-0510, USA.
Claire C
CC
Yang
David A
DA
Burks
Kathleen J
KJ
Propert
Robert D
RD
Mayer
Kenneth M
KM
Peters
J Curtis
JC
Nickel
Christopher K
CK
Payne
Mary P
MP
FitzGerald
Philip M
PM
Hanno
Toby C
TC
Chai
Karl J
KJ
Kreder
Emily S
ES
Lukacz
Harris E
HE
Foster
Liyi
L
Cen
J Richard
JR
Landis
John W
JW
Kusek
Leroy M
LM
Nyberg
D018856:Cystitis, Interstitial / Q000188:drug therapy*; D004311:Double-Blind Method; D057239:Early Termination of Clinical Trials; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D013997:Time Factors
D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid
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