Interstitial CystitisPhase 3ICCRN RCT2NCT00451867
A Randomized Multicenter Double-Blind CT to Evaluate the Efficacy and Safety of Mycophenolate Mofetil . . .
A Phase 3 study testing Mycophenolate Mofetil and Mycofenolate Mofetil (MMF) in people with Interstitial Cystitis and Painful Bladder Syndrome, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 11 sites in 2 countries.
Trial summary
The purpose of this study is to investigate the safety and effectiveness of a medication called CellCept in treating refractory (has not responded to other treatments) interstitial cystitis. CellCept belongs to a class of medications called immuno-suppressants. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Interstitial Cystitis, Painful Bladder Syndrome
- What is being tested?
- Mycophenolate Mofetil and Mycofenolate Mofetil (MMF)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 210 participants
- Where
- 11 sitesin 2 countries
- Study dates
- Mar 2007to Apr 2008
- Phase
- Phase 3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant at least 18 years of age and received a diagnosis of PBS/IC, confirmed by cystoscopy and hydrodistention in the past with findings of glomerulations and/or ulceration.
- Participant has symptoms of urinary frequency and pain/discomfort (at least 4 on each 0-10 Likert scale) at entry.
- Participant failed at least 24 weeks of active treatment with a minimum of 3 standard forms of therapy (including hydrodistension) or combination of therapies for PBS/IC.
- Participant will receive cystoscopy to be performed in the office at baseline visit before randomization if none has been conducted within the previous 24 weeks. Cystoscopy results must show no unevaluated lesions.
- Female participants with a cervix are required to have Pap smear exam within the past 12 months prior to enrollment with normal results reported.
- Participant (female) with child-bearing potential must agree to use two reliable/medically approved methods of birth control.
Who cannot join
- History of cancer or known pre-malignant conditions, including skin cancer.
- History of bladder calculus, tuberculous cystitis; neurologic disease affecting bladder function.
- Current immunocompromised condition, including current or chronic treatment with immunosuppressive agents, or known positive for HIV (positive antibody confirmed by Western Blot or IFA); active tuberculosis requiring on-going therapy; current systemic steroid treatment at any dose.
- Liver function test or creatinine results greater than 2x the upper limit of normal at home institution laboratory.
- Any baseline leukopenia (an absolute neutrophil count <1,500/µL), thrombocytopenia (a platelet count less than 150,000/microL), or anemia - HGB < 12 or < 11 g/dLin men and in women respectively.
- Is seropositive for Hepatitis B surface antigen; or is seropositive for Hepatitis B surface antibody (if not previously vaccinated); is seropositive for Hepatitis C antibody or HIV antigen or antibody.
Show all 20Show fewer
- Allergy or hypersensitivity to study medication.
- Unable to void spontaneously.
- Active urethral or ureteral calculi, urethral diverticulum.
- Any severe debilitating or urgent concurrent medical condition.
- Previous cytoxan/cyclophosphamide treatment, pelvic radiation therapy; augmentation cystoplasty, cystectomy, or cystolysis; neurectomy.
- Participants with history of treatment for genital tract dysplasia or genital warts or genital herpes.
- Patients with active or a history of peptic ulcer disease, inflammatory bowel disease or gastrointestinal bleeding.
- Patients with hypertension not adequately controlled with medication.
- Patient currently taking H2 blockers or proton pump inhibitors.
- Patients who cannot tolerate or refuse an office cystoscopy.
- Currently being treated for chronic bacterial prostatitis, as documented by a positive urine culture or prior history of recurrent bacterial urinary infections.
- Unevaluated suspicious prostate exam.
- Lactation, pregnancy, or refusal of two types of (medically approved/reliable) birth control in women of child-bearing potential.
- Pain, frequency, urgency symptoms present only during menses.
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States10 sites
University of Maryland
Baltimore, Maryland, 21201
Henry Ford Hospital
Detroit, Michigan, 48202
University of Iowa
Iowa City, Iowa, 52242
Loyola University Medical Center
Maywood, Illinois, 60153
University of Pennsylvania Health System
Philadelphia, Pennsylvania, 19104
University of Rochester
Rochester, New York, 14642
William Beaumont Hospital
Royal Oak, Michigan, 48073
Univeristy of California San Diego
San Diego, California, 92093
University of Washington
Seattle, Washington, 98195
Stanford University Medical center
Stanford, California, 94305
About this study
Official title: A Randomized Multicenter Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Mycophenolate Mofetil (CellCept) for the Treatment of Refractory Interstitial Cystitis (IC)
The purpose of this study is to investigate the safety and effectiveness of a medication called CellCept in treating refractory (has not responded to other treatments) interstitial cystitis.
CellCept belongs to a class of medications called immuno-suppressants. Immuno-suppressants work in the body by reducing the immune system's ability to produce certain reactions that can cause inflammation. In some people, the inflammation produced by their immune system can damage healthy tissues and cause symptoms of pain and discomfort. CellCept is approved by the U.S. Food and Drug Administration (FDA) for use in patients who have had an organ transplant. When used in combination with other drugs, CellCept helps to prevent the rejection of the transplanted organ and is used widely in patients who have received kidney, liver and heart transplants. CellCept is also frequently used but not FDA approved for the treatment of severe rheumatoid arthritis which is a disease caused when the body's immune system acts against healthy tissues in the joints.
Due to its special activity, CellCept may be useful in treating certain inflammatory diseases or conditions like interstitial cystitis.
Read the detailed description
Interstitial Cystitis (IC) is a bladder syndrome characterized as painful, debilitating and chronic, with no universally successful treatment option currently available. Characteristic symptoms include pain with bladder filling, and marked urinary frequency (to relieve pain). The only FDA-approved oral medication for treatment of IC is pentosan polysulfate (Elmiron), recently demonstrated by our collaborative research network to perform with little more efficacy than placebo (ref), and which is expensive and has associated side effects. Current clinical treatment protocols are empiric and usually aimed at relieving pain. There is a pressing need for an effective oral medication for treatment of IC. The presentation of symptoms can be quite variable among patients, suggesting that IC is a multi-factorial syndrome with several proposed etiologies, some of which may be interrelated.
- Conditions
- Interstitial Cystitis, Painful Bladder Syndrome
- Being tested
- Mycophenolate Mofetil, Mycofenolate Mofetil (MMF), Placebo
- Keywords
- interstitial cystitis research, painful bladder syndrome research, pelvic pain research, interstitial cystitis and autoimmune, interstitial cystitis and cellcept, interstitial cystitis and mycophenolate mofetil, interstitial cystitis and MMF, interstitial cystitis and inflammation, inflammatory diseases and immunosupression
- Medical subject headings
- Cystitis, Interstitial, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called CellCept
Mycofenolate Mofetil (MMF)Drug
Also called CellCept
2000 mg per day divided into 2 equal doses.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| A | Active comparator | 2000 mg per day of CellCept (MMF) divided into 2 equal doses. Receives: Drug: Mycophenolate Mofetil; Drug: Mycofenolate Mofetil (MMF) |
| B | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To compare CellCept 2 grams daily to placebo for effects on overall IC symptoms and well being in patients with refractory PBS/IC.
Measured: 12 Weeks
To assess the safety profile of CellCept in the treatment of refractory PBS/IC.
Measured: 12 Weeks
Secondary outcomes (6)
To investigate the association between clinical subgroups, characterized by differences in baseline characteristics (such as presence of ulcers, duration of symptoms, significant co-morbid diseases, serological abnormalities), and efficacy of CellCept.
Measured: 12 Weeks
To assess the patterns of patient expectations, associations with symptom severity, and the potential impact of patient expectations on response to treatment.
Measured: 12 Weeks
To assess patterns of treatment goals and goal achievement in this study population, as well as baseline characteristics and factors related to goal selection and achievement.
Measured: 12 Weeks
To assess impact of study medication on pain medication use.
Measured: 12 Weeks
To assess the frequency and mechanism of un-blinding on study results and assess how the patient's perception of which treatment they received changes over time and influences ultimate outcome.
Measured: 12 Weeks
To assess the rate of detectable immune disorders in patients with PBS/IC refractory to standard treatment using CellCept.
Measured: 12 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2007 (estimated)
- Study startMar 2007
- Primary completionFeb 2008
- Study completionApr 2008 (estimated)
Why it stopped: The major and primary reason for the study termination is the observed reduced efficacy of CellCept compared to placebo.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Interstitial CystitisStudies of the same condition, open and under way.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 210 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Investigators
- John Kusek, PhD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)LeRoy Nyberg, MD, PhD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Richard Landis, PhD, Principal investigator, University of PennsylnaniaDavid Burks, MD, Study chair, Henry Ford HospitalHarris Foster, MD, Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00451867
- Sponsor's protocol ID
- DK765209-Cellcept (IND)
- Acronym
- ICCRN RCT2
- Status last verified
- Jan 2010
- First submitted
- 23 Mar 2007
- Last updated
- 14 Jan 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Multicenter Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Mycophenolate Mofetil (CellCept) for the Treatment of Refractory Interstitial Cystitis (IC)
- ClinicalTrials.gov ID
- NCT00451867
- Acronym / sponsor ID
- ICCRN RCT2 / DK765209-Cellcept (IND)
- Conditions
- Interstitial Cystitis, Painful Bladder Syndrome
- Keywords
- interstitial cystitis research, painful bladder syndrome research, pelvic pain research, interstitial cystitis and autoimmune, interstitial cystitis and cellcept, interstitial cystitis and mycophenolate mofetil, interstitial cystitis and MMF, interstitial cystitis and inflammation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The major and primary reason for the study termination is the observed reduced efficacy of CellCept compared to placebo.
- Enrolment
- 210 participants (actual)
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Study start
- Mar 2007
- Primary completion
- Feb 2008 (actual)
- Study completion
- Apr 2008 (estimated)
- First posted
- 26 Mar 2007 (estimated)
- 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 purpose of this study is to investigate the safety and effectiveness of a medication called CellCept in treating refractory (has not responded to other treatments) interstitial cystitis.
CellCept belongs to a class of medications called immuno-suppressants. Immuno-suppressants work in the body by reducing the immune system's ability to produce certain reactions that can cause inflammation. In some people, the inflammation produced by their immune system can damage healthy tissues and cause symptoms of pain and discomfort. CellCept is approved by the U.S. Food and Drug Administration (FDA) for use in patients who have had an organ transplant. When used in combination with other drugs, CellCept helps to prevent the rejection of the transplanted organ and is used widely in patients who have received kidney, liver and heart transplants. CellCept is also frequently used but not FDA approved for the treatment of severe rheumatoid arthritis which is a disease caused when the body's immune system acts against healthy tissues in the joints.
Due to its special activity, CellCept may be useful in treating certain inflammatory diseases or conditions like interstitial cystitis.
Detailed description
Interstitial Cystitis (IC) is a bladder syndrome characterized as painful, debilitating and chronic, with no universally successful treatment option currently available. Characteristic symptoms include pain with bladder filling, and marked urinary frequency (to relieve pain). The only FDA-approved oral medication for treatment of IC is pentosan polysulfate (Elmiron), recently demonstrated by our collaborative research network to perform with little more efficacy than placebo (ref), and which is expensive and has associated side effects. Current clinical treatment protocols are empiric and usually aimed at relieving pain. There is a pressing need for an effective oral medication for treatment of IC. The presentation of symptoms can be quite variable among patients, suggesting that IC is a multi-factorial syndrome with several proposed etiologies, some of which may be interrelated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A | Active comparator | Drug: Mycophenolate Mofetil, Drug: Mycofenolate Mofetil (MMF)2000 mg per day of CellCept (MMF) divided into 2 equal doses. |
| B | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Participant at least 18 years of age and received a diagnosis of PBS/IC, confirmed by cystoscopy and hydrodistention in the past with findings of glomerulations and/or ulceration.
- Participant has symptoms of urinary frequency and pain/discomfort (at least 4 on each 0-10 Likert scale) at entry.
- Participant failed at least 24 weeks of active treatment with a minimum of 3 standard forms of therapy (including hydrodistension) or combination of therapies for PBS/IC.
- Participant will receive cystoscopy to be performed in the office at baseline visit before randomization if none has been conducted within the previous 24 weeks. Cystoscopy results must show no unevaluated lesions.
- Female participants with a cervix are required to have Pap smear exam within the past 12 months prior to enrollment with normal results reported.
Key exclusion criteria (20 listed)
- History of cancer or known pre-malignant conditions, including skin cancer.
- History of bladder calculus, tuberculous cystitis; neurologic disease affecting bladder function.
- Current immunocompromised condition, including current or chronic treatment with immunosuppressive agents, or known positive for HIV (positive antibody confirmed by Western Blot or IFA); active tuberculosis requiring on-going therapy; current systemic steroid treatment at any dose.
- Liver function test or creatinine results greater than 2x the upper limit of normal at home institution laboratory.
- Any baseline leukopenia (an absolute neutrophil count <1,500/µL), thrombocytopenia (a platelet count less than 150,000/microL), or anemia - HGB < 12 or < 11 g/dLin men and in women respectively.
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 |
|---|---|---|
| Mycophenolate MofetilAlso: CellCept | Drug | A |
| Mycofenolate Mofetil (MMF)Also: CellCept2000 mg per day divided into 2 equal doses. | Drug | A |
| PlaceboPlacebo | Drug | B |
Primary outcome
To compare CellCept 2 grams daily to placebo for effects on overall IC symptoms and well being in patients with refractory PBS/IC.
Time frame: 12 Weeks
To assess the safety profile of CellCept in the treatment of refractory PBS/IC.
Time frame: 12 Weeks
6 secondary outcomes
To investigate the association between clinical subgroups, characterized by differences in baseline characteristics (such as presence of ulcers, duration of symptoms, significant co-morbid diseases, serological abnormalities), and efficacy of CellCept.
Time frame: 12 Weeks
To assess the patterns of patient expectations, associations with symptom severity, and the potential impact of patient expectations on response to treatment.
Time frame: 12 Weeks
To assess patterns of treatment goals and goal achievement in this study population, as well as baseline characteristics and factors related to goal selection and achievement.
Time frame: 12 Weeks
To assess impact of study medication on pain medication use.
Time frame: 12 Weeks
To assess the frequency and mechanism of un-blinding on study results and assess how the patient's perception of which treatment they received changes over time and influences ultimate outcome.
Time frame: 12 Weeks
To assess the rate of detectable immune disorders in patients with PBS/IC refractory to standard treatment using CellCept.
Time frame: 12 Weeks
Results and safety
The study was stopped early (reason given: The major and primary reason for the study termination is the observed reduced efficacy of CellCept compared to placebo.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2010.
- Interstitial Cystitis AssociationLink provided by the sponsor.
Related trials for Interstitial Cystitis
Enrolling by invitationObservational study
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EPPIC: Easing Pelvic Pain Interventions Clinical Research Program
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Investigation of PEMF Therapy for Female Patients With IC/BPS
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Aerobic Exercise and Connective Tissue Massage in Painful Bladder Syndrome
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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.
John Kusek, PhD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
LeRoy Nyberg, MD, PhD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Richard Landis, PhD principal investigator
University of Pennsylnania
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mycophenolate Mofetil and Mycofenolate Mofetil (MMF) in people with Interstitial Cystitis and Painful Bladder Syndrome, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 11 sites in 2 countries.
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 6 inclusion and 20 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: stopped early, as of the sponsor's last update on 14 Jan 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Canada. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The major and primary reason for the study termination is the observed reduced efficacy of CellCept compared to placebo.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To compare CellCept 2 grams daily to placebo for effects on overall IC symptoms and well being in patients with refractory PBS/IC. (time frame: 12 Weeks). 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 14 Jan 2010.