Overactive BladderPhase 1NCT00495053
Safety Study of hMaxi-K Gene Transfer to Treat Overactive Bladder and Detrusor Overactivity
A Phase 1 study testing hMaxi-K in people with Overactive Bladder, run by Urovant Sciences GmbH at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the safety of a new product that uses human gene transfer (called hMaxi-K) when it is given to patients with overactive bladder. Human gene transfer is a new type of therapy that is the process of placing genetic material (DNA or RNA) into a person. Read more about this study
- Who is it for?
- Ages 18 years and older, women onlyHealthy volunteers can take part
- Conditions the study is for
- Overactive Bladder
- What is being tested?
- hMaxi-K
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2007to Nov 2009
- Phase
- Phase 1
- 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
- Women only
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Healthy women of 18 years of age or older and of non-childbearing potential
- Clinical symptoms of overactive bladder for 6 months or longer including at least one of the following:
- frequent urination (that is, having to go to the bathroom 8 or more times per day);
- symptoms of urinary urgency, which is experiencing a sudden need to pass urine that is difficult to hold back; or the complaint of waking at night two ore more times to urinate;
- urge urinary incontinence, which is the complaint of leakage of urine that cannot be stopped, accompanied by or immediately preceded by urgency 5 or more times per week.
- Non-response or poor tolerance to previous treatment for symptoms of overactive bladder and do not wish to continue with that treatment.
Who cannot join
- A woman with a positive serum (HCG) pregnancy test or who is lactating
- History of three or more culture-documented recurrent urinary tract infections per year
- Current history or previous diagnosis of painful bladder syndrome (interstitial cystitis) with pain in the region of the pelvis, perineum, or lower abdomen relieved by voiding
- Current history of neurological bladder dysfunction
- A life expectancy of less than 12 months
- Current history of Grade 2 or greater cystocele
Show all 11Show fewer
- An indwelling urethral catheter or need for clean intermittent self- catheterization
- Recent heart attack
- Uncontrolled diabetes
- Latex allergy
- Stress urinary incontinence as determined by observation of the participant coughing while standing with a full bladder and/or response of 2 or 3 on the following Stress Urinary Incontinence question: Do you experience leakage when laughing, coughing, lifting heavy objects or other types of discreet, moderately intense activities? 0=NONE: No leakage. 1=MILD: Minimal leakage on rare occasions during these types of activities; easily tolerated; do not use pads for this. 2=MODERATE: Enough leakage that it requires occasional use of pads and may interfere with usual activity and tasks. 3=SEVERE: Extreme leakage and discomfort that stops all activity and tasks and requires the use of pads on all occasions
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Multicenter Study Evaluating the Safety and Potential Activity Of Three Escalating Doses of hMaxi-K Gene Transfer In Female Participants With Overactive Bladder Syndrome and Detrusor Overactivity: Double Blind, Imbalanced Placebo Controlled Design Within 3 Sequential Active Treatment Groups
The purpose of this study is to evaluate the safety of a new product that uses human gene transfer (called hMaxi-K) when it is given to patients with overactive bladder. Human gene transfer is a new type of therapy that is the process of placing genetic material (DNA or RNA) into a person. The primary objective of this study is to evaluate safety parameters occurring subsequent to administration of a single intravesical instillation of study drug.
Two different dose groups of hMaxi-K will be tested in this study: 5000 and 10000 micrograms. A 15000 microgram group was planned; however, the study was terminated before participants were dosed. hMaxi-K will be given as a single administration into the bladder through a catheter. Each women's participation in the study will last for up to 24 weeks (followed by an additional 18-month follow-up period).
Read the detailed description
The safety parameters to be monitored include: adverse events, clinical laboratory tests, electrocardiograms, and physical examinations.
The secondary objective is to evaluate the efficacy of a single intravesical instillation of hMaxi-K compared to the control group. Efficacy parameters that will be evaluated are: mean number of micturitions per 24 hours, mean number of urge incontinence episodes per 24 hours, mean number of urgency episodes per 24 hours, overall maximum bladder capacity, number of uninhibited contractions during the cystometry procedure, participant rating of urgency score, participant rating of perceived bladder condition severity, participant assessment of response to treatment, participant rating of Quality of Life (Kings Health Questionnaire), SF-12 Health Survey, International Consultation on Incontinence Questionnaire (ICIQ-SF), and pad weight measurement of accidental bladder leaks.
- Conditions
- Overactive Bladder
- Being tested
- hMaxi-K, Placebo
- Keywords
- overactive bladder, urinary urgency
- Medical subject headings
- Urinary Bladder, Overactive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hMaxi-KDrug
Also called URO-902
Two dose levels (5000 µg/90 mL intravesical instillation and 10000 µg/90 mL intravesical instillation)
PlaceboOther
Matching placebo (PBS-20% sucrose)
| Study group | Role | What they receive |
|---|---|---|
| hMaxi-K 5000 µg/mL | Experimental | 5000 micrograms (µg)/90 milliliter (mL) intravesical instillation Receives: Drug: hMaxi-K |
| hMaxi-K 10000 µg/mL | Experimental | 10000 µg/90 mL intravesical instillation Receives: Drug: hMaxi-K |
| Placebo | Placebo comparator | Matching placebo (PBS-20% sucrose) Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with any treatment-emergent adverse event during the treatment period
Measured: 6 months (24 weeks per participant)
Number of participants with any treatment-emergent adverse event during the safety follow-up period
Measured: 18 months
Number of participants with any abnormal, clinically significant clinical laboratory test value during the treatment period
Measured: 6 months (24 weeks per participant)
Number of participants with any abnormal, clinically significant clinical laboratory test value during the safety follow-up period
Measured: 18 months
Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the treatment period
Measured: 6 months (24 weeks per participant)
Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the safety follow-up period
Measured: 18 months
Number of participants with any abnormal, clinically significant clinical physical examination finding during the treatment period
Measured: 6 months (24 weeks per participant)
Number of participants with any abnormal, clinically significant clinical physical examination finding during the safety follow-up period
Measured: 18 months
Secondary outcomes (12)
Change from Baseline in the mean number of micturitions per 24 hours
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the mean number of urge incontinence episodes per 24 hours (collected over a 3-day period)
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the mean number of urgency episodes per 24 hours
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the overall maximum bladder capacity
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the number of uninhibited contractions during the cystometry procedure
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the participant rating of urgency score
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the participant rating of perceived bladder condition severity
Measured: Baseline; 6 months (24 weeks) per participant
Number of participants with the indicated response to the following question, as a measure of the participants' assessment of response to treatment: "Has the treatment been of benefit to you"?
Measured: 6 months (24 weeks) per participant
Change from Baseline in the Kings Health Questionnaire-Quality of Life (KHQ-QOL) general health perceptions domain score and impact on life domain score
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the Short Form-12 (SF-12) Health Survey
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the International Consultation on Incontinence Questionnaire - Short Form (ICIQ-SF)
Measured: Baseline; 6 months (24 weeks) per participant
Change from Baseline in pad weight
Measured: Baseline; 6 months (24 weeks) per participant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2007 (estimated)
- Study startApr 2007
- Primary completionJul 2008
- Study completionNov 2009
Why it stopped: Slow enrollment and financial issues
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Overactive BladderStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- sequential, dose escalation
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Urovant Sciences GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ion Channel Innovations
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00495053
- Sponsor's protocol ID
- ION-02
- Status last verified
- Jun 2019
- First submitted
- 29 Jun 2007
- Last updated
- 28 Jun 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Multicenter Study Evaluating the Safety and Potential Activity Of Three Escalating Doses of hMaxi-K Gene Transfer In Female Participants With Overactive Bladder Syndrome and Detrusor Overactivity: Double Blind, Imbalanced Placebo Controlled Design Within 3 Sequential Active Treatment Groups
- ClinicalTrials.gov ID
- NCT00495053
- Acronym / sponsor ID
- ION-02
- Conditions
- Overactive Bladder
- Keywords
- overactive bladder, urinary urgency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow enrollment and financial issues
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Urovant Sciences GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Ion Channel Innovations
- Study start
- Apr 2007
- Primary completion
- Jul 2008 (actual)
- Study completion
- Nov 2009 (actual)
- First posted
- 2 Jul 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 evaluate the safety of a new product that uses human gene transfer (called hMaxi-K) when it is given to patients with overactive bladder. Human gene transfer is a new type of therapy that is the process of placing genetic material (DNA or RNA) into a person. The primary objective of this study is to evaluate safety parameters occurring subsequent to administration of a single intravesical instillation of study drug.
Two different dose groups of hMaxi-K will be tested in this study: 5000 and 10000 micrograms. A 15000 microgram group was planned; however, the study was terminated before participants were dosed. hMaxi-K will be given as a single administration into the bladder through a catheter. Each women's participation in the study will last for up to 24 weeks (followed by an additional 18-month follow-up period).
Detailed description
The safety parameters to be monitored include: adverse events, clinical laboratory tests, electrocardiograms, and physical examinations.
The secondary objective is to evaluate the efficacy of a single intravesical instillation of hMaxi-K compared to the control group. Efficacy parameters that will be evaluated are: mean number of micturitions per 24 hours, mean number of urge incontinence episodes per 24 hours, mean number of urgency episodes per 24 hours, overall maximum bladder capacity, number of uninhibited contractions during the cystometry procedure, participant rating of urgency score, participant rating of perceived bladder condition severity, participant assessment of response to treatment, participant rating of Quality of Life (Kings Health Questionnaire), SF-12 Health Survey, International Consultation on Incontinence Questionnaire (ICIQ-SF), and pad weight measurement of accidental bladder leaks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- sequential, dose escalation
| Arm | Type | Receives |
|---|---|---|
| hMaxi-K 5000 µg/mL | Experimental | Drug: hMaxi-K5000 micrograms (µg)/90 milliliter (mL) intravesical instillation |
| hMaxi-K 10000 µg/mL | Experimental | Drug: hMaxi-K10000 µg/90 mL intravesical instillation |
| Placebo | Placebo comparator | Other: PlaceboMatching placebo (PBS-20% sucrose) |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Healthy women of 18 years of age or older and of non-childbearing potential
- Clinical symptoms of overactive bladder for 6 months or longer including at least one of the following:
- Non-response or poor tolerance to previous treatment for symptoms of overactive bladder and do not wish to continue with that treatment.
Key exclusion criteria (11 listed)
- A woman with a positive serum (HCG) pregnancy test or who is lactating
- History of three or more culture-documented recurrent urinary tract infections per year
- Current history or previous diagnosis of painful bladder syndrome (interstitial cystitis) with pain in the region of the pelvis, perineum, or lower abdomen relieved by voiding
- Current history of neurological bladder dysfunction
- A life expectancy of less than 12 months
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 |
|---|---|---|
| hMaxi-KAlso: URO-902Two dose levels (5000 µg/90 mL intravesical instillation and 10000 µg/90 mL intravesical instillation) | Drug | hMaxi-K 10000 µg/mL, hMaxi-K 5000 µg/mL |
| PlaceboMatching placebo (PBS-20% sucrose) | Other | Placebo |
Primary outcome
Number of participants with any treatment-emergent adverse event during the treatment period
Time frame: 6 months (24 weeks per participant)
Number of participants with any treatment-emergent adverse event during the safety follow-up period
Time frame: 18 months
Number of participants with any abnormal, clinically significant clinical laboratory test value during the treatment period
Time frame: 6 months (24 weeks per participant)
Number of participants with any abnormal, clinically significant clinical laboratory test value during the safety follow-up period
Time frame: 18 months
Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the treatment period
Time frame: 6 months (24 weeks per participant)
Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the safety follow-up period
Time frame: 18 months
Number of participants with any abnormal, clinically significant clinical physical examination finding during the treatment period
Time frame: 6 months (24 weeks per participant)
Number of participants with any abnormal, clinically significant clinical physical examination finding during the safety follow-up period
Time frame: 18 months
12 secondary outcomes
Change from Baseline in the mean number of micturitions per 24 hours
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the mean number of urge incontinence episodes per 24 hours (collected over a 3-day period)
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the mean number of urgency episodes per 24 hours
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the overall maximum bladder capacity
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the number of uninhibited contractions during the cystometry procedure
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the participant rating of urgency score
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the participant rating of perceived bladder condition severity
Time frame: Baseline; 6 months (24 weeks) per participant
Number of participants with the indicated response to the following question, as a measure of the participants' assessment of response to treatment: "Has the treatment been of benefit to you"?
Time frame: 6 months (24 weeks) per participant
Change from Baseline in the Kings Health Questionnaire-Quality of Life (KHQ-QOL) general health perceptions domain score and impact on life domain score
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the Short Form-12 (SF-12) Health Survey
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the International Consultation on Incontinence Questionnaire - Short Form (ICIQ-SF)
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in pad weight
Time frame: Baseline; 6 months (24 weeks) per participant
Results and safety
The study was stopped early (reason given: Slow enrollment and financial issues). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2019.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing hMaxi-K in people with Overactive Bladder, run by Urovant Sciences GmbH at 1 site in United States.
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 11 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 28 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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.
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: Slow enrollment and financial issues). 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?
Number of participants with any treatment-emergent adverse event during the treatment period (time frame: 6 months (24 weeks per participant)). The study lists 8 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 28 Jun 2019.