Phase 1 Study in Subjects With OAB Assessing the Safety and Activity of hMaxi-K Gene Transfer

Conditions:Overactive Bladder
Therapuetic Areas:Gastroenterology
Age Range:18 - Any
Start Date:September 2013
End Date:July 2017
Contact:Wendy Espinal

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Phase 1 Double Blind, Placebo Controlled Study Assessing Safety and Activity Of 2 Escalating Doses of hMaxi-K Gene Transfer By Direct Injection Into the Bladder Wall In Female Subjects With OAB (Overactive Bladder Syndrome and Detrusor Overactivity)

The primary objective of this study is to evaluate the safety of a single treatment of
hMaxi-K compared to placebo (PBS-20% sucrose) administered by direct bladder wall
injections. Two dose levels (16000 µg and 24000 µg -20 or 30 bladder wall injections,
respectively) in females with moderate OAB/DO of ≥ six months duration will be evaluated. In
each dose level, 6 participants will receive hMaxi-K and 3 will receive placebo. .

In addition secondary efficacy parameters will be evaluated (change from baseline compared
to placebo), including number of micturitions per day, volume of micturitions, incontinence
episodes, pad weight, uninhibited contractions during cystoscopy, and quality of life

This is a Phase 1 double blind, placebo controlled study assessing safety and activity of 2
escalating doses of hMaxi-K gene transfer by direct injection into the bladder wall in
female subjects with OAB (Overactive Bladder Syndrome and Detrusor Overactivity).

There will be a total of 9 visits and a 1 and 3 day post dosing telephone contact with the
patient to evaluate for any new complaints. Following screening visits(V1 and V1A) and study
drug administration at week 0, eligible participants will be evaluated for safety post
dosing with study drug at weeks 1, 2, 4, 8, 12, and 24.

At Screening, Baseline (prior to dosing) and 1, 2, 4, 8, 12 and 24 weeks post dosing
participants will have a complete physical exam including urogenital examination. ECG will
be performed at Screening, at Baseline, at one week post dosing and at the final visit.

At Screening, Baseline (prior to dosing) and 1, 2, 4, 12 and 24 weeks post dosing,
laboratory evaluations including chemistry, hematology, and urinalysis will be performed.
Urine cultures will be done throughout the study (including prior to catheterization and at
discharge) to insure the absence of infection.

Participants will be assessed for study drug effects on incontinence at Baseline (prior to
dosing) and at weeks 4 and 24 post dosing by cystometry. At Screening (V1) and at 2, 8 and
24 weeks post dosing participants will have a bladder scan to evaluate for residual volume.

Subjects will complete a daily diary at home for 7 days prior to the baseline (V2). Diaries
will capture number of micturitions, volume of micturitions, number of urge incontinence
episodes, and rating of episode, and the number of pads used every 24 hours. The
participants will continue to complete this daily diary during the course of the trial and
the diary will be evaluated at each follow-up visit.

The participants will evaluate their perception of their bladder condition, QoL using Kings
Health Questionnaire (KHQ) and SF-12. International Consultation on Incontinence
Questionnaire - Short Form (ICIQ-SF) will be completed throughout the study.

Inclusion Criteria:

1. Healthy women of ≥18 years of age and non-childbearing potential

2. Symptoms of overactive bladder for ≥6 months including at least one of the following:

1. Frequent micturition ≥8 times per 24 hrs

2. urinary urgency or nocturia

3. Urge urinary incontinence five or more incontinence episodes per week

3. Detrusor overactivity with ≥1 uncontrolled phasic contraction(s) of the detrusor of
at least 5 cm/ H20 pressure documented on cystometry at Screening Visit 1A

4. residual volume of ≤200 ml

5. Non-response or poor tolerance to previous treatment for symptoms of OAB/urinary
incontinence and do not wish to continue these treatments

6. Have screening laboratory values and ECG that are within the normal range

7. Able to understand study requirements (i.e., literate in English), give written
informed consent, and comply with all study procedures and requirements.

Exclusion Criteria:

1. A woman with a positive serum (HCG) pregnancy test or lactating.

2. History of≥3 culture documented recurrent UTIs per year.

3. Current history of bladder outlet obstruction secondary to urethral stenosis, third
degree cystocele, or obstruction from prior urethral sling surgery documented by
cystoscopy, urothelial cancer, or other significant genitourinary disorders except

4. 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.

5. A screening urine culture (≥ 1000 colonies/ml) of a urinary pathogen from a freshly
voided urine.

6. Current history of neurological bladder dysfunction.

7. A life expectancy of <12 months.

8. Current history of ≥Grade 2 cystocele.

9. Stress or mixed urinary incontinence:

10. Indwelling urethral catheter or need for clean intermittent self-catheterization.

11. Any screening laboratory values outside of the normal laboratory range.

12. A hemoglobin A1c >7% in a person with diabetes.

13. A history of sickle-cell disease, sickle cell trait, or any other medical condition
that would produce significant risk to the patient.

14. Any condition that would interfere with participation in the study (including
geographical inaccessibility)

15. A current malignancy (basal cell carcinoma is not an exclusion).

16. Had within six months prior to enrollment any of the following:

- Myocardial infarction

- Cerebrovascular accident

- Uncontrolled hypertension (systolic >160 or diastolic >100mmHg)

- Arrhythmia

- Congestive heart failure (dyspnea on minimal exertion or while supine)

- Unstable angina (chest pain greater than three times weekly while on therapy)

- Required treatment with calcium channel, beta-blocker medication, nitrates, or
anti-epileptic drugs

17. Latex, lidocaine, xylocaine or other local anesthetics allergy

18. Known current drug addiction and/or alcohol abuse.

19. Mental or legal incapacity.

20. Abnormal ECG.

21. QTc of ≥470ms and/or family history of sudden death or participants with a family
history of long-QT Syndrome.

22. Any current treatment or treatment within the last 14 days for OAB including herbal
preparations and Botox within the last 6 months.

23. History of urinary retention, or residual bladder volume > 200 ml)

24. Treatment with any compound likely to increase urine production rate, or any compound
likely to affect detrusor mechanics or voiding habits

25. Participation in any other interventional or investigational clinical study during
the last 2 months before inclusion, or during the study period.
We found this trial at
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Garden City, New York 11530
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New York, New York
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