An Open Label, Phase 1/2a Trial to Evaluate the Efficacy, Safety, and Tolerability of KU002 Given as Intravesical Instillations in Subjects with Interstitial Cystitis (IC)/Bladder Pain Syndrome (BPS)*

Phase 2

Completed

• Conditions: Bladder Pain Syndrome; Interstitial Cystitis; 10004994

• Registration Number: NL-OMON52788
• Lead Sponsor: Kuste Biopharma

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 2023-07-24
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 14

Inclusion Criteria

1. Be at least 18 years of age.
2. Willing to provide written informed consent.
3. Willing and able to comply with study requirements and visit schedule.
4. Have a diagnosis of IC/BPS for at least 6 weeks prior to screening.
5. Have undergone a cystoscopy and have visible signs of Hunner*s lesion (photo
documented) within 6 weeks prior to study inclusion. If the cystoscopy was not
performed within 6 weeks of study inclusion, a new cystoscopy (incl photo of
the Hunner*s lesion) must form part of the screening eligibility procedure.
6. Have an average daily pain score (recorded over 3 days) > 4 but <9 on
the 11-point Numerical Rating Scale (NRS) in the 3 days prior to inclusion.
7. Be ambulatory and able to use the toilet independently.
8. Have a body mass index >=19 kg/m2 but <=35 kg/m2.
9. Have a pre-dose mean systolic/diastolic blood pressure of <=140/90 mmHg
before randomization can occur.
10. Must not be pregnant, lactating, or actively trying to become pregnant,
Subjects who are premenopausal and of childbearing potential must have a
negative pregnancy test at Screening (serum) and at Day 0 (urine) and must use
a medically acceptable and effective method of birth control for the duration
of the study, which can include:
a. Having a male partner who is sterile prior to the female subject*s entry
into the study and is the sole sexual partner for that female subject.
b. Use of double-barrier methods of contraception; condoms with the use of caps
(with spermicide) and intra-uterine devices are acceptable.
c. Use of hormonal contraceptives (oral, depots, patches, etc.) with
double-barrier methods of contraception as outline above.
d. True abstinence: When this is in line with the preferred and usual lifestyle
of the subject (period abstinence [eg, calendar, ovulation, symptothermal,
post-ovulation methods] and withdrawal are not acceptable methods of
contraception).
e. Male study participants must use a condom for sexual intercourse from
screening until at least 90 days after last dose of study drug, unless they
have been surgically sterilized (vasectomy).

Exclusion Criteria

1. Current or recent (30 days prior to screening) change in any pharmacologic
agent or invasive procedure (e.g. fulguration) used to treat the IC/BPS
condition. Patient is eligible to participate in the study if their treatment
for IC/BPS has remained stable for the past month prior to entry.
2. Concurrent (at Screening), recent (within 30 days), chronic, or recurrent
(>4 per year) urinary tract infections (positive dipstick for urinary tract
infection and abnormal microscopic evaluation, signs and symptoms) or
unevaluated microhematuria.
3. Having a history of previous procedure that affects bladder function (e.g.
augmentation, cystoplasty, cystectomy, partial bladder resection, cystolysis
etc.).
4. History of cyclophosphamide or chemical cystitis, urinary tuberculosis,
radiation cystitis or history of pelvic irradiation.
5. Electrostimulation, biofeedback, or bladder training therapy (behavioral
therapy), during the previous month prior to Screening, or the intention to
initiate such therapies during the study.
6. Postvoid residual (PVR) urine volume >150 mL.
7. Diagnosis of dementia.
8. Subjects with uncontrolled hypertension.
9. Documented history of myocardial infarction, unstable angina, and/or has
undergone coronary artery bypass surgery and/or percutaneous transluminal
coronary angioplasty in the past year.
10. Congestive heart failure (New York Heart Association Class III or IV heart
failure).
11. Any concurrent condition or any clinically significant abnormality on the
Screening physical examination, laboratory tests, electrocardiogram (ECG;
including ischemic heart disease), Hepatitis B or C, which, in the opinion of
the Investigator, may affect the interpretation of efficacy or safety data, or
which otherwise contraindicates participation in a clinical study with KU002.
a) Hypersensitivity to KU002 or any of its ingredients.
b) History of clinically significant drug hypersensitivity.
c) History of urogenital neoplasms or malignancies including bladder, urethra,
uterine, cervical or vaginal cancer.
d) For men: history of prostate surgery (transurethral resection of the
prostate [TUR-P], transurethral resection of tumor, [TUR-T], transurethral
incision of the prostate [TUIP], transurethral needle ablation [TUNA]), history
of prostate cancer or currently (within 30 days) being treated for chronic
bacterial prostatitis.
e) Subjects with neuropathology that could affect the lower urinary tract or
nerve supply, including but not limited to multiple sclerosis, stroke,
Parkinsonism, or spinal cord injury.
f) Clinically significant or unstable endocrine, hepatic, renal, immunologic
(incl immune suppressive and autoimmune) disease or malignancy other than
non-melanomatous skin cancer.
12. Severe renal impairment (estimated glomerular filtration rate < 30
mL/min/1.73m2).
13. Hepatic impairment (Child-Pugh B or greater).
14. Use of medications for BPH (eg Tamsulosin, silodosin, alfulozin and
finasteride) within a month prior to study entry.
15. History of an addiction to drugs or alcohol within the last 2 years prior
to Screening as determined by the Investigator.
16. Participation in a clinical study within a month prior to Screening, or
exposure to an investigational drug which has not washed out for at least 5
half-lives since the las

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>-Incidence and severity of AEs over the course of the study.<br /><br>• Hematology, biochemistry, and urinalysis. While total white blood cell (WBC)<br /><br>count will be expressed in absolute values, differential count will be<br /><br>expressed as both absolute count and percentage of WBCs.<br /><br>• Absolute change from baseline to EoT+1 in ECG readings.<br /><br>• ECG parameters of interest include ventricular rate, QT interval, corrected<br /><br>QT interval, PR interval, and QRS duration.<br /><br>• Absolute change from baseline to each visit in standardized cuff systolic and<br /><br>diastolic blood pressure and radial heart rate.<br /><br>• Pharmacokinetics: Determination of concentration of KU002 in plasma at the<br /><br>given time points, presented descriptively.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>-Absolute change in pain, measured as mean value over 3 days, on the 11-point<br /><br>Numerical Rating Scale from baseline to EoT+1.<br /><br>• Change in O*Leary Sant ICSI-ICPI participant reported questionnaire from<br /><br>baseline to EoT+1.<br /><br>• Responder analysis: Proportion of subjects who have at least a 2-point<br /><br>reduction in pain score measured by NRS between baseline and EoT+1.<br /><br>• Change in the maximum daily pain score from baseline to EoT+1.<br /><br>• Change in Subject*s Global Response Assessment (GRA) at EoT+1.<br /><br>• Change in NRS, O*Leary Sant ICSI-ICPI after 3 instillations and to EoT+1.<br /><br>• Change in 3-day voiding diary from baseline to EoT+1.<br /><br>• A visual inspection of Hunner*s lesion before and after treatment to<br /><br>determine any effect on the lesion.</p><br>