A Study to Investigate Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of ASP6294 in the Treatment of Female Subjects With Bladder Pain Syndrome/Interstitial Cystitis (SERENITY)
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|ClinicalTrials.gov Identifier: NCT03282318|
Recruitment Status : Recruiting
First Posted : September 13, 2017
Last Update Posted : June 29, 2018
|Condition or disease||Intervention/treatment||Phase|
|Bladder Pain Syndrome Interstitial Cystitis||Drug: ASP6294 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||163 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase 2a, Randomized, Double-blind, Placebo-controlled, Parallel-group, Proof of Concept Study to Investigate Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of ASP6294 in the Treatment of Female Subjects With Bladder Pain Syndrome/Interstitial Cystitis|
|Actual Study Start Date :||September 28, 2017|
|Estimated Primary Completion Date :||May 2019|
|Estimated Study Completion Date :||May 2019|
Participants will receive subcutaneous (SC) administrations of ASP6294 at weeks 0, 4 and 8.
Subcutaneous (SC) injection
Placebo Comparator: Placebo
Participants will receive SC administrations of Placebo at weeks 0, 4 and 8.
- Change from baseline in average mean daily pain (MDP) at Visit 6/Week 12 [ Time Frame: Baseline and Week 12 ]Participants will record participant's MDP each day in the evening into an e-diary. The MDP is the average pain experienced over the past 24 hours. MDP is measured using the 11-point numerical rating scale (NRS), (0-10) with 0 being no bladder pain and 10 being the worst possible pain.
- Change from baseline in average worst daily pain (WDP) at Visit 6/Week 12 [ Time Frame: Baseline and Week 12 ]Participants will record participant's WDP each day in the evening into an e-diary. The WDP is the worst pain experienced over the past 24 hours. WDP is measured using the 11-point numerical rating scale (NRS), (0-10) with 0 being no bladder pain and 10 being the worst possible pain.
- Change from baseline in mean voiding frequency at Visit 6/Week 12 [ Time Frame: Baseline and Week 12 ]The mean voiding frequency is the mean of the recordings of voiding frequency in the 3 day electronic micturition diary in the week prior to the visit, with at least 2 days recorded in that week.
- Change from baseline in mean number of level 3 or 4 urgency episodes (using the PPIUS) per 24 hours as assessed with the 3 day electronic micturition diary within the week preceding the study visit, at Visit 6/Week 12 [ Time Frame: Baseline and Week 12 ]The patient perception of intensity of urgency scale (PPIUS) is a 5-point categorical scale and has a range from 0-4. 0 meaning "No urgency, I felt no need to empty my bladder, but did so for other reasons." and 4 meaning "Urge incontinence, I leaked before arriving to the toilet."
- Assessment of the Global Response Assessment (GRA) at Visit 6/Week 12 [ Time Frame: Up to Week 12 ]The GRA is self-reported and is a 7 grade GRA used to evaluate a participant's clinical condition relative to Baseline. The GRA reads: As compared to when the participant started the study, how would the participant rate the participant's overall symptoms now? The 7 GRA grades are "markedly worse", "moderately worse", "slightly worse", "no change", "slightly improved", "moderately improved" or "markedly improved". A successful GRA response is defined as the scores "moderately improved" or "markedly improved" disease.
- Change from baseline in Bladder Pain/ Interstitial Cystitis Symptom Score (BPIC-SS) at Visit 6/Week 12 [ Time Frame: Baseline and Week 12 ]The BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over the previous 7 days. The BPIC-SS addresses urinary symptoms in 5 questions, all rated 0 - 4 (how often urinated because of pain, need to urinate just after previous urination, urination to avoid pain, pressure in the bladder and pain in the bladder), 2 questions address the impact of bladder pain (bothered by frequent urination during daytime and nighttime) and ends with a 0 - 10 NRS on worst pain over the last 7 days with 0 being no bladder pain and 10 being the worst possible pain. The total score will range from 0 to 38 with higher scores representing a worse situation, with a score of 19 or more indicating moderate/severe disease activity. Question 8 from the BPIC-SS is the question on worst bladder pain over the last 7 days on a 0-10 point NRS.
- Safety assessed by frequency, nature and severity of Adverse Events (AEs) [ Time Frame: Up to Week 18 ]An AE is considered "serious" if the investigator or sponsor view any of the following outcomes: Death, life-threatening, persistent or significant disability/incapacity, congenital anomaly or birth defect, hospitalization, or medically important event.
- Number of participants with laboratory value abnormalities and/or AEs [ Time Frame: Up to Week 18 ]Number of participants with potentially clinically significant laboratory values.
- Number of participants with vital signs abnormalities and/or AEs [ Time Frame: Up to Week 18 ]Number of participants with potentially clinically significant vital sign values.
- Safety assessed by 12- lead electrocardiogram (ECG) [ Time Frame: Up to Week 18 ]ECGs will be recorded with the participant in the supine position, after the participant has been lying down for approximately 5 minutes. There should be at least 5 minutes between ECG measurements in case a repeat is needed. Any clinically significant adverse changes on the ECG will be reported as an AE.
- Safety assessed by sensory testing [ Time Frame: Up to Week 18 ]The sensory status will be assessed bilaterally on the participant's first toe, using 3 tests: the vibration test, the light touch test and the pin prick test.
- Number of participants with Physical Exam abnormalities and/or AEs/Serious Adverse Events (SAEs) [ Time Frame: Up to Week 18 ]Number of participants with potentially clinically significant physical exam values.
- Pharmacokinetics assessed by serum level of ASP6294 [ Time Frame: Up to Week 18 ]ASP6294 serum levels will be evaluated in blood samples collected.
- Pharmacodynamics assessed by serum level of total nerve growth factor (NGF) [ Time Frame: Up to Week 18 ]Total NGF serum levels will be evaluated in blood samples collected.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03282318
|Contact: Clinical Science||+31 (0) 71 5455 firstname.lastname@example.org|
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|Study Director:||Project Physician||Astellas Pharma Europe B.V.|