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Bladder Pain SyndromePhase 2SERENITYNCT03282318

A Study to Investigate Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of ASP6294 in the Treatment of Female Subjects With Bladder Pain Syndrome/Interstitial Cystitis

A Phase 2 study testing ASP6294 in people with Bladder Pain Syndrome and Interstitial Cystitis, run by Astellas Pharma Europe B.V. at 28 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to investigate efficacy, safety and tolerability of ASP6294 in female participants with Bladder Pain Syndrome/Interstitial Cystitis (BPS/IC). This study will also investigate the pharmacokinetics (PK) and pharmacodynamics (PD) of ASP6294 in female participants with BPS/IC. Read more about this study

Who is it for?
Ages 18 years and older, women only
Conditions the study is for
Bladder Pain Syndrome, Interstitial Cystitis
What is being tested?
ASP6294
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
119 participants
Where
28 sitesin 10 countries
Study dates
28 Sep 2017to 21 Mar 2019
Phase
Phase 2
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
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subject's signs, symptoms and diagnostic work-up are in accordance with the international society for the study of bladder pain syndrome (ESSIC) definition for bladder pain syndrome/interstitial cystitis (BPS/IC): pelvic pain, pressure or discomfort perceived to be related to the urinary bladder accompanied by at least 1 other urinary symptom such as persistent urge to void or frequency, for at least 6 months in absence of urinary infection or other obvious pathology or identifiable causes. There is documented proof of the diagnosis BPS/IC that has been entered into the subject's records at least 2 months prior to Visit 1/Screening.
  • Subject has a score of ≥ 4 and ≤ 9 for pain as assessed by scoring the average pain of the week preceding Visit 1/Screening, using an 11-point NRS (0-10).
  • Subject has an estimated voiding frequency of ≥ 8 and ≤ 30 voids per 24 hours.
  • Subject has a score of ≥ 7 on the interstitial cystitis symptom index (ICSI) questionnaire.
  • Subject must either:
  • Be of nonchildbearing potential:
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  • Postmenopausal (defined as at least 1 year without any menses for which there is no other obvious pathological or physiological cause) prior to screening, or
  • Documented surgically sterile (e.g., hysterectomy, bilateral salpingectomy, bilateral oophorectomy)
  • Or, if of childbearing potential,
  • Agree not to try to become pregnant during the study and for 5 half-lives (i.e., 70 days) after the final study drug administration at Visit 5/Week 8, and
  • Have a negative urine pregnancy test at Visit 1/Screening, and
  • If heterosexually active, agree to consistently use 1 form of highly effective birth control starting at screening and throughout the study period and for 5 half-lives (i.e., 70 days) after the final study drug administration at Visit 5/Week 8.
  • Subject must agree not to breastfeed starting at screening and throughout the study period, and for 5 half-lives (i.e., 70 days) after the final study drug administration at Visit 5/Week 8.
  • Subject must agree not to donate ova starting at screening and throughout the study period, and for 5 half-lives (i.e., 70 days) after the final study drug administration at Visit 5/Week 8.
  • Subject must be willing and able to comply with study requirements (e.g., complete questionnaires and diaries, able to read and attend all required study visits).
  • Subject agrees not to participate in another interventional study while participating in the present study (i.e., between Visit 1/Screening and Visit 7/Week 18).
  • Subject has undergone at least 2 different therapies for BPS/IC with unsatisfactory results, prior to study entry.
  • Subject has at least moderate pain as reflected by an average MDP of ≥ 4.0 and ≤ 9.0. The average MDP is the average of daily assessments of MDP in the week prior to the visit with at least 5 recordings. Additionally, the MDP recordings must not differ over 4 points between consecutive days.
  • Subject has a mean voiding frequency of ≥ 8.0 and ≤ 30.0 per 24 hours as assessed with the 3 day electronic micturition diary in the week prior to the visit.
  • Subject is confirmed to be willing to comply and has shown to be compliant with all study requirements during the run-in period.

Who cannot join

  • Subject has osteoarthritis or has a history of rapidly progressive osteoarthritis.
  • Subject has a score of ≥ 30 on the Pain Catastrophizing Scale (PCS).
  • Subject has a score of > 12 on the HADS-D (Hospital Anxiety and Depression Scale - Depression subscale).
  • Subject has significant pelvic floor pain or spasm which is considered the main cause of the chronic pelvic/bladder pain as concluded by the investigator based on the pelvic floor examination.
  • Subject has undergone a fulguration or excision of a Hunner's lesion any time prior to the screening visit.
  • Subject has recently undergone or started treatment for BPS/IC as specified below:
Show all 41Show fewer
  • subject has undergone a cystoscopy with hydrodistension or Botox injections in the bladder within 6 months prior to the screening visit.
  • subject has received non-pharmacological interventions for BPS/IC (including but not limited to electric stimulation therapy or acupuncture therapy) within 3 months prior to the screening visit.
  • subject has received any intravesical pharmacological treatment for BPS/IC (including but not limited to heparin or dimethyl sulfoxide) within 4 weeks prior to the screening visit
  • subject had an initiation, discontinuation, or variation in the dose and/or frequency of antimuscarinics, mirabegron, antidepressants (including amitriptyline), anticonvulsants, benzodiazepines, skeletal muscle relaxants, nonsteroidal anti-inflammatory drugs, non-opioid analgesics, pentosan polysulphate sodium, homeopathic medication and/or herbal therapies during the last 4 weeks prior to the screening visit.
  • subject has had changes in non-pharmacological treatment for BPS/IC (e.g., diet or physical therapy) during the last 4 weeks prior to the screening visit.
  • Subject has bladder pathology as specified below:
  • a post-void residual (PVR) >200 mL.
  • subject has a known currently symptomatic urethral diverticulum.
  • subject has genital tract condition or pelvic pathology (e.g., post-partum, post-pelvic surgery, post-hysterectomy) that may complicate diagnosis and the evaluation of pelvic pain and urinary symptoms. Note: A history of a Cesarean section is not a reason for exclusion.
  • subject has a known currently symptomatic bladder or ureteral calculi.
  • subject currently has cystitis (radiation cystitis, Bacillus Calmette-Guérin-induced cystitis, bacterial/tuberculous cystitis, cyclophosphamide cystitis) or has had a documented symptomatic bacterial cystitis within the last 1 month prior to the screening visit. In case of bacterial cystitis (UTI), the subject can be re-screened 1 month after successful treatment.
  • subject has currently clinically significant urinary bladder abnormalities (e.g., bladder mass, bladder stone, bladder diverticulum, small contracted end-stage bladder), except for abnormalities associated with BPS/IC.
  • subject has had any invasive procedures of either the urinary bladder, urethra, ureter or renal pelvis (e.g., transurethral resection of bladder [including bladder biopsy], urethral dilatation, endovesicular lithotripsy) within 3 months prior to the screening visit.
  • subject has a known current neurologic disease or a defect affecting urinary bladder function (e.g., neurogenic bladder, systemic or central neurological disease, such as Multiple Sclerosis or Parkinson's disease).
  • subject has a known current lower urinary tract malignancy. In case of positive sediment (micro) hematuria results, local procedures/guidelines will need to be followed to exclude malignancy. Only if hematuria has been present within the last 6 months and malignancy has been adequately ruled out by the investigator according to local diagnostic procedures, the subject does not have to be excluded. Note that if the subject had a (negative) bladder biopsy, the subject can only be re-screened after 3 months following this biopsy. Documentation of all diagnostic outcomes and investigator's decisions need to be available.
  • Subject has a known history of, or currently has inflammatory bowel disease (i.e., Crohn's Disease or Ulcerative Colitis) and/or Sjögren Syndrome.
  • Subject has a known current severe constipation and/or severe diarrhea, severe active diverticulitis and/or severe gastrointestinal bleeding.
  • Subject has a known or suspected hypersensitivity to ASP6294 or any components of the formulation used.
  • Subject has been pregnant within 6 months prior to screening assessment or breast feeding within 3 months prior to screening.
  • Subject has a known history of an allergic or anaphylactic reaction to biological drugs (e.g., [monoclonal] antibodies including tanezumab or fusion proteins).
  • Subject has received a biological drug (e.g. [monoclonal] antibodies including tanezumab or fusion proteins) during the last 6 months prior to the screening visit.
  • Subject has a known history of hepatitis B or C or human immunodeficiency virus (HIV) infection.
  • Subject has a known history of or has a currently active or treated sexually transmittable disease (including genital herpes).
  • Subject has a known current substance abuse issue (including alcoholism).
  • Subject has peripheral neuropathy, or an abnormality has been observed during the sensory testing at Visit 1/Screening.
  • Subject has a known currently clinically severe, unstable or uncontrolled renal, hepatic, respiratory, hematological, genitourinary (except BPS/IC), cardiovascular, endocrine, neurological, psychiatric, or other medical illness that may put the subject at safety risk or may mask measures of efficacy.
  • Subject has had any malignancy diagnosed within 5 years prior to the screening visit, except for curative treated localized non-melanoma skin cancer (e.g. basal cell or squamous cell carcinoma).
  • Subject has a known current psychiatric condition, mental incapacity, language barrier or the inability to read that would impair the subject's successful participation in the study.
  • Subject has a body mass index of ≥ 40 kg/m^2 as sign of morbid obesity.
  • Subject has any condition that makes the subject unsuitable for study participation.
  • Subject has received investigational therapy (i.e., not yet approved experimental medicine) within 28 days or 5 half-lives, whichever is longer, prior to the screening visit.
  • Subject is an employee of the Astellas Group, the Contract Research Organization (CRO) involved, or the investigator site executing the study.
  • Results of the Visit 1/Screening blood sample indicate that the subject has active hepatic and/or biliary disease, defined as: liver enzymes aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 2 times the upper limit of normal (ULN), or total bilirubin (TBL) > 1.5 times the ULN.
  • Result of the Visit 1/Screening serum pregnancy test is positive.
  • Results of the Visit 1/Screening blood/urine samples indicate that the subject has clinically significant abnormal biochemistry, hematology or urinalysis safety laboratory values.

Trial locations and contacts

28 sites in 10 countries.

28 sites in 10 countries

Russia5 sites
  • Site RU70001

    Saint Petersburg

  • Site RU70002

    Saint Petersburg

  • Site RU70003

    Saint Petersburg

  • Site RU70006

    Saint Petersburg

  • Site RU70008

    Saint Petersburg

Other trials in Russia

Germany4 sites
  • Site DE49003

    Duisburg

  • Site DE49007

    Duisburg

  • Site DE49001

    Holzminden

  • Site DE49005

    Markkleeberg

Other trials in Germany

Latvia4 sites
  • Site LV37103

    Daugavpils

  • Site LV37101

    Riga

  • Site LV37102

    Riga

  • Site LV37105

    Riga

Other trials in Latvia

Poland4 sites
  • Site PL48009

    Lodz

  • Site PL48002

    Szczecin

  • Site PL48010

    Warsaw

  • Site PL48007

    Wroclaw

Other trials in Poland

Spain4 sites
  • Site ES34001

    Barcelona

  • Site ES34008

    Madrid

  • Site ES34002

    Málaga

  • Site ES34007

    Valencia

Other trials in Spain

Hungary2 sites
  • Site HU36002

    Budapest

  • Site HU36001

    Csongrád

Other trials in Hungary

United Kingdom2 sites
  • Site GB44002

    Manchester

  • Site GB44006

    Plymouth

Other trials in United Kingdom

Belgium1 site
  • Site BE32001

    Leuven

Other trials in Belgium

Czechia1 site
  • Site CZ42002

    Pilsen

Other trials in Czechia

Netherlands1 site
  • Site NL31007

    Sneek

Other trials in Netherlands

About this study

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

The purpose of this study is to investigate efficacy, safety and tolerability of ASP6294 in female participants with Bladder Pain Syndrome/Interstitial Cystitis (BPS/IC). This study will also investigate the pharmacokinetics (PK) and pharmacodynamics (PD) of ASP6294 in female participants with BPS/IC.

Being tested
ASP6294, Placebo
Keywords
Bladder Pain Syndrome, Interstitial Cystitis, ASP6294
Medical subject headings
Cystitis, Interstitial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ASP6294Drug

    Participants will receive 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

  • PlaceboDrug

    Participants will receive placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

Study groups in NCT03282318
Study groupRoleWhat they receive
ASP6294ExperimentalParticipants will receive 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.
Receives: Drug: ASP6294
PlaceboPlacebo comparatorParticipants will receive placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from Baseline in Average Mean Daily Pain (MDP) Score at Week 12

    Participants will record their MDP each day in the evening into an e-diary. The MDP is the average pain experienced over the past 24 hours. The average MDP is the mean of daily assessments of MDP in the week prior to the visit with at least 5 recordings in that week. MDP is measured using an 11-point Numerical Rating Scale (NRS) ranging from 0 (no bladder pain) to 10 (worst imaginable bladder pain). A negative change indicates a reduction/improvement from baseline.

    Measured: Baseline and Week 12

Secondary outcomes (6)
  1. Change from Baseline in Average Worst Daily Pain (WDP) Score at Week 12

    Participants will record their WDP each day in the evening into an e-diary. The WDP is the worst pain experienced over the past 24 hours. The average WDP is the mean of daily assessments of WDP in the week prior to the visit with at least 5 recordings in that week. WDP is measured using an 11-point NRS ranging from 0 (no bladder pain) to 10 (worst imaginable bladder pain). A negative change indicates a reduction/improvement from baseline.

    Measured: Baseline and Week 12

  2. Change From Baseline in Mean Voiding Frequency per 24 hours at Week 12

    Mean voiding frequency is the mean of the recordings of voiding frequency in the electronic micturition diary in the week prior to the visit with at least 2 days recorded in that week. A negative change indicates a reduction/improvement from baseline.

    Measured: Baseline and Week 12

  3. Change From Baseline in Mean Number of Level 3 or 4 Urgency Episodes (Based on Patient Perception of Intensity of Urgency Scale [PPIUS]) per 24 hours at Week 12

    The perceived level of urinary urgency is measured using PPIUS. For each micturition episode, participant will be asked to rate the degree of associated urgency severity according to PPIUS. PPIUS is a 5-point categorical scale ranging from 0 to 4, where 0 = no urgency (participant felt no need to empty the bladder, but did so for other reasons), 1 = mild urgency (participant could postpone voiding as long as necessary, without fear of wetting herself), 2 = moderate urgency (participant could postpone voiding for a short while, without fear of wetting herself), 3 = severe urgency (participant could not postpone voiding, had to rush to the toilet in order not to wet herself), and 4 = urge incontinence (participant leaked before arriving to the toilet). Mean number of Level 3 or 4 urgency episodes is the mean of recordings of Level 3 or 4 urgency episodes in 3-day electronic micturition diary in the week prior to visit.

    Measured: Baseline and Week 12

  4. Change From Baseline in Bladder Pain/ Interstitial Cystitis Symptom Score (BPIC-SS) Total Score at Week 12

    The BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over the previous 7 days. Question (Q) 1 to Q5 assess urinary symptoms (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) and are rated 0 (never) to 4 (always). Q6 and Q7 assess the impact of bladder pain (bothered by frequent urination during daytime and nighttime) and are rated 0 (not at all) to 4 (a great deal). Q8 assesses the worst pain on a 0 (no bladder pain) to 10 (worst possible bladder pain) NRS. The BPIC-SS total score is the sum of the individual question scores and range from 0 to 38, with higher scores indicating a worse situation. A score of 19 or more represents moderate/severe disease activity. A negative change indicates a reduction/improvement from baseline.

    Measured: Baseline and Week 12

  5. Change From Baseline in BPIC-SS Worst Bladder Pain (Question 8) Score at Week 12

    The BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over the previous 7 days. Q8 of BPIC-SS assesses the worst pain on a 0 (no bladder pain) to 10 (worst possible bladder pain) NRS. A negative change indicates a reduction/improvement from baseline.

    Measured: Baseline and Week 12

  6. Percentage of Participants With Moderately Improved or Better Grade on the Global Response Assessment (GRA) at Week 12

    A self-reported 7 grade GRA is 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". Percentage of participants with a successful GRA response (defined as the response of "moderately improved" or better ["markedly improved"] will be reported.

    Measured: Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Sep 2017
  2. Study start28 Sep 2017
  3. Primary completion21 Mar 2019
  4. Study completion21 Mar 2019

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
119 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03282318
Sponsor's protocol ID
6294-CL-0101
Other IDs
2016-004138-12, 2016
Acronym
SERENITY
Status last verified
Oct 2024
First submitted
12 Sep 2017
Last updated
31 Oct 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Publications

  • Imamura M, Scott NW, Wallace SA, Ogah JA, Ford AA, Dubos YA, Brazzelli M. Interventions for treating people with symptoms of bladder pain syndrome: a network meta-analysis. Cochrane Database Syst Rev. 2020 Jul 30;7(7):CD013325. doi: 10.1002/14651858.CD013325.pub2.

    PubMed 32734597

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ASP6294 in people with Bladder Pain Syndrome and Interstitial Cystitis, run by Astellas Pharma Europe B.V. at 28 sites in 10 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 20 inclusion and 41 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: finished, as of the sponsor's last update on 31 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in Russia, Germany, Latvia, Poland, Spain and 5 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Change from Baseline in Average Mean Daily Pain (MDP) Score at Week 12 (time frame: Baseline and Week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03282318, which also lists 1 linked publication.

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 31 Oct 2024.

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