General Design and Study Schema. This is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of a 200-mcg dose of IPP-201101 plus SOC compared with placebo plus SOC in patients with active SLE. The study consists of a 2-week screening period (visit 1), a 48 week treatment period beginning with a baseline visit in which randomization will be completed and study drug treatment will start (visit 2 and visits 4 through 14), and a final assessment 4 weeks after the last dose of study drug (visit 16 [week 52]). NOTE: If needed during the screening period, up to 14 additional days are permitted to confirm eligibility (eg, BILAG-2004, SLEDAI-2K, and PhGA central system assessments, laboratory results ….). In addition to the typical assessments obtained at the screening visit, such as obtaining medical, psychiatric, and medication history (medication history to be obtained from start of screening) and performing a physical examination and tests to determine the patients’ health status and eligibility for participation in the study, procedures will include assessment of disease activity using the SLEDAI-2K and BILAG-2004 disease activity indices and the SFI, and disease marker testing (ie, at screening, anti-dsDNA Ab, C3, C4, and XXX only]). Eligibility will be confirmed by using a central system to validate the BILAG-2004, SLEDAI-2K, and PhGA assessments . Eligible patients will return to the study center at baseline and will be randomly assigned (stratified by region, SLEDAI-2K screening total score [6 to 9 or ≥10], and racial-ethnic group classification [black/Hispanic or others]) at baseline with an equal probability and in a blinded fashion to receive either IPP-201101 or placebo sc every 4 weeks for 48 weeks. Pretreatment assessments will be performed and the 1st dose of 200 mcg sc of IPP-201101 or placebo (sc) will be administered after all assessments have been completed. Patients will be monitored for systemic symptoms for at least 1 hour after the first 2 doses of study drug have been administered. For subsequent doses, patients will be monitored, at the discretion of the investigator, in the study center after study drug treatment. Patients will return to the study center 2 weeks after administration of the 1st dose of study drug for immunogenicity testing, and every 4 weeks for 48 weeks (weeks 4, 8, 12,16, 20, 24, 28, 32, 36, 40, 44, and 48) to receive study drug. Patients may continue on their usual treatment for SLE (ie, SOC) as long as the inclusi...
General Design and Study Schema. This is a phase III, multicentre, randomised, double blind, parallel group, placebo controlled study to assess the efficacy and safety of two doses of Dysport® (600 U and 800 U) in adult subjects with SCI or MS with UI due to NDO, who have not been adequately managed with oral medication and who routinely require CIC to manage their bladder function. The study will be conducted at approximately 90 study sites and 330 subjects are planned. This study has two treatment periods: • A double blind placebo controlled period, where subjects receive a single IMP treatment administration of either 600 U or 800 U Dysport® or placebo • A subsequent double blind active treatment period, where subjects can receive multiple active IMP retreatment administrations of either 600 U or 800 U Dysport®. This second period commences when subjects receive their first study retreatment. There is significant variability of local clinical practice in conducting urodynamic (filling cystometry) assessments. Due to the difficulties in standardising and performing study specific urodynamics in a global program, this study will include sites that are unable to alter their local site practice sufficiently to perform standardised urodynamics. The study therefore has two subsets: • Urodynamic subset (planned ≥200 subjects) o All subjects in this subset must have standardised urodynamics (as described in the Study Specific Urodynamic Manual) during Screening and at Week 6 following each study treatment o Screening standardised urodynamics must confirm the presence of NDO • Non-Urodynamic subset o All subjects in this subset must have documented urodynamic proven NDO in the 12 months prior to Screening. If not, then urodynamics must be performed per local practice during Screening to confirm the diagnosis of NDO o Urodynamics will not be performed in the post-screening period in this subset Eligibility will be assessed during Screening (up to 30 days per subject although can be extended in certain circumstances see Section 5.2.1) although can be extended in certain circumstances see Section 5.2.1). All subjects will complete study Baseline safety and efficacy evaluations (including bladder diary, urodynamics (if required) and patient-reported outcomes) during this period. Eligible subjects will be randomised to receive either 600 U Dysport®, 800 U Dysport®, or placebo during the initial IMP administration which will occur up to 14 days after screening period (can be extended where UTI preven...
General Design and Study Schema. This is a phase III, multicentre, randomised, double blind, parallel group, placebo controlled study to assess the efficacy and safety of two doses of Dysport® (600 U and 800 U) in adult subjects with SCI or MS with UI due to NDO, who have not been adequately managed with oral medication and who routinely require CIC to manage their bladder function. The study will be conducted at approximately 90 study sites and 330 subjects are planned. This study has two treatment periods: • A double-blind placebo controlled period, where subjects receive a single IMP treatment administration of either 600 U or 800 U Dysport® or placebo • A subsequent double blind active treatment period, where subjects can receive multiple active IMP retreatment administrations of either 600 U or 800 U Dysport®. This second period commences when subjects receive their first study retreatment. Eligibility will be assessed during Screening (up to 30 days per subject, although can be extended in certain circumstances see Section 5.2.1). All subjects will complete study Baseline safety and efficacy evaluations (including bladder diary, urodynamics and patient- reported outcomes) during this period. Eligible subjects will be randomised to receive either 600 U Dysport®, 800 U Dysport®, or placebo during the initial IMP administration which will occur up to 14 days after screening period (but can be extended where UTI prevents administration of IMP see Section 5.2.3. The IMP will be administered into the detrusor via cystoscopy divided into 30 injection points of 0.5 mL each, which will be evenly distributed throughout the detrusor (avoiding the trigone). See Section 6 for full details of the treatment procedure. Subjects will be followed-up by telephone at Week 1 and Week 4; and will attend clinic visits at Week 2, Week 6 (primary study timepoint), and Week 12. Thereafter telephone visits will be scheduled every 12 weeks until end of study (EOS), or until retreatment is required. Details of the procedures performed at each study visit are described in Section 0.
