CROFELEMER for Microvillus inclusion disease (MVID)

Inclusion criteria

• {"criterion_text":"-Participants (and/or their legal caregiver and/or participant assent for participants older than 7 years of age) sign an Informed Consent Form (ICF) indicating they understand the purpose of the procedures required for the study and are willing to participate."}
• {"criterion_text":"-When appropriate, pediatric participants, whose age, cognitive skills, reading abilities and maturity allow the understanding of the study protocol should provide written assent to participate."}
• {"criterion_text":"-Male or female participants between the ages of 3 months to 17 years at the time of signing the informed consent by their legal caregiver and/or participant assent for eligible participants"}
• {"criterion_text":"-Have a confirmed diagnosis (genetic and/or histologic) of MVID"}
• {"criterion_text":"-Are able to ingest reconstituted Crofelemer Powder for Oral Solution either orally (PO) or through a previously placed G-tube or GJ-Tube (not via J-Tube)"}
• {"criterion_text":"-Have required, during the 8 weeks prior to baseline, an average weekly volume of PS that represents ≥ 50% of the participant’s average weekly hydration volume requirements"}
• {"criterion_text":"-If female participants have reached menarche, the participant (and caregiver) agree that the participant will remain abstinent or use two accepted methods of birth control during the course of the treatment period and for an additional 30 days following the last dose of study drug."}
• {"criterion_text":"-Male participants (and caregiver) agree that the participant will remain abstinent or use contraception during the course of the treatment period and continue on for an additional 90 days following the last dose of study drug."}

Exclusion criteria

• {"criterion_text":"-1. Within the last 4 weeks before study day -2 (day of study initiation), participants have: a. had significant changes to PS requirements (i.e., ± > 20%)"}
• {"criterion_text":"-4. is pregnant or breastfeeding"}
• {"criterion_text":"-6.\t Known hypersensitivity to any of the components of study drug"}
• {"criterion_text":"-5. any investigator determined criteria for inability to participate in this study"}
• {"criterion_text":"-b. had a new requirement for diuretics"}
• {"criterion_text":"-c. had any infection requiring IV antibiotic administration"}
• {"criterion_text":"-d. had a documented active gastrointestinal infection"}
• {"criterion_text":"-e. initiated any new anti-diarrheal drug"}
• {"criterion_text":"-f. had an increase in ALT, AST, or total bilirubin that is ≥2 times the participant’s usual laboratory values within the past 3 months04. had a documented active gastrointestinal infection"}
• {"criterion_text":"-2. previously received an organ transplant"}
• {"criterion_text":"-3. any currently diagnosed malignancy"}

Primary endpoints

• {"endpoint_text":"-Assessment of AEs, SAEs, laboratory evaluations, physical exam (including height, weight, BMI, and assessment of hydration status), and urine output","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"-Assessment of change from baseline, compared to placebo, within each dose level and over the 24 weeks of treatment in the following endpoints: • Average daily loose/watery stool volume during 24-hour stool collections every 2 weeks.","definition_or_measurement_approach":"Average daily loose/watery stool volume measured during 24-hour stool collections every 2 weeks over 24 weeks; comparison to placebo and baseline within each dose level."}
• {"endpoint_text":"-• Average daily loose/watery stool frequency during 24-hour stool collections every 2 weeks","definition_or_measurement_approach":"Average daily loose/watery stool frequency measured during 24-hour stool collections every 2 weeks."}
• {"endpoint_text":"-• Average daily stool consistency using the 7point pediatric version of the Bristol Stool Form Scale during 24-hour stool collections every 2 weeks","definition_or_measurement_approach":"Stool consistency assessed using the 7-point pediatric Bristol Stool Form Scale during 24-hour stool collections every 2 weeks."}
• {"endpoint_text":"-• Daily stool electrolytes changes (Na+, K+, Cl-) at the end of dose level 3 (at 20 and 24 weeks)","definition_or_measurement_approach":"Daily stool electrolyte measurements (Na+, K+, Cl-) evaluated at end of dose level 3, specifically at weeks 20 and 24."}
• {"endpoint_text":"-• Average weekly acetate supplementation","definition_or_measurement_approach":"Average weekly acetate supplementation (measurement approach not further specified)."}
• {"endpoint_text":"-• Average weekly PS volume requirements","definition_or_measurement_approach":"Average weekly parenteral support (PS) volume requirements (measurement approach not further specified)."}
• {"endpoint_text":"-• Average weekly TPN volume requirements","definition_or_measurement_approach":"Average weekly total parenteral nutrition (TPN) volume requirements (measurement approach not further specified)."}
• {"endpoint_text":"-• Average weekly supplemental IV fluid volume requirements","definition_or_measurement_approach":"Average weekly supplemental IV fluid volume requirements (measurement approach not further specified)."}
• {"endpoint_text":"-• Average weekly supplemental electrolytes (Na+, K+, Cl-) in PS and, separately, in TPN and IV fluids","definition_or_measurement_approach":"Average weekly supplemental electrolytes (Na+, K+, Cl-) measured in PS and separately in TPN and IV fluids (measurement approach not further specified)."}

Italy

Earliest CTIS Part Ii Submission Date

16-05-2024

Latest Decision Or Authorization Date

23-05-2025

Processing Time Days

372

Number Of Sites

1

Number Of Participants

4

Primary sponsor

Full Name

Napo Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

CTI Clinical Trial and Consulting Services Europe GmbH

Responsibilities

sponsorDuties codes: [1,11,12,5,6,7,8]; regulatory and trial management responsibilities indicated

Third parties

• {"country":"United States","full_name":"Sannova Analytical LLC","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Patient Primary - MDgroup","duties_or_roles":"sponsorDuties codes: [15]; value: IP shipment to patients","organisation_type":"Health care"}
• {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"sponsorDuties codes: [1,11,12,5,6,7,8]","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Merative Healthcare Ireland Limited","duties_or_roles":"sponsorDuties codes: [7]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Manufacturing Packaging Farmaca (MPF) B.V.","duties_or_roles":"sponsorDuties codes: [14]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Safe Harbor Pharmacovigilance LLC","duties_or_roles":"sponsorDuties codes: [8]","organisation_type":"Pharmaceutical company"}