Crofelemer oral solution for children with Microvillus Inclusion Disease

Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease (MVID)

This randomized, double-blind trial will test whether crofelemer oral solution taken three times daily can help children (3 months–17 years) with MVID who depend on parenteral support, comparing multiple doses to placebo with a within-dose crossover design.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 3 Months to 17 Years
• Sex: All
• Sponsor: Napo Pharmaceuticals, Inc. (Industry-sponsored)
• Locations: 3 sites (Boston, Massachusetts and 2 other locations)
• Trial ID: NCT06721871 on ClinicalTrials.gov

What this trial studies

This is a phase 2, randomized, double-blind, placebo-controlled, dose-escalation trial enrolling pediatric participants with genetically or histologically confirmed MVID who receive significant parenteral support. Blinded crofelemer powder for oral solution or matching placebo will be reconstituted and given orally or via G-/GJ-tube three times daily at three ascending dose levels, with a placebo crossover within each dose level. The primary focus is safety and tolerability over a 24–32 week period, with secondary analyses comparing changes from an 8-week pre-treatment baseline within participants and between groups as feasible. Efficacy signals will be explored by comparing fluid/feeding outcomes and parenteral support requirements between crofelemer and placebo periods.

Who should consider this trial

Good fit: Children aged 3 months to 17 years with a confirmed genetic or histologic diagnosis of MVID who receive at least 50% of weekly hydration via parenteral support and can take medication orally or via a G-/GJ-tube are ideal candidates.

Not a fit: Patients who do not have confirmed MVID, who receive less than 50% of weekly hydration via parenteral support, or who cannot ingest study medication orally or via an allowed tube type (e.g., only J-tube) are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, crofelemer could reduce intestinal fluid losses and lower reliance on parenteral support for children with MVID.

How similar studies have performed: Crofelemer has shown benefit for certain types of chronic diarrhea in adults, but its use in MVID is novel and largely untested in this specific pediatric population.

Eligibility criteria

Inclusion Criteria:

1. Participants (assent for participants older than 7 years of age) and/or their legal parent/guardian sign an Informed Consent Form (ICF) indicating that they understand the purpose of the procedures required for the study and are willing to participate
2. 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.
3. Male or female participants between the ages of 3 months to 17 years at the time of signing the informed consent or providing assent
4. Have a confirmed diagnosis (genetic and/or histologic) of MVID
5. 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)
6. Have, during the 8 weeks prior to baseline, a volume of PS that represents at least 50% (≥ 50%) of the participant's weekly hydration volume requirements
7. 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.
8. 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:

Within the last 4 weeks before study initiation, participants have:

1. had significant changes to PS requirements (i.e., ± \> 20%)
2. had a new requirement for diuretics
3. had any infection requiring IV antibiotic administration
4. had a documented active gastrointestinal infection
5. initiated any new anti-diarrheal drug
6. had an increase in ALT, AST, or total bilirubin that is ≥2 times the participant's usual laboratory values
7. previously received an organ transplant
8. any currently-diagnosed malignancy
9. is pregnant or breastfeeding
10. any investigator determined criteria for inability to participate in this study

Where this trial is running

Boston, Massachusetts and 2 other locations

• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
• UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4 — Rome, Italy (Not_yet_recruiting)
• Al Jalila Children's Hospital — Dubai, United Arab Emirates (Recruiting)

Study contacts

• Principal investigator: Lissette Jimenez, MD, MPH — Boston Children's Hospital
• Study coordinator: Maha Dakhloul, BSc.Pharmacy

Conditions Microvillus Inclusion Disease; Congenital Disorders; Rare Diseases