The genetics of monogenic intestinal epithelial disorders.
Stephen J Babcock, David Flores-Marin, Jay R Thiagarajah
https://pubmed.ncbi.nlm.nih.gov/36422736Actively Recruiting
Phase 2
Age: 3Months - 17Years
All Genders
ID06721871
Safety, Tolerability, and Efficacy of Crofelemer Powder for Oral Solution in Children With Microvillus Inclusion Disease (MVID) Using Multiple Ascending Doses
Led by Napo Pharmaceuticals, Inc. · Updated on 2025-06-12
12
Participants Needed
3
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety, tolerability, and preliminary effects of different doses of crofelemer in children with Microvillus Inclusion Disease (MVID) who require parenteral support. This phase 2 trial uses a randomized, double-blind, placebo-controlled, crossover design to compare crofelemer powder for oral solution to a placebo within each dose level over a 32-week period. The study aims to understand how crofelemer affects stool volume, frequency, consistency, and electrolyte levels compared to placebo. Participants will receive crofelemer or placebo reconstituted as a liquid and taken orally or through a feeding tube three times daily. The study includes three dose levels: 1 mg/kg, 3 mg/kg, and 10 mg/kg, each given for one month with crossover to the other treatment within the dose level. This dose-escalation design allows participants to experience both crofelemer and placebo across treatment periods while monitoring safety and response. During the study, participants will be closely monitored for safety through physical exams and lab tests. Researchers will track stool characteristics, parenteral support volume, and electrolyte supplementation weekly. The total study duration is 32 weeks, including an 8-week baseline period and treatment periods with crossover. The trial evaluates multiple outcomes to assess crofelemer's effects on this rare pediatric condition under medical supervision.
CONDITIONS
Brief Title
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Who Can Participate
Age: 3Months - 17Years
All Genders
Eligibility Criteria
You may qualify if you...
- Participants or their legal guardians provide informed consent and, when appropriate, participants provide assent
- Participants aged between 3 months and 17 years at time of consent or assent
- Confirmed diagnosis of Microvillus Inclusion Disease (MVID) by genetic or histologic testing
- Able to take Crofelemer Powder for Oral Solution orally or via G-tube or GJ-tube (not J-tube)
- Receiving parenteral support covering at least 50% of weekly hydration needs for 8 weeks prior to baseline
- Female participants post-menarche must agree to remain abstinent or use two methods of birth control during treatment and 30 days after
- Male participants must agree to remain abstinent or use contraception during treatment and 90 days after
You will not qualify if you...
- Significant changes in parenteral support requirements (>20%) within 4 weeks before study start
- New need for diuretics within 4 weeks before study start
- Any infection requiring IV antibiotics within 4 weeks before study start
- Active gastrointestinal infection documented within 4 weeks before study start
- Started any new anti-diarrheal drug within 4 weeks before study start
- Liver enzyme or bilirubin levels twice above usual values within 4 weeks before study start
- History of organ transplant
- Any current malignancy
- Pregnant or breastfeeding
- Any other investigator-determined reason preventing participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Baseline Period
Duration - 8 weeks
Participants undergo an 8-week pre-treatment baseline period to establish baseline measurements before starting treatment.
Visits as scheduled to monitor baseline measurements
Treatment
Duration - Up to 24 weeks
Participants receive crofelemer powder for oral solution or placebo in a randomized, double-blind, placebo-controlled crossover design across multiple dose levels.
Multiple visits during each treatment period across three dose levels, each lasting about 1 month, with crossover between crofelemer and placebo
Follow-up
Duration - Up to 8 weeks after treatment
Participants are monitored for safety and tolerability after completing treatment.
Visits scheduled as needed for safety monitoring
Trial Site Locations
Total: 3 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
2
UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4
Rome, Italy
Not Yet Recruiting
3
Al Jalila Children's Hospital
Dubai, United Arab Emirates
Actively Recruiting
Loading map...
Research Team
M
Maha Dakhloul, BSc.Pharmacy
S
Sara Papetti, MA
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
12
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Congenital diarrhoeal disorders: advances in this evolving web of inherited enteropathies.
Roberto Berni Canani, Giuseppe Castaldo, Rosa Bacchetta...
https://pubmed.ncbi.nlm.nih.gov/25782092Congenital diarrheal disorders: improved understanding of gene defects is leading to advances in intestinal physiology and clinical management.
Roberto Berni Canani, Gianluca Terrin, Giuseppe Cardillo...
https://pubmed.ncbi.nlm.nih.gov/20216094Altered MYO5B Function Underlies Microvillus Inclusion Disease: Opportunities for Intervention at a Cellular Level.
Deanna M Bowman, Izumi Kaji, James R Goldenring
https://pubmed.ncbi.nlm.nih.gov/35660026HALT-D: A Phase II Evaluation of Crofelemer for the Prevention and Prophylaxis of Diarrhea in Patients With Breast Cancer on Pertuzumab-Based Regimens.
Jennifer J Gao, Ming Tan, Paula R Pohlmann...
https://pubmed.ncbi.nlm.nih.gov/27692565Improved Control of Tyrosine Kinase Inhibitor-Induced Diarrhea with a Novel Chloride Channel Modulator: A Case Report.
Claire Greene, Brigid Barlesi, Sigrid Tarroza-David...
https://pubmed.ncbi.nlm.nih.gov/33826111CD10: a valuable tool for the light microscopic diagnosis of microvillous inclusion disease (familial microvillous atrophy).
Gabriel M Groisman, Mary Amar, Erella Livne
https://pubmed.ncbi.nlm.nih.gov/12131157Congenital short bowel syndrome: a case report and review of the literature.
Mohammed Hasosah, Daniel A Lemberg, Eric Skarsgard...
https://pubmed.ncbi.nlm.nih.gov/18209785Myosin Vb uncoupling from RAB8A and RAB11A elicits microvillus inclusion disease.
Byron C Knowles, Joseph T Roland, Moorthy Krishnan...
https://pubmed.ncbi.nlm.nih.gov/24892806Predicting contraceptive use in Bangladesh: a logistic regression analysis.
A I Chowdhury, J F Phillips
https://pubmed.ncbi.nlm.nih.gov/2722912