Microvillus Inclusion DiseasePhase 2/3NCT06721871
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
A Phase 2/3 study testing Crofelemer Powder for Oral Solution in people with Microvillus Inclusion Disease and Congenital Disorders, run by Napo Pharmaceuticals, Inc. at 3 sites in 3 countries.
Trial summary
A study that will evaluate the safety, tolerability and preliminary efficacy of multiple ascending doses of crofelemer, compared to placebo, using a randomized cross-over design within each dose level, when administered to participants with MVID receiving parenteral support (PS, defined as TPN with or without… Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Microvillus Inclusion Disease, Congenital Disorders, Rare Diseases
- What is being tested?
- Crofelemer Powder for Oral Solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 1 May 2025to Dec 2026
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
- 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.
- Male or female participants from birth to < 18 years at the time of signing the informed consent or providing assent
- Have a confirmed diagnosis (genetic and/or histologic) of MVID
- 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)
- Have, during the 8 weeks prior to baseline, an average weekly volume of PS that represents at least 50% (≥ 50%) of the participant's weekly hydration volume requirements
- 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.
- 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.
Who cannot join
- Within the last 4 weeks before study initiation, participants have:
- had significant changes to PS requirements (i.e., ± > 20%)
- had a new requirement for diuretics
- had any infection requiring IV antibiotic administration
- had a documented active gastrointestinal infection
- initiated any new anti-diarrheal drug
Show all 12Show fewer
- had an increase in ALT, AST, or total bilirubin that is ≥2 times the participant's usual laboratory values
- previously received an organ transplant
- any currently-diagnosed malignancy
- is pregnant or breastfeeding
- any investigator determined criteria for inability to participate in this study
- Known hypersensitivity to any of the components of study drug
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
Italy1 site
UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4
Rome
About this study
Official title: 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)
A study that will evaluate the safety, tolerability and preliminary efficacy of multiple ascending doses of crofelemer, compared to placebo, using a randomized cross-over design within each dose level, when administered to participants with MVID receiving parenteral support (PS, defined as TPN with or without supplementary IV fluid requirements). Blinded study drug will be administered as a novel crofelemer formulation, Crofelemer Powder for Oral Solution, or a matching placebo powder formulation for oral solution. Assigned study drug will be reconstituted and administered orally (or enterally) three times daily (TID) as a concentrated liquid formulation in each of the three dose levels.
Read the detailed description
The study consists of an initial randomized double-blind placebo-controlled study, followed by an up to 48-week partially blinded extension phase.
The double-blind study phase is a randomized, double-blind, placebo-controlled, dose-escalating study with a placebo crossover design within each dose level in this ultra-rare MVID participant population. For the primary objective, safety and tolerability, comparisons between the crofelemer and placebo, across treatment periods within each dose level and through the end of the double-blind treatment period will be descriptively summarized. For secondary objectives, changes from the Baseline Period will be assessed.
After completion of the study and the No Treatment Period, if the Investigator and the DMC consider it appropriate for the participant's best interest based on safety and tolerability, the participant will be eligible to enter a Partially Blinded Extension Phase. Following approval by the DMC, the participant will enter the extension phase for up to 48 additional weeks of treatment with crofelemer at the dose selected by the DMC. The Investigator and the Sponsor will remain blinded to the selected dose.
- Being tested
- Crofelemer Powder for Oral Solution, Placebo Powder for Oral Solution
- Medical subject headings
- Microvillus inclusion disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Crofelemer Powder for Oral SolutionDrug
Crofelemer Powder for Oral Solution
Placebo Powder for Oral SolutionDrug
Matching Placebo Powder for Oral Solution
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1/Treatment Period 1: 1mg/kg/dose 3x/day | Experimental | Participants may be randomized to crofelemer powder for oral solution during Treatment Period 1 (1 month duration) Receives: Drug: Crofelemer Powder for Oral Solution |
| Dose Level 1/Treatment Period 1: Placebo 3x/day | Placebo comparator | Participants may be randomized to the placebo comparator during Treatment Period 1 (1 month duration) Receives: Drug: Placebo Powder for Oral Solution |
| Dose Level 1/Treatment Period 2: 1mg/kg/dose 3x/day | Experimental | Participants will crossover to either crofelemer powder for oral solution or placebo comparator Receives: Drug: Crofelemer Powder for Oral Solution |
| Dose Level 2/Treatment Period 1: 3mg/kg/dose 3x/day | Experimental | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration) Receives: Drug: Crofelemer Powder for Oral Solution |
| Dose Level 2/Treatment Period 1: Placebo 3x/day | Placebo comparator | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration) Receives: Drug: Placebo Powder for Oral Solution |
| Dose Level 2/Treatment Period 2: 3mg/kg/dose 3x/day | Experimental | Participants will crossover to either crofelemer powder for oral solution or placebo comparator Receives: Drug: Crofelemer Powder for Oral Solution |
| Dose Level 2/Treatment Period 2: Placebo 3x/day | Placebo comparator | Participants will crossover to either crofelemer powder for oral solution or placebo comparator Receives: Drug: Placebo Powder for Oral Solution |
| Dose Level 3/Treatment Period 1: 10mg/kg/dose 3x/day | Experimental | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 2 (1 month duration) Receives: Drug: Crofelemer Powder for Oral Solution |
| Dose Level 3/Treatment Period 1: Placebo 3x/day | Placebo comparator | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solutionor the placebo comparator that they were previously on during Dose Level 2 (1 month duration) Receives: Drug: Placebo Powder for Oral Solution |
| Dose Level 3/Treatment Period 2: 10mg/kg/dose 3x/day | Experimental | Participants will crossover to either crofelemer powder for oral solution or placebo comparator Receives: Drug: Crofelemer Powder for Oral Solution |
| Dose Level 3/Treatment Period 2: Placebo 3x/day | Placebo comparator | Participants will crossover to either crofelemer powder for oral solution or placebo comparator Receives: Drug: Placebo Powder for Oral Solution |
| Dose Level 1/Treatment Period 2: Placebo 3x/day | Placebo comparator | Participants will crossover to either crofelemer powder for oral solution or placebo comparator Receives: Drug: Placebo Powder for Oral Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Incidence of Adverse Events and Serious Adverse Events
Measured: 32 Weeks
Changes in Physical Examination
Changes from baseline in physical exam and signs of dehydration, such as decreased urine output, sunken eyes, lethargy and abnormal skin turgor.
Measured: 32 Weeks
Changes in Laboratory Values
Incidence in changes from baseline of individual lab values within a chemistry, hematology and metabolic panel analysis.
Measured: 32 Weeks
Secondary outcomes (7)
Average PS Volume Requirements Normalized to Body Weight
Record daily weekly TPN and IV fluid volume requirements in the Daily TPN and IV Fluid Diary, and divide the value per body weight for the corresponding study visit (mL/kg)
Measured: Average Weekly for 32 weeks
Average Loose/Watery Stool Volume
Measure and record the volume of loose/watery stools using a toilet hat for stool collection during 24 hours before each study visit
Measured: Average every 2 weeks for 32 weeks
Average TPN (including lipids) Volume Requirements
Record daily weekly TPN volume requirements in the Daily TPN and IV Fluid Diary
Measured: Average weekly for 32 weeks
Average Supplemental IV Fluids Volume Requirements
Record daily weekly supplemental IV fluid volume requirements in the Daily TPN and IV Fluid Diary
Measured: Average weekly for 32 weeks
Average Supplemental Electrolytes
Record daily any supplements of Na+, K+, Cl- in PS and, separately in TPN and in IV fluids, in the Daily PS Diary
Measured: Average weekly for 32 weeks
Average Acetate or Lactate Supplementation
Record daily any supplements of acetate or lactate in PS and, separately in TPN and in IV fluids, in the Daily PS Diary
Measured: Average weekly for 32 weeks
Stool Electrolytes
Stool electrolytes (Na+, K+, Cl-) concentration measured in mEq/L
Measured: Measurement at baseline, week 20 and week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2024
- Study start1 May 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (30)Papers the sponsor has linked to this study.
- Other trials for Microvillus Inclusion DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Each dose level will include 2 4-week Treatment Periods for a total of 8 weeks. There will be 3 ascending dose levels for a total of 24 weeks of treatment with either crofelemer or placebo, with safety reviews between dose levels. Within the 24 weeks of treatment, each of crofelemer and placebo will have been administered at 3 ascending dose levels for 4 weeks each, totaling 12 weeks. After completion of the study and the No Treatment Period, eligible participants will be able to receive crofelemer at a blinded dose selected by the DMC for up to 48 additional weeks of treatment.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Napo Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lissette Jimenez, MD, MPH, Principal investigator, Boston Children's HospitalPravin Chaturvedi, PhD, Study chair, Napo Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06721871
- Sponsor's protocol ID
- NP303-104
- Status last verified
- Jun 2026
- First submitted
- 20 Nov 2024
- Last updated
- 9 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Senn S. The analysis of continuous data from n-of-1 trials using paired cycles: a simple tutorial. Trials. 2024 Feb 16;25(1):128. doi: 10.1186/s13063-024-07964-7.
PubMed 38365817Mirza RD, Punja S, Vohra S, Guyatt G. The history and development of N-of-1 trials. J R Soc Med. 2017 Aug;110(8):330-340. doi: 10.1177/0141076817721131. No abstract available.
PubMed 28776473Guyatt GH, Haynes RB, Jaeschke RZ, Cook DJ, Green L, Naylor CD, Wilson MC, Richardson WS. Users' Guides to the Medical Literature: XXV. Evidence-based medicine: principles for applying the Users' Guides to patient care. Evidence-Based Medicine Working Group. JAMA. 2000 Sep 13;284(10):1290-6. doi: 10.1001/jama.284.10.1290.
PubMed 10979117Cornu C, Kassai B, Fisch R, Chiron C, Alberti C, Guerrini R, Rosati A, Pons G, Tiddens H, Chabaud S, Caudri D, Ballot C, Kurbatova P, Castellan AC, Bajard A, Nony P; CRESim & Epi-CRESim Project Groups; Aarons L, Bajard A, Ballot C, Bertrand Y, Bretz F, Caudri D, Castellan C, Chabaud S, Cornu C, Dufour F, Dunger-Baldauf C, Dupont JM, Fisch R, Guerrini R, Jullien V, Kassai B, Nony P, Ogungbenro K, Perol D, Pons G, Tiddens H, Rosati A, Alberti C, Chiron C, Kurbatova P, Nabbout R. Experimental designs for small randomised clinical trials: an algorithm for choice. Orphanet J Rare Dis. 2013 Mar 25;8:48. doi: 10.1186/1750-1172-8-48.
PubMed 23531234Babcock SJ, Flores-Marin D, Thiagarajah JR. The genetics of monogenic intestinal epithelial disorders. Hum Genet. 2023 May;142(5):613-654. doi: 10.1007/s00439-022-02501-5. Epub 2022 Nov 23.
PubMed 36422736Canani RB, Castaldo G, Bacchetta R, Martin MG, Goulet O. Congenital diarrhoeal disorders: advances in this evolving web of inherited enteropathies. Nat Rev Gastroenterol Hepatol. 2015 May;12(5):293-302. doi: 10.1038/nrgastro.2015.44. Epub 2015 Mar 17.
PubMed 25782092Berni Canani R, Terrin G, Cardillo G, Tomaiuolo R, Castaldo G. Congenital diarrheal disorders: improved understanding of gene defects is leading to advances in intestinal physiology and clinical management. J Pediatr Gastroenterol Nutr. 2010 Apr;50(4):360-6. doi: 10.1097/MPG.0b013e3181d135ef.
PubMed 20216094Bowman DM, Kaji I, Goldenring JR. Altered MYO5B Function Underlies Microvillus Inclusion Disease: Opportunities for Intervention at a Cellular Level. Cell Mol Gastroenterol Hepatol. 2022;14(3):553-565. doi: 10.1016/j.jcmgh.2022.04.015. Epub 2022 Jun 1.
PubMed 35660026Gao JJ, Tan M, Pohlmann PR, Swain SM. HALT-D: A Phase II Evaluation of Crofelemer for the Prevention and Prophylaxis of Diarrhea in Patients With Breast Cancer on Pertuzumab-Based Regimens. Clin Breast Cancer. 2017 Feb;17(1):76-78. doi: 10.1016/j.clbc.2016.08.005. Epub 2016 Aug 27.
PubMed 27692565Greene C, Barlesi B, Tarroza-David S, Friedlander T. Improved Control of Tyrosine Kinase Inhibitor-Induced Diarrhea with a Novel Chloride Channel Modulator: A Case Report. Oncol Ther. 2021 Jun;9(1):247-253. doi: 10.1007/s40487-021-00147-3. Epub 2021 Apr 7.
PubMed 33826111Groisman GM, Amar M, Livne E. CD10: a valuable tool for the light microscopic diagnosis of microvillous inclusion disease (familial microvillous atrophy). Am J Surg Pathol. 2002 Jul;26(7):902-7. doi: 10.1097/00000478-200207000-00008.
PubMed 12131157Hasosah M, Lemberg DA, Skarsgard E, Schreiber R. Congenital short bowel syndrome: a case report and review of the literature. Can J Gastroenterol. 2008 Jan;22(1):71-4. doi: 10.1155/2008/590143.
PubMed 18209785Knowles BC, Roland JT, Krishnan M, Tyska MJ, Lapierre LA, Dickman PS, Goldenring JR, Shub MD. Myosin Vb uncoupling from RAB8A and RAB11A elicits microvillus inclusion disease. J Clin Invest. 2014 Jul;124(7):2947-62. doi: 10.1172/JCI71651. Epub 2014 Jun 2.
PubMed 24892806Chowdhury AI, Phillips JF. Predicting contraceptive use in Bangladesh: a logistic regression analysis. J Biosoc Sci. 1989 Apr;21(2):161-8. doi: 10.1017/s0021932000017855.
PubMed 2722912Leng C, Rings EHHM, de Wildt SN, van IJzendoorn SCD. Pharmacological and Parenteral Nutrition-Based Interventions in Microvillus Inclusion Disease. J Clin Med. 2020 Dec 23;10(1):22. doi: 10.3390/jcm10010022.
PubMed 33374831
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06721871
- Acronym / sponsor ID
- NP303-104
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Napo Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 May 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 6 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study that will evaluate the safety, tolerability and preliminary efficacy of multiple ascending doses of crofelemer, compared to placebo, using a randomized cross-over design within each dose level, when administered to participants with MVID receiving parenteral support (PS, defined as TPN with or without supplementary IV fluid requirements). Blinded study drug will be administered as a novel crofelemer formulation, Crofelemer Powder for Oral Solution, or a matching placebo powder formulation for oral solution. Assigned study drug will be reconstituted and administered orally (or enterally) three times daily (TID) as a concentrated liquid formulation in each of the three dose levels.
Detailed description
The study consists of an initial randomized double-blind placebo-controlled study, followed by an up to 48-week partially blinded extension phase.
The double-blind study phase is a randomized, double-blind, placebo-controlled, dose-escalating study with a placebo crossover design within each dose level in this ultra-rare MVID participant population. For the primary objective, safety and tolerability, comparisons between the crofelemer and placebo, across treatment periods within each dose level and through the end of the double-blind treatment period will be descriptively summarized. For secondary objectives, changes from the Baseline Period will be assessed.
After completion of the study and the No Treatment Period, if the Investigator and the DMC consider it appropriate for the participant's best interest based on safety and tolerability, the participant will be eligible to enter a Partially Blinded Extension Phase. Following approval by the DMC, the participant will enter the extension phase for up to 48 additional weeks of treatment with crofelemer at the dose selected by the DMC. The Investigator and the Sponsor will remain blinded to the selected dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 12
- Model description
- Each dose level will include 2 4-week Treatment Periods for a total of 8 weeks. There will be 3 ascending dose levels for a total of 24 weeks of treatment with either crofelemer or placebo, with safety reviews between dose levels. Within the 24 weeks of treatment, each of crofelemer and placebo will have been administered at 3 ascending dose levels for 4 weeks each, totaling 12 weeks. After completion of the study and the No Treatment Period, eligible participants will be able to receive crofelemer at a blinded dose selected by the DMC for up to 48 additional weeks of treatment.
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1/Treatment Period 1: 1mg/kg/dose 3x/day | Experimental | Drug: Crofelemer Powder for Oral SolutionParticipants may be randomized to crofelemer powder for oral solution during Treatment Period 1 (1 month duration) |
| Dose Level 1/Treatment Period 1: Placebo 3x/day | Placebo comparator | Drug: Placebo Powder for Oral SolutionParticipants may be randomized to the placebo comparator during Treatment Period 1 (1 month duration) |
| Dose Level 1/Treatment Period 2: 1mg/kg/dose 3x/day | Experimental | Drug: Crofelemer Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 2/Treatment Period 1: 3mg/kg/dose 3x/day | Experimental | Drug: Crofelemer Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration) |
| Dose Level 2/Treatment Period 1: Placebo 3x/day | Placebo comparator | Drug: Placebo Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration) |
| Dose Level 2/Treatment Period 2: 3mg/kg/dose 3x/day | Experimental | Drug: Crofelemer Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 2/Treatment Period 2: Placebo 3x/day | Placebo comparator | Drug: Placebo Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 3/Treatment Period 1: 10mg/kg/dose 3x/day | Experimental | Drug: Crofelemer Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 2 (1 month duration) |
| Dose Level 3/Treatment Period 1: Placebo 3x/day | Placebo comparator | Drug: Placebo Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solutionor the placebo comparator that they were previously on during Dose Level 2 (1 month duration) |
| Dose Level 3/Treatment Period 2: 10mg/kg/dose 3x/day | Experimental | Drug: Crofelemer Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 3/Treatment Period 2: Placebo 3x/day | Placebo comparator | Drug: Placebo Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 1/Treatment Period 2: Placebo 3x/day | Placebo comparator | Drug: Placebo Powder for Oral SolutionParticipants will crossover to either crofelemer powder for oral solution or placebo comparator |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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
- 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.
- Male or female participants from birth to < 18 years at the time of signing the informed consent or providing assent
- Have a confirmed diagnosis (genetic and/or histologic) of MVID
- 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)
Key exclusion criteria (12 listed)
- Within the last 4 weeks before study initiation, participants have:
- previously received an organ transplant
- any currently-diagnosed malignancy
- is pregnant or breastfeeding
- any investigator determined criteria for inability to participate in this study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Crofelemer Powder for Oral SolutionCrofelemer Powder for Oral Solution | Drug | Dose Level 1/Treatment Period 1: 1mg/kg/dose 3x/day, Dose Level 1/Treatment Period 2: 1mg/kg/dose 3x/day, Dose Level 2/Treatment Period 1: 3mg/kg/dose 3x/day, Dose Level 2/Treatment Period 2: 3mg/kg/dose 3x/day, Dose Level 3/Treatment Period 1: 10mg/kg/dose 3x/day, Dose Level 3/Treatment Period 2: 10mg/kg/dose 3x/day |
| Placebo Powder for Oral SolutionMatching Placebo Powder for Oral Solution | Drug | Dose Level 1/Treatment Period 1: Placebo 3x/day, Dose Level 1/Treatment Period 2: Placebo 3x/day, Dose Level 2/Treatment Period 1: Placebo 3x/day, Dose Level 2/Treatment Period 2: Placebo 3x/day, Dose Level 3/Treatment Period 1: Placebo 3x/day, Dose Level 3/Treatment Period 2: Placebo 3x/day |
Primary outcome
Safety and Tolerability
Time frame: 32 Weeks
Incidence of Adverse Events and Serious Adverse Events
Changes in Physical Examination
Time frame: 32 Weeks
Changes from baseline in physical exam and signs of dehydration, such as decreased urine output, sunken eyes, lethargy and abnormal skin turgor.
Changes in Laboratory Values
Time frame: 32 Weeks
Incidence in changes from baseline of individual lab values within a chemistry, hematology and metabolic panel analysis.
7 secondary outcomes
Average PS Volume Requirements Normalized to Body Weight
Time frame: Average Weekly for 32 weeks
Average Loose/Watery Stool Volume
Time frame: Average every 2 weeks for 32 weeks
Average TPN (including lipids) Volume Requirements
Time frame: Average weekly for 32 weeks
Average Supplemental IV Fluids Volume Requirements
Time frame: Average weekly for 32 weeks
Average Supplemental Electrolytes
Time frame: Average weekly for 32 weeks
Average Acetate or Lactate Supplementation
Time frame: Average weekly for 32 weeks
Stool Electrolytes
Time frame: Measurement at baseline, week 20 and week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2026.
- Background: PubMed 38365817Senn S. The analysis of continuous data from n-of-1 trials using paired cycles: a simple tutorial. Trials. 2024 Feb 16;25(1):128. doi: 10.1186/s13063-024-07964-7.
- Background: PubMed 28776473Mirza RD, Punja S, Vohra S, Guyatt G. The history and development of N-of-1 trials. J R Soc Med. 2017 Aug;110(8):330-340. doi: 10.1177/0141076817721131. No abstract available.
- Background: PubMed 10979117Guyatt GH, Haynes RB, Jaeschke RZ, Cook DJ, Green L, Naylor CD, Wilson MC, Richardson WS. Users' Guides to the Medical Literature: XXV. Evidence-based medicine: principles for applying the Users' Guides to patient care. Evidence-Based Medicine Working Group. JAMA. 2000 Sep 13;284(10):1290-6. doi: 10.1001/jama.284.10.1290.
- Background: PubMed 23531234Cornu C, Kassai B, Fisch R, Chiron C, Alberti C, Guerrini R, Rosati A, Pons G, Tiddens H, Chabaud S, Caudri D, Ballot C, Kurbatova P, Castellan AC, Bajard A, Nony P; CRESim & Epi-CRESim Project Groups; Aarons L, Bajard A, Ballot C, Bertrand Y, Bretz F, Caudri D, Castellan C, Chabaud S, Cornu C, Dufour F, Dunger-Baldauf C, Dupont JM, Fisch R, Guerrini R, Jullien V, Kassai B, Nony P, Ogungbenro K, Perol D, Pons G, Tiddens H, Rosati A, Alberti C, Chiron C, Kurbatova P, Nabbout R. Experimental designs for small randomised clinical trials: an algorithm for choice. Orphanet J Rare Dis. 2013 Mar 25;8:48. doi: 10.1186/1750-1172-8-48.
- Background: PubMed 36422736Babcock SJ, Flores-Marin D, Thiagarajah JR. The genetics of monogenic intestinal epithelial disorders. Hum Genet. 2023 May;142(5):613-654. doi: 10.1007/s00439-022-02501-5. Epub 2022 Nov 23.
- Background: PubMed 25782092Canani RB, Castaldo G, Bacchetta R, Martin MG, Goulet O. Congenital diarrhoeal disorders: advances in this evolving web of inherited enteropathies. Nat Rev Gastroenterol Hepatol. 2015 May;12(5):293-302. doi: 10.1038/nrgastro.2015.44. Epub 2015 Mar 17.
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- Background: PubMed 35660026Bowman DM, Kaji I, Goldenring JR. Altered MYO5B Function Underlies Microvillus Inclusion Disease: Opportunities for Intervention at a Cellular Level. Cell Mol Gastroenterol Hepatol. 2022;14(3):553-565. doi: 10.1016/j.jcmgh.2022.04.015. Epub 2022 Jun 1.
- Background: PubMed 27692565Gao JJ, Tan M, Pohlmann PR, Swain SM. HALT-D: A Phase II Evaluation of Crofelemer for the Prevention and Prophylaxis of Diarrhea in Patients With Breast Cancer on Pertuzumab-Based Regimens. Clin Breast Cancer. 2017 Feb;17(1):76-78. doi: 10.1016/j.clbc.2016.08.005. Epub 2016 Aug 27.
- Background: PubMed 33826111Greene C, Barlesi B, Tarroza-David S, Friedlander T. Improved Control of Tyrosine Kinase Inhibitor-Induced Diarrhea with a Novel Chloride Channel Modulator: A Case Report. Oncol Ther. 2021 Jun;9(1):247-253. doi: 10.1007/s40487-021-00147-3. Epub 2021 Apr 7.
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- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lissette Jimenez, MD, MPH principal investigator
Boston Children's Hospital
Pravin Chaturvedi, PhD study chair
Napo Pharmaceuticals, Inc.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Crofelemer Powder for Oral Solution in people with Microvillus Inclusion Disease and Congenital Disorders, run by Napo Pharmaceuticals, Inc. at 3 sites in 3 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 8 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 9 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Italy, United Arab Emirates. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Safety and Tolerability (time frame: 32 Weeks). The study lists 3 primary outcomes in all. 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 NCT06721871, which also lists 30 linked publications.
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 9 Jun 2026.