Short Bowel SyndromePhase 2NCT03582384
Use of Tocofersolan (Vedrop®) to Prevent or Treat Refractive Vitamin E Deficiency in Infants and Children
A Phase 2 study testing Tocofersolan in people with Short Bowel Syndrome and Vitamin E Deficiency, run by Boston Children's Hospital.
Trial summary
Patients with short bowel syndrome or other forms of intestinal failure/fat malabsorption are unable to tolerate adequate oral or enteral feedings. They require that nutrition be given as enteral nutrition that is delivered by feeding tube. Read more about this study
- Who is it for?
- Ages 30 days to 17 years
- Conditions the study is for
- Short Bowel Syndrome, Vitamin E Deficiency
- What is being tested?
- Tocofersolan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2020to Jan 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 30 days to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 1. Patients with intestinal failure or fat malabsorption who have refractory vitamin E deficiency (defined as vitamin E level < 5 mg/L) despite receiving vitamin E supplementation with currently available enteral vitamin E products for at least 6 months or requiring the provision of vitamin parenterally as part of a multivitamin infusion.
Who cannot join
- Pregnancy
- Use of warfarin or any related vitamin K antagonist.
- Enrollment in any other clinical trial involving an investigational agent (unless approved by the principal investigators of the other trial)
- The parent or guardian or child unwilling to provide consent or assent
About this study
Patients with short bowel syndrome or other forms of intestinal failure/fat malabsorption are unable to tolerate adequate oral or enteral feedings. They require that nutrition be given as enteral nutrition that is delivered by feeding tube. Often these children take supplements such as vitamins to help improve their nutritional status but, due to their condition, they have difficulty absorbing the supplement sufficiently and most of it is lost in the stool. The drug that will be studied, Tocofersolan (Vedrop®) is a form of vitamin E, a type of the fat soluble vitamin needed in the human diet. It has been formulated in such a way that it may be more easily absorbed by patients with this condition. The main purpose of the study is to learn about the safety and tolerability of this form of vitamin E. Before receiving the study drug, the severity of the child's vitamin E deficiency will be determined by a blood sample, followed by giving them a daily dose of tocofersolan (Vedrop®) either orally or through their feeding tube. After a 4 weeks of therapy, a second blood sample will be checked and the child will continue either same dose of tocoferssolan or it will be adjusted in response to the blood levels. If the study drug works as it is designed to do, there should be an increase in the concentration of the vitamin E in the child's blood, suggesting that the drug was absorbed. At each visit, a sample of blood will be obtained to assess the child's vitamin E status and general health.
Patients will remain on tocofersolan for approximately one year or as long as the study remains open. Based on the European pediatric experience, patients should be expected be on tocofersolan a minimum of 3 months, ideally 6 months to see optimal clinical response.
- Conditions
- Short Bowel Syndrome, Vitamin E Deficiency
- Being tested
- Tocofersolan
- Medical subject headings
- Short Bowel Syndrome, Vitamin E Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TocofersolanDrug
After baseline labs are obtained, therapy with tocofersolan will be initiated at an initial dose of 17 mg/kg of d-alpha-tocopherol in the form of tocofersolan (0.34 ml/kg/day) and titrated based on blood levels.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Not described Receives: Drug: Tocofersolan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Vitamin E Level
Primary outcomes measured will include whether the vitamin E level increased to > 5 mg/L
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2018
- Study startDec 2020 (estimated)
- Primary completionJan 2022 (estimated)
- Study completionJan 2023 (estimated)
Why it stopped: Study never started
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Short Bowel SyndromeStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Daniel Elman, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03582384
- Sponsor's protocol ID
- IRB-P00026926
- Status last verified
- May 2020
- First submitted
- 26 Jun 2018
- Last updated
- 13 May 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Westergren T, Kalikstad B. Dosage and formulation issues: oral vitamin E therapy in children. Eur J Clin Pharmacol. 2010 Feb;66(2):109-18. doi: 10.1007/s00228-009-0729-1. Epub 2009 Oct 13.
PubMed 19823814European Medicines Agency Evaluation of Medicines for Human Use, CHMP Assessment Report for Vedrop, London, 29 May 2009 Doc.Ref.: EMEA/485798/2009.
Sokol RJ. A New Old Treatment for Vitamin E Deficiency in Cholestasis. J Pediatr Gastroenterol Nutr. 2016 Dec;63(6):577-578. doi: 10.1097/MPG.0000000000001330. No abstract available.
PubMed 27429361Thebaut A, Nemeth A, Le Mouhaer J, Scheenstra R, Baumann U, Koot B, Gottrand F, Houwen R, Monard L, de Micheaux SL, Habes D, Jacquemin E. Oral Tocofersolan Corrects or Prevents Vitamin E Deficiency in Children With Chronic Cholestasis. J Pediatr Gastroenterol Nutr. 2016 Dec;63(6):610-615. doi: 10.1097/MPG.0000000000001331.
PubMed 27429423Ubesie AC, Kocoshis SA, Mezoff AG, Henderson CJ, Helmrath MA, Cole CR. Multiple micronutrient deficiencies among patients with intestinal failure during and after transition to enteral nutrition. J Pediatr. 2013 Dec;163(6):1692-6. doi: 10.1016/j.jpeds.2013.07.015. Epub 2013 Aug 24.
PubMed 23978355Zondlo Fiume M. Final report on the safety assessment of Tocopherol, Tocopheryl Acetate, Tocopheryl Linoleate, Tocopheryl Linoleate/Oleate, Tocopheryl Nicotinate, Tocopheryl Succinate, Dioleyl Tocopheryl Methylsilanol, Potassium Ascorbyl Tocopheryl Phosphate, and Tocophersolan. Int J Toxicol. 2002;21 Suppl 3:51-116. doi: 10.1080/10915810290169819. No abstract available.
PubMed 12537931Ash, M. (2004). Handbook of green chemicals (2nd ed.). Endicott, N.Y.: Synapse Information Resources.
Yang CF, Duro D, Zurakowski D, Lee M, Jaksic T, Duggan C. High prevalence of multiple micronutrient deficiencies in children with intestinal failure: a longitudinal study. J Pediatr. 2011 Jul;159(1):39-44.e1. doi: 10.1016/j.jpeds.2010.12.049. Epub 2011 Feb 16.
PubMed 21324480
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Tocofersolan (Vedrop®) to Prevent or Treat Refractive Vitamin E Deficiency in Infants and Children
- ClinicalTrials.gov ID
- NCT03582384
- Acronym / sponsor ID
- IRB-P00026926
- Conditions
- Short Bowel Syndrome, Vitamin E Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study never started
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Study start
- Dec 2020 (estimated)
- Primary completion
- Jan 2022 (estimated)
- Study completion
- Jan 2023 (estimated)
- First posted
- 11 Jul 2018 (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.
Patients with short bowel syndrome or other forms of intestinal failure/fat malabsorption are unable to tolerate adequate oral or enteral feedings. They require that nutrition be given as enteral nutrition that is delivered by feeding tube. Often these children take supplements such as vitamins to help improve their nutritional status but, due to their condition, they have difficulty absorbing the supplement sufficiently and most of it is lost in the stool. The drug that will be studied, Tocofersolan (Vedrop®) is a form of vitamin E, a type of the fat soluble vitamin needed in the human diet. It has been formulated in such a way that it may be more easily absorbed by patients with this condition. The main purpose of the study is to learn about the safety and tolerability of this form of vitamin E. Before receiving the study drug, the severity of the child's vitamin E deficiency will be determined by a blood sample, followed by giving them a daily dose of tocofersolan (Vedrop®) either orally or through their feeding tube. After a 4 weeks of therapy, a second blood sample will be checked and the child will continue either same dose of tocoferssolan or it will be adjusted in response to the blood levels. If the study drug works as it is designed to do, there should be an increase in the concentration of the vitamin E in the child's blood, suggesting that the drug was absorbed. At each visit, a sample of blood will be obtained to assess the child's vitamin E status and general health.
Patients will remain on tocofersolan for approximately one year or as long as the study remains open. Based on the European pediatric experience, patients should be expected be on tocofersolan a minimum of 3 months, ideally 6 months to see optimal clinical response.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: Tocofersolan |
Who may qualify
- Ages
- 30 days to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- 1. Patients with intestinal failure or fat malabsorption who have refractory vitamin E deficiency (defined as vitamin E level < 5 mg/L) despite receiving vitamin E supplementation with currently available enteral vitamin E products for at least 6 months or requiring the provision of vitamin parenterally as part of a multivitamin infusion.
Key exclusion criteria (4 listed)
- Pregnancy
- Use of warfarin or any related vitamin K antagonist.
- Enrollment in any other clinical trial involving an investigational agent (unless approved by the principal investigators of the other trial)
- The parent or guardian or child unwilling to provide consent or assent
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 |
|---|---|---|
| TocofersolanAfter baseline labs are obtained, therapy with tocofersolan will be initiated at an initial dose of 17 mg/kg of d-alpha-tocopherol in the form of tocofersolan (0.34 ml/kg/day) and titrated based on blood levels. | Drug | Treatment |
Primary outcome
Vitamin E Level
Time frame: 1 Year
Primary outcomes measured will include whether the vitamin E level increased to > 5 mg/L
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2020.
- Background: PubMed 19823814Westergren T, Kalikstad B. Dosage and formulation issues: oral vitamin E therapy in children. Eur J Clin Pharmacol. 2010 Feb;66(2):109-18. doi: 10.1007/s00228-009-0729-1. Epub 2009 Oct 13.
- BackgroundEuropean Medicines Agency Evaluation of Medicines for Human Use, CHMP Assessment Report for Vedrop, London, 29 May 2009 Doc.Ref.: EMEA/485798/2009.
- Background: PubMed 27429361Sokol RJ. A New Old Treatment for Vitamin E Deficiency in Cholestasis. J Pediatr Gastroenterol Nutr. 2016 Dec;63(6):577-578. doi: 10.1097/MPG.0000000000001330. No abstract available.
- Background: PubMed 27429423Thebaut A, Nemeth A, Le Mouhaer J, Scheenstra R, Baumann U, Koot B, Gottrand F, Houwen R, Monard L, de Micheaux SL, Habes D, Jacquemin E. Oral Tocofersolan Corrects or Prevents Vitamin E Deficiency in Children With Chronic Cholestasis. J Pediatr Gastroenterol Nutr. 2016 Dec;63(6):610-615. doi: 10.1097/MPG.0000000000001331.
- Background: PubMed 23978355Ubesie AC, Kocoshis SA, Mezoff AG, Henderson CJ, Helmrath MA, Cole CR. Multiple micronutrient deficiencies among patients with intestinal failure during and after transition to enteral nutrition. J Pediatr. 2013 Dec;163(6):1692-6. doi: 10.1016/j.jpeds.2013.07.015. Epub 2013 Aug 24.
- Background: PubMed 12537931Zondlo Fiume M. Final report on the safety assessment of Tocopherol, Tocopheryl Acetate, Tocopheryl Linoleate, Tocopheryl Linoleate/Oleate, Tocopheryl Nicotinate, Tocopheryl Succinate, Dioleyl Tocopheryl Methylsilanol, Potassium Ascorbyl Tocopheryl Phosphate, and Tocophersolan. Int J Toxicol. 2002;21 Suppl 3:51-116. doi: 10.1080/10915810290169819. No abstract available.
- BackgroundAsh, M. (2004). Handbook of green chemicals (2nd ed.). Endicott, N.Y.: Synapse Information Resources.
- Results publication: PubMed 21324480Yang CF, Duro D, Zurakowski D, Lee M, Jaksic T, Duggan C. High prevalence of multiple micronutrient deficiencies in children with intestinal failure: a longitudinal study. J Pediatr. 2011 Jul;159(1):39-44.e1. doi: 10.1016/j.jpeds.2010.12.049. Epub 2011 Feb 16.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tocofersolan in people with Short Bowel Syndrome and Vitamin E Deficiency, run by Boston Children's Hospital.
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 1 inclusion and 4 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: withdrawn, as of the sponsor's last update on 13 May 2020. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Vitamin E Level (time frame: 1 Year). 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 NCT03582384, which also lists 8 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 13 May 2020; AzTrials last refreshed its copy on 8 Nov 2026.