Top

Published in:

Open Access 01-12-2024 | Cystic Fibrosis | Study protocol

Evaluation of the efficiency of nanomicellar formulation of fat-soluble vitamins in patients with cystic fibrosis: the study protocol for a randomized controlled trial

Authors: Mahsa Soleimanzadeh, Saeedeh Talebi, Mahmoud Reza Jaafari, Seyed Javad Sayedi, Maryam Emadzadeh, Hamid Reza Kianifar

Published in: Trials | Issue 1/2024

Abstract

Background

Cystic fibrosis is an inherited disease, which is caused by the CFTR protein defects due to mutations in the CFTR gene. Along with CFTR dysfunction, exocrine pancreatic insufficiency plays a key role in persistent fat malabsorption in CF patients; therefore, deficiency of fat-soluble vitamins (A, D, E, and K) is still a therapeutic challenge. Even with efficient pancreatic enzyme medication and CF-specific vitamins, many patients with CF have fat-soluble vitamins deficiency. The present study aims to evaluate the efficiency of nanomicelle formulation of fat-soluble vitamins in children with CF in order to achieve the appropriate serum levels of these vitamins.

Methods

This prospective, single-blind control trial will be conducted at the Akbar Children’s Hospital in Mashhad, Iran. Patients with CF will be enrolled based on the eligibility criteria. The control group will receive the standard formulation of fat-soluble vitamins similar to the routine CF treatment, and for the intervention group, the nanomicelle formulation of fat-soluble vitamins will be administered for 3 months. The primary outcome of this study is the measurement of serum levels of fat-soluble vitamins. The secondary outcomes are clinical assessment by the Shwachman-Kulczycki score, anthropometrics, and quality of life. Outcomes will be assessed before and after 3 months.

Discussion

Due to persistent fat-soluble vitamin deficiency in CF disease, the nanomicelle formulation could be proposed as a new delivery method of fat-soluble vitamins in the treatment of cystic fibrosis.

Trial registration

Iranian Registry of Clinical Trials IRCT20220415054541N1. Registered on July 23, 2022.

Keogh RH, Szczesniak R, Taylor-Robinson D, Bilton D. Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: a longitudinal study using UK patient registry data. J Cystic Fibrosis. 2018;17(2):218–27.CrossRef

Sommerburg O, Hämmerling S, Schneider SP, Okun J, Langhans CD, Leutz-Schmidt P, Wielpütz MO, Siems W, Gräber SY, Mall MA, Stahl M. CFTR modulator therapy with lumacaftor/ivacaftor alters plasma concentrations of lipid-soluble vitamins A and E in patients with cystic fibrosis. Antioxidants. 2021;10(3):483.

Nowak JK, Krzyżanowska-Jankowska P, Drzymała-Czyż S, Goździk-Spychalska J, Wojsyk-Banaszak I, Skorupa W, Sapiejka E, Miśkiewicz-Chotnicka A, Brylak J, Zielińska-Psuja B, Lisowska A. Fat-Soluble Vitamins in Standard vs. Liposomal Form Enriched with Vitamin K2 in Cystic Fibrosis: A Randomized Multi-Center Trial. J Clin Med. 2022;11(2):462.

Krzyżanowska P, Pogorzelski A, Skorupa W, Moczko J, Grebowiec P, Walkowiak J. Exogenous and endogenous determinants of vitamin K status in cystic fibrosis. Sci Rep. 2015;5(1):12000.CrossRefPubMedPubMedCentral

Papas K, Kalbfleisch J, Mohon R. Bioavailability of a novel, water-soluble vitamin E formulation in malabsorbing patients. Digest Dis Sci. 2007;52(2):347–52.CrossRefPubMed

Mobasheri M, Attar H, Rezayat Sorkhabadi SM, Khamesipour A, Jaafari MR. Solubilization behavior of polyene antibiotics in nanomicellar system: insights from molecular dynamics simulation of the amphotericin B and nystatin interactions with polysorbate 80. Molecules (Basel, Switzerland). 2015;21(1):E6.CrossRefPubMed

Shakeri A, Sahebkar A. Opinion paper: nanotechnology: a successful approach to improve oral bioavailability of phytochemicals. Recent Pat Drug Deliv Formul. 2016;10(1):4-6.

Goncalves A, Roi S, Nowicki M, Dhaussy A, Huertas A, Amiot MJ, et al. Fat-soluble vitamin intestinal absorption: absorption sites in the intestine and interactions for absorption. Food Chem. 2015;172:155–60.CrossRefPubMed

Ravisankar P, Reddy AA, Nagalakshmi B, Koushik OS, Kumar BV, Anvith PS. The comprehensive review on fat soluble vitamins. IOSR J Pharm. 2015;5(11):12-28.

10.

Durie PR. The pathophysiology of the pancreatic defect in cystic fibrosis. Acta Paediatrica Scandinavica Suppl. 1989;363:41–4.CrossRef

11.

Matel JL, Milla CE. Nutrition in cystic fibrosis. Semin Respir Crit Care Med. 2009;30(5):579–86.CrossRefPubMed

12.

Gaskin KJ, Durie PR, Lee L, Hill R, Forstner GG. Colipase and lipase secretion in childhood-onset pancreatic insufficiency. Delineation of patients with steatorrhea secondary to relative colipase deficiency. Gastroenterology. 1984;86(1):1–7.CrossRefPubMed

13.

Bertolaso C, Groleau V, Schall JI, Maqbool A, Mascarenhas M, Latham NE, et al. Fat-soluble vitamins in cystic fibrosis and pancreatic insufficiency: efficacy of a nutrition intervention. J Pediat Gastroenterol Nutr. 2014;58(4):443–8.CrossRef

14.

Dougherty KA, Schall JI, Stallings VA. Suboptimal vitamin K status despite supplementation in children and young adults with cystic fibrosis. Am J Clin Nutr. 2010;92(3):660–7.CrossRefPubMedPubMedCentral

15.

Graham-Maar RC, Schall JI, Stettler N, Zemel BS, Stallings VA. Elevated vitamin A intake and serum retinol in preadolescent children with cystic fibrosis. Am J Clin Nutr. 2006;84(1):174–82.CrossRefPubMed

16.

Huang SH, Schall JI, Zemel BS, Stallings VA. Vitamin E status in children with cystic fibrosis and pancreatic insufficiency. J Pediatr. 2006;148(4):556–9.CrossRefPubMed

17.

Maqbool A, Graham-Maar RC, Schall JI, Zemel BS, Stallings VA. Vitamin A intake and elevated serum retinol levels in children and young adults with cystic fibrosis. J Cystic Fibrosis. 2008;7(2):137–41.CrossRef

18.

Rovner AJ, Stallings VA, Schall JI, Leonard MB, Zemel BS. Vitamin D insufficiency in children, adolescents, and young adults with cystic fibrosis despite routine oral supplementation. Am J Clin Nutr. 2007;86(6):1694–9.CrossRefPubMed

19.

Wilcken B. An evaluation of screening for cystic fibrosis. Progress Clin Biol Res. 1987;254:201–15.

20.

Kalepu S, Manthina M, Padavala V. Oral lipid-based drug delivery systems–an overview. Acta Pharmaceutica Sinica B. 2013;3(6):361-72.

Title: Evaluation of the efficiency of nanomicellar formulation of fat-soluble vitamins in patients with cystic fibrosis: the study protocol for a randomized controlled trial
Authors: Mahsa Soleimanzadeh
Saeedeh Talebi
Mahmoud Reza Jaafari
Seyed Javad Sayedi
Maryam Emadzadeh
Hamid Reza Kianifar
Publication date: 01-12-2024
Publisher: BioMed Central
Keywords: Cystic Fibrosis
Cystic Fibrosis
Pancreatic Insufficiency
Published in: Trials / Issue 1/2024
Electronic ISSN: 1745-6215
DOI: https://doi.org/10.1186/s13063-023-07896-8

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Evaluation of the efficiency of nanomicellar formulation of fat-soluble vitamins in patients with cystic fibrosis: the study protocol for a randomized controlled trial

Abstract

Background

Methods

Discussion

Trial registration

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Methods

Discussion

Trial registration

Please log in to get access to this content

Other articles of this Issue 1/2024

Pneumococcal conjugate vaccination schedules in infants—acquisition, immunogenicity, and pneumococcal conjugate and yellow fever vaccine co-administration study: statistical analysis plan

Vitamin D as an intervention for improving quadriceps muscle strength in patients after anterior cruciate ligament reconstruction: study protocol for a randomized double-blinded, placebo-controlled clinical trial

The clinical effectiveness of a self-management intervention for patients with persistent depressive disorder and their partners/caregivers: results from a multicenter, pragmatic randomized controlled trial

Aspin: neurosurgical aspirin intervention prognostic study — perioperative continuation versus discontinuation of aspirin in lumbar spinal surgery, a randomized controlled, noninferiority trial

Impact evaluation of a maternal and child cash transfer intervention, integrated with nutrition, early childhood development, and agriculture messaging (MAZIKO-IE): a study protocol for a cluster-randomised controlled trial

Lifestyle intervention in children with obesity and nonalcoholic fatty liver disease (NAFLD): study protocol for a randomized controlled trial in Ningbo city (the SCIENT study)