NCT03734744

Brief Summary

Despite the extensive literature on adverse clinical outcomes associated with vitamin D deficiency, there are currently no proven treatment strategy that effectively achieves and maintains optimal serum vitamin D status in cystic fibrosis (CF) patients. For the treatment of vitamin D deficiency, CF Foundation currently recommends 2,000 IU daily. However, because achieving adequate serum 25(OH)D levels is a challenge in CF, higher doses of vitamin D may be necessary to reach and maintain vitamin D sufficiency. Poor oral bioavailability of ergocalciferol has been demonstrated in CF patients, which may potentially also be an issue with cholecalciferol. In order to optimize the treatment of vitamin D deficiency in CF, the kinetic disposition must be well understood. However, there are very few data currently available describing the kinetics of both vitamin D and 25-hydroxyvitamin D, and to the investigator's knowledge, no studies have yet characterized the pharmacokinetic disposition of vitamin D and its metabolites in cystic fibrosis. Addressing this issue is crucial in effectively and safely correcting vitamin D deficiency in CF.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jun 2019

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 6, 2018

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 8, 2018

Completed
7 months until next milestone

Study Start

First participant enrolled

June 17, 2019

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 16, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 16, 2022

Completed
Last Updated

May 17, 2022

Status Verified

May 1, 2022

Enrollment Period

2.9 years

First QC Date

November 6, 2018

Last Update Submit

May 16, 2022

Conditions

Vitamin D DeficiencyCystic Fibrosis

Keywords

PharmacokineticsPharmacodynamics

Outcome Measures

Primary Outcomes (3)

  • Peak plasma concentrations (Cmax)

    10 weeks

  • Time taken to reach the maximum concentration (Tmax)

    10 weeks

  • Area under the plasma concentration versus time curve (AUC)

    10 weeks

Secondary Outcomes (1)

  • Levels of serum inflammatory biomarkers

    10 weeks

Study Arms (2)

Adults with Cystic Fibrosis

EXPERIMENTAL

CF adults with vitamin D insufficiency or deficiency will receive 300,000-600,000 IU vitamin D3 (cholecalciferol)

Dietary Supplement: Vitamin D3

Non-CF Controls with Low vitamin D

EXPERIMENTAL

Non-CF controls with vitamin D insufficiency or deficiency will receive 300,000-600,000 IU vitamin D3 (cholecalciferol)

Dietary Supplement: Vitamin D3

Interventions

Vitamin D3DIETARY_SUPPLEMENT

Vitamin D is a fat-soluble vitamin that plays an important role in immune modulation in addition to its classical roles in regulation of calcium homeostasis and bone health

Also known as: Cholecalciferol
Adults with Cystic FibrosisNon-CF Controls with Low vitamin D

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • For CF, diagnosis of CF based on positive sweat chloride or known CF mutation
  • For CF, Patients with pancreatic insufficiency
  • Age ≥ 18 years
  • Serum 25(OH)D concentrations below 30 ng/mL (75 nmol/L)

You may not qualify if:

  • Pregnancy
  • History of lung transplant,
  • Severe anemia (hemoglobin concentration \< 7 g/dL),
  • Liver disease (AST/ALT \> 3x ULN), kidney disease (GFR ≤ 40 mL/min), or granulomatous conditions
  • Patients taking steroids, cholesterol-lowering drug (cholestyramine), weight-loss drugs (orlistat) , statins, anti-tuberculosis drugs (rifampin and isoniazid), phenobarbital, phenytoin, carbamazepine, immunosuppressants (cyclosporine, tacrolimus)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Keck Hospital of University of Southern California

Los Angeles, California, 90033, United States

Location

Related Publications (5)

  • Thacher TD, Clarke BL. Vitamin D insufficiency. Mayo Clin Proc. 2011 Jan;86(1):50-60. doi: 10.4065/mcp.2010.0567.

    PMID: 21193656BACKGROUND

  • Tangpricha V, Kelly A, Stephenson A, Maguiness K, Enders J, Robinson KA, Marshall BC, Borowitz D; Cystic Fibrosis Foundation Vitamin D Evidence-Based Review Committee. An update on the screening, diagnosis, management, and treatment of vitamin D deficiency in individuals with cystic fibrosis: evidence-based recommendations from the Cystic Fibrosis Foundation. J Clin Endocrinol Metab. 2012 Apr;97(4):1082-93. doi: 10.1210/jc.2011-3050. Epub 2012 Mar 7.

    PMID: 22399505BACKGROUND

  • Shepherd D, Belessis Y, Katz T, Morton J, Field P, Jaffe A. Single high-dose oral vitamin D3 (stoss) therapy--a solution to vitamin D deficiency in children with cystic fibrosis? J Cyst Fibros. 2013 Mar;12(2):177-82. doi: 10.1016/j.jcf.2012.08.007. Epub 2012 Sep 19.

    PMID: 22998937BACKGROUND

  • Grossmann RE, Zughaier SM, Kumari M, Seydafkan S, Lyles RH, Liu S, Sueblinvong V, Schechter MS, Stecenko AA, Ziegler TR, Tangpricha V. Pilot study of vitamin D supplementation in adults with cystic fibrosis pulmonary exacerbation: A randomized, controlled trial. Dermatoendocrinol. 2012 Apr 1;4(2):191-7. doi: 10.4161/derm.20332.

    PMID: 22928076BACKGROUND

  • Lark RK, Lester GE, Ontjes DA, Blackwood AD, Hollis BW, Hensler MM, Aris RM. Diminished and erratic absorption of ergocalciferol in adult cystic fibrosis patients. Am J Clin Nutr. 2001 Mar;73(3):602-6. doi: 10.1093/ajcn/73.3.602.

    PMID: 11237938BACKGROUND

MeSH Terms

Conditions

Vitamin D DeficiencyCystic Fibrosis

Interventions

Cholecalciferol

Condition Hierarchy (Ancestors)

AvitaminosisDeficiency DiseasesMalnutritionNutrition DisordersNutritional and Metabolic DiseasesPancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

CholestenesCholestanesSteroidsFused-Ring CompoundsPolycyclic CompoundsSterolsVitamin DSecosteroidsMembrane LipidsLipids

Study Officials

  • Paul M Beringer, Pharm.D.

    University of Southern California

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Clinical Pharmacy

Study Record Dates

First Submitted

November 6, 2018

First Posted

November 8, 2018

Study Start

June 17, 2019

Primary Completion

May 16, 2022

Study Completion

May 16, 2022

Last Updated

May 17, 2022

Record last verified: 2022-05

Data Sharing

IPD Sharing
Will not share

Locations

US(1)