Vitamin D for Sickle-cell Respiratory Complications

NCT01443728 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: AAAE3244R01FD003894
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease. Lung complications are the leading causes of morbidity and of death in sickle cell disease. Infections and increased inflammation play important roles in the development of the lung problems in sickle cell disease. Emerging evidence shows that vitamin D helps the immune system to fight infection and to control inflammation and could potentially help prevent respiratory complications in patients with sickle cell disease. The investigators hypothesize that oral vitamin D3, 100,000 IU (2.5 mg), given once a month to a group of children and adolescents with sickle cell disease, will reduce the rate of respiratory events (infection, asthma exacerbation and acute chest syndrome) compared to the rate in a group given standard dose oral vitamin D3, 12,000 IU (0.3 mg) given once a month. Funding Source - U.S. Food \& Drug Administration, Office of Orphan Products Development

Conditions

Sickle Cell Disease; Vitamin D Deficiency; Acute Chest Syndrome; Asthma; Respiratory Infections

Keywords

sickle cell disease; acute chest syndrome; respiratory complications; vitamin D

Outcome Measures

Primary Outcomes (1)

• Mean Annual Rate of Respiratory Events

  Defined as respiratory infection, acute asthma exacerbation, and acute chest syndrome.

  Up to 2 years

Secondary Outcomes (13)

• Mean 25-Hydroxyvitamin D (25-OHD)

  2 years

• Forced Vital Capacity (FVC)

  Up to 2 years

• Forced Expiratory Volume (FEV) in 1 Second (FEV1)

  Up to 2 years

• FEV1/FVC Ratio

  Up to 2 years

• FEF 25-75

  Up to 2 years

Study Arms (2)

Vitamin D3 100,000 IU

EXPERIMENTAL

Oral vitamin D3, 100,000 IU \[2.5 mg\] given once a month

Drug: Experimental: Vitamin D3 100,000 IU

Vitamin D3 12,000 IU

ACTIVE COMPARATOR

Standard dose oral vitamin D3 12,000 IU \[0.3 mg\] given once a month

Drug: Active Comparator: Vitamin D3 12,000 IU

Interventions

Experimental: Vitamin D3 100,000 IU DRUG

Oral vitamin D3, 100,000 IU \[2.5 mg\] given once a month

Also known as: Cholecalciferol

Vitamin D3 100,000 IU

Active Comparator: Vitamin D3 12,000 IU DRUG

Standard dose oral vitamin D3 12,000 IU \[0.3 mg\] given once a month

Also known as: Cholecalciferol

Vitamin D3 12,000 IU

Eligibility Criteria

• Age: 3 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of sickle cell disease (HbSS, HbSC, HbS Beta-thalassemia)
• Age 3 to 20 years old

You may not qualify if:

• Patient (or parent or guardian) unwilling or unable to provide written informed consent (and assent, if applicable)
• Patient unable or unwilling to comply with requirements of the clinical trial
• Participation in other therapeutic clinical trial
• Current diagnosis of rickets
• History of hypercalcemia or diagnosis of any medical condition associated with hypercalcemia, including primary hyperparathyroidism, malignancy, sarcoidosis, tuberculosis, granulomatous disease, familial hypocalciuric hypercalcemia
• Current use of corticosteroids, excluding inhaled steroids
• Current use of anticonvulsants (phenytoin, phenobarbital, carbamazepine)
• Therapy with thiazide diuretics or lithium carbonate
• Known liver or renal disease
• Patients taking medications for pulmonary complications of sickle cell disease not on a stable dose of medications, as defined by a change in medications or doses within the three months prior to study entry
• Patients on chronic red blood cell transfusion therapy
• Absence of baseline record of respiratory events (respiratory infections, asthma exacerbations, episodes of acute chest syndrome) for the preceding year
• Pregnancy

Sponsors & Collaborators

• Gary M Brittenham, MD: lead

Study Sites (1)

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Related Publications (1)

• Lee MT, Kattan M, Fennoy I, Arpadi SM, Miller RL, Cremers S, McMahon DJ, Nieves JW, Brittenham GM. Randomized phase 2 trial of monthly vitamin D to prevent respiratory complications in children with sickle cell disease. Blood Adv. 2018 May 8;2(9):969-978. doi: 10.1182/bloodadvances.2017013979.

  PMID: 29712666 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell; Vitamin D Deficiency; Acute Chest Syndrome; Asthma; Respiratory Tract Infections

Interventions

Cholecalciferol

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Bronchial Diseases; Lung Diseases, Obstructive; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Infections

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Results Point of Contact

• Title: Gary Brittenham, MD
• Organization: Columbia University

gmb31@cumc.columbia.edu

212-305-7005

Study Officials

• Gary Brittenham, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

• Margaret T. Lee, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: James A. Wolff Professor of Pediatrics

Study Record Dates

• First Submitted: September 27, 2011
• First Posted: September 30, 2011
• Study Start: December 13, 2011
• Primary Completion: June 20, 2013
• Study Completion: February 15, 2015
• Last Updated: August 9, 2024
• Results First Posted: August 9, 2024

Record last verified: 2024-07

Locations

US (1)