Statins To Treat Adult Cystic Fibrosis

NCT01092572 | Withdrawn Phase 1

• 1 other identifier: CF-Man-Statin-1
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Cystic fibrosis (CF) is a lethal genetic condition that affects 30,000 children and adults in the United States. Although CF management has improved substantially over the past two decades, there is still no cure and most patients with CF die before reaching their 50th birthday, largely due to lung failure. There is growing evidence that excess lung and blood inflammation that occurs in response to infections in the lungs cause CF patients to be sicker. Simvastatin is a drug that is used to lower cholesterol, but many researchers have found that this drug may also treat blood and lung inflammation. In this study, we will determine whether or not simvastatin can treat blood and lung inflammation in patients with CF and most importantly determine whether or not it can make these patients feel better and have better lung function.

Conditions

Cystic Fibrosis; Systemic Inflammation

Keywords

cystic fibrosis; adult cystic fibrosis

Outcome Measures

Primary Outcomes (1)

• C-reactive protein

  The difference in the change in plasma C-reactive protein concentrations from baseline to 12 weeks of treatment between those randomized to simvastatin 40 mg/d and those randomized to placebo

  12 weeks

Secondary Outcomes (3)

• Changes in forced expiratory volume in one second (FEV1)

  12 weeks

• Changes in exacerbation rates

  12 weeks

• Changes in blood pro-inflammatory markers such as IL-6, TNF, IL-1beta, LPS, LBP, sCD14, EndoCAB, SP-D, CCL-18)

  12 weeks

Study Arms (2)

Simvastatin 40 mg/d

EXPERIMENTAL

simvastatin 40 mg per day taken orally

Drug: Simvastatin

Sugar pill

PLACEBO COMPARATOR

Drug: placebo

Interventions

Simvastatin DRUG

simvastatin 40 mg per day orally for 12 weeks.

Simvastatin 40 mg/d

placebo DRUG

placebo 1 tablet once daily for 12 weeks

Sugar pill

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients of provincial legal age of majority in British Columbia (≥19 years of age);
• Confirmed diagnosis of CF based on the following criteria:
• One or more clinical features consistent with the CF phenotype
• A genotype with identifiable classes I or II CFTR mutations
• Ability to provide informed consent.
• Clinically stable at enrollment as assessed by the treating physician.
• Ability to comply with medication use, study visits and study procedures, such as spirometry, and venipunctures.

You may not qualify if:

• Allergy or clinical reaction to simvastatin.
• The following abnormal lab values within the last six months or at screening:
• AST/ALT \> 1.5 ULN, CK \> 1.5 ULN, and eGFR \< 40ml/min/1.73m2.
• Use of intravenous antibiotics or oral quinolones within 14 days of screening.
• With the exception of Azithromycin the use of oral antibiotics including prophylactic antibiotics (e.g., augmentin, tetracycline, cloxacillin, cephalosporins, trimethoprim/sulfamethoxazole) within 14 days of screening.
• Initiation of high dose ibuprofen, dornase alpha, hypertonic saline or aerosolized antibiotics within 30 days of screening.
• On medications that are known to have potential serious interactions with simvastatin (as listed on page 10 of this protocol).
• Use of systemic corticosteroids within 30 days of screening.
• Investigational drug use within 30 days of screening.
• Other major organ dysfunction excluding pancreatic dysfunction.
• History of lung transplantation or currently on lung transplant list.
• Pregnant, breast feeding, or if post-menarche female, unwilling to practice birth control during participation in the study.
• Chronic users of niacin, azole antifungals (itraconazole, ketoconazole, voriconazole), telithromycin, fibric acid derivatives, HIV protease inhibitors, amiodarone, digoxin and/or cyclosporine (to decrease the risk of statin-related myotoxicity).
• Patients who are colonized or infected with Burkholderia cepacia complex are excluded.

Sponsors & Collaborators

• University of British Columbia: lead

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Simvastatin

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Lovastatin; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Officials

• Paul Man, MD

  University of British Columbia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2010
• First Posted: March 25, 2010
• Study Start: May 1, 2010
• Primary Completion: May 1, 2010
• Study Completion: May 1, 2010
• Last Updated: January 30, 2014

Record last verified: 2014-01