Orkambi Exercise Study

NCT02821130 | Completed

• 1 other identifier: H16-01164
• Study Type: observational
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

Shortness of breath (dyspnea) during exercise is a major source of distress and is a commonly reported symptom in patients with cystic fibrosis (CF). A recent treatment option known as Orkambi, which combines the drugs lumacaftor and ivacaftor, may be used in patients with CF to help improve lung health. However, the effects of this combination therapy on dyspnea and exercise performance, a known predictor of survival in CF, are not clear. The investigators aim to understand the effects of Orkambi on these symptoms and to gain new insight into the potential health improvements in CF from using this treatment option.

Conditions

Lung; Disease; Cystic Fibrosis

Keywords

Dyspnea; Exercise; Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Change in iso-time dyspnea rating from baseline (visit 2) to visit 3 and 4 during constant-load exercise tests.

  Dyspnea rating measured using the Borg 0-10 category ratio scale.

  Parameters will be measured during 3 visits. Visit 2 will occur before the participants go on drug (Orkambi). Visit 3 and 4 will occur at 1 month and 3 months after initiating full dose of drug, respectively. All visits will be completed within 4 months.

Secondary Outcomes (5)

• Change from baseline cardio-respiratory responses during constant-load exercise tests (prior to Orkambi initiation to full dose) at 1 and 3 months after initiating full dose of Orkambi.

  Parameters will be measured during 3 visits. Visit 2 will occur before the participants go on drug (Orkambi). Visit 3 and 4 will occur at 1 month and 3 months after initiating full dose of drug, respectively. All visits will be completed within 4 months.

• Change from baseline chronic activity-related dyspnea (prior to Orkambi initiation to full dose) at 1 and 3 months after initiating full dose of Orkambi.

  Parameters will be measured during 3 visits. Visit 2 will occur before the participants go on drug (Orkambi). Visit 3 and 4 will occur at 1 month and 3 months after initiating full dose of drug, respectively. All visits will be completed within 4 months.

• Change from baseline quality of life (prior to Orkambi initiation to full dose) at 1 and 3 months after initiating full dose of Orkambi.

  Parameters will be measured during 3 visits. Visit 2 will occur before the participants go on drug (Orkambi). Visit 3 and 4 will occur at 1 month and 3 months after initiating full dose of drug, respectively. All visits will be completed within 4 months.

• Change from baseline physical activity (prior to Orkambi initiation to full dose) at 1 and 3 months after initiating full dose of Orkambi.

  Parameters will be measured during 3 visits. Visit 2 will occur before the participants go on drug (Orkambi). Visit 3 and 4 will occur at 1 month and 3 months after initiating full dose of drug, respectively. All visits will be completed within 4 months.

• Change from baseline pulmonary function measures (prior to Orkambi initiation to full dose) at 1 and 3 months after initiating full dose of Orkambi.

  Parameters will be measured during 3 visits. Visit 2 will occur before the participants go on drug (Orkambi). Visit 3 and 4 will occur at 1 month and 3 months after initiating full dose of drug, respectively. All visits will be completed within 4 months.

Study Arms (1)

Cystic Fibrosis Patients

Participants diagnosed with cystic fibrosis

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The CF patients will be recruited from the St. Paul's Hospital UBC Adult CF Clinic.

You may qualify if:

• Confirmed diagnosis of CF and homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
• Plan to initiate Orkambi™ by the treating physician within 30 days of the enrolment visit
• Aged 19 years or older
• Stable clinical status based on clinical judgment of the treating physicians (Drs Wilcox, Quon or Goodwin)
• Forced Expiratory Volume in 1 second (FEV1.0) \< 90% predicted
• Body mass index greater than 16 or less than 30 kg/m2
• Currently non-smoking or a past smoking history of less than 20 pack-years
• Able to read and understand English

You may not qualify if:

• A disease other than CF that could importantly contribute to dyspnea or exercise limitation
• Chronic airway infection with Mycobacterium abscessus, Burkholderia cepacia complex, or other organisms with infection control implications based on the treating physicians
• Contraindications to clinical exercise testing
• Use of supplemental oxygen or desaturation less than 85% with exercise
• Diagnosis of pneumothorax in the past 4 weeks
• History of organ transplantation

Sponsors & Collaborators

• University of British Columbia: lead

Study Sites (1)

UBC James Hogg Research Centre, St. Paul's Hospital

Vancouver, British Columbia, V6Z1Y6, Canada

Location

Related Publications (1)

• Quon BS, Ramsook AH, Dhillon SS, Mitchell RA, Boyle KG, Wilcox PG, Guenette JA. Short-term effects of Lumacaftor/Ivacaftor (Orkambi) on exertional symptoms, exercise performance, and ventilatory responses in adults with cystic fibrosis. Respir Res. 2020 Jun 1;21(1):135. doi: 10.1186/s12931-020-01406-z.

  PMID: 32487229 DERIVED

MeSH Terms

Conditions

Lung Diseases; Cystic Fibrosis; Dyspnea; Motor Activity

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Pancreatic Diseases; Digestive System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Respiration Disorders; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Behavior

Study Officials

• Jordan A Guenette, PhD

  University of British Columbia - Centre for Heart Lung Innovation

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 6, 2016
• First Posted: July 1, 2016
• Study Start: July 1, 2016
• Primary Completion: December 1, 2019
• Study Completion: June 1, 2020
• Last Updated: November 17, 2020

Record last verified: 2020-11

Locations

CA (1)