|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
13 December 2021 |
|
Main ID: |
NCT03795363 |
|
Date of registration:
|
28/12/2018 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Orkambi Treatment in 2 to 5 Year Old Children With CF
|
|
Scientific title:
|
Nutritional Impact of Orkambi Treatment in 2 to 5 Year Old Children Homozygous for F508del Mutations |
|
Date of first enrolment:
|
April 10, 2019 |
|
Target sample size:
|
28 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT03795363 |
|
Study type:
|
Observational |
|
Study design:
|
|
|
Phase:
|
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Virginia Stallings, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Children's Hospital of Philadelphia |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Cystic fibrosis and homozygous for F508del mutations, approved for treatment
- Age: 2.0 to 5.9 years
- In usual state of good health
- A clinical decision has been made for subject to begin Orkambi treatment
- Family committed to the 6 to 8 month study protocol with visits to the Children's
Hospital of Philadelphia (CHOP) that will last 2-3 days for the baseline visit (Visit
1) prior to Orkambi and the 24 week visit (Visit 3) after clinically prescribed
Orkambi treatment has begun, and will last up to 2 days for the 12 week visit (Visit
2) after Orkambi treatment has begun.
Exclusion Criteria:
- On parenteral nutrition
- Use of any medications that inhibit or induce cytochrome P450 (CYP) 3A
- Liver function tests elevated above 3x the reference range for age and sex
- Lung disease considered severe based on clinical impression by home CF center.
- Other illness affecting growth or nutritional status
- Other contraindications described for Orkambi therapy
Age minimum:
2 Years
Age maximum:
5 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Cystic Fibrosis
|
|
Intervention(s)
|
|
Drug: Orkambi
|
|
Primary Outcome(s)
|
|
Anthropometric Assessment
[Time Frame: 24 Weeks]
|
|
Sleeping or Resting Energy Expenditure
[Time Frame: 24 Weeks]
|
|
Secondary Outcome(s)
|
|
Fecal Elastase I/Pancreatic Function
[Time Frame: 24 Weeks]
|
|
Fecal Calprotectin/Gut Inflammation
[Time Frame: 24 Weeks]
|
|
Plasma Total Fatty Acids
[Time Frame: 24 Weeks]
|
|
Secondary ID(s)
|
|
18-015669
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|