Orkambi Treatment in 2 to 5 Year Old Children With CF
| Status: | Recruiting |
|---|---|
| Conditions: | Pulmonary |
| Therapuetic Areas: | Pulmonary / Respiratory Diseases |
| Healthy: | No |
| Age Range: | 2 - 5 |
| Updated: | 3/14/2019 |
| Start Date: | April 2019 |
| End Date: | December 2020 |
| Contact: | Caroline Heres, M.S. |
| Email: | heresc@email.chop.edu |
| Phone: | 267-425-5311 |
Nutritional Impact of Orkambi Treatment in 2 to 5 Year Old Children Homozygous for F508del Mutations
The purpose of this observational research study is to determine the effects of clinically
prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations
in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy
expenditure, growth status and gut health and function.
prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations
in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy
expenditure, growth status and gut health and function.
Orkambi is a novel FDA approved (August, 2018) therapy for use in patients with cystic
fibrosis (CF) who are 2 to 5 years of age and homozygous for F508del mutations in the CFTR
gene. It is a combination of lumacaftor and ivacaftor that addresses both the processing and
gating defects of the F508del mutation. This investigator-initiated study is designed to
evaluate the nutritional, growth and GI impact of Orkambi treatment for this unique younger
(2 to 5 years) patient cohort. This proposal extends previous highly informative nutrition
and weight gain investigation of ivacaftor treatment in people with CF gating mutations to
another CFTR modulator treatment (Orkambi) in people homozygous for F508del mutations. The
primary aims of the study are to evaluate the impact of 24 weeks of Orkambi treatment in 2 to
5 year old subjects with CF homozygous for F508del mutations on sleeping or resting energy
expenditure, growth status and gut health and function in n=32 children ages 2.0 to 5.9 years
of age. Protocol evaluations will occur at baseline (pre-treatment) and 12 and 24 weeks after
clinically prescribed Orkambi treatment has begun. Other outcomes of significant clinical
interest in young subjects with CF will be explored. All subjects will be evaluated as
outpatient at The Children's Hospital of Philadelphia, and will be recruited both regionally
and nationally to ensure timely enrollment.
fibrosis (CF) who are 2 to 5 years of age and homozygous for F508del mutations in the CFTR
gene. It is a combination of lumacaftor and ivacaftor that addresses both the processing and
gating defects of the F508del mutation. This investigator-initiated study is designed to
evaluate the nutritional, growth and GI impact of Orkambi treatment for this unique younger
(2 to 5 years) patient cohort. This proposal extends previous highly informative nutrition
and weight gain investigation of ivacaftor treatment in people with CF gating mutations to
another CFTR modulator treatment (Orkambi) in people homozygous for F508del mutations. The
primary aims of the study are to evaluate the impact of 24 weeks of Orkambi treatment in 2 to
5 year old subjects with CF homozygous for F508del mutations on sleeping or resting energy
expenditure, growth status and gut health and function in n=32 children ages 2.0 to 5.9 years
of age. Protocol evaluations will occur at baseline (pre-treatment) and 12 and 24 weeks after
clinically prescribed Orkambi treatment has begun. Other outcomes of significant clinical
interest in young subjects with CF will be explored. All subjects will be evaluated as
outpatient at The Children's Hospital of Philadelphia, and will be recruited both regionally
and nationally to ensure timely enrollment.
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
We found this trial at
1
site
South 34th Street
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
215-590-1000
Principal Investigator: Virginia Stallings, MD
Phone: 267-425-5311
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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