DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
299	嚢胞性線維症	2

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT03894657 (ClinicalTrials.gov)	December 18, 2019	12/2/2019	Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.	Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis	Cystic Fibrosis;Homozygous F508del Mutation	Diagnostic Test: Nasal brushing;Other: Sputum sample;Other: blood sample;Drug: Orkambi	Assistance Publique - Hôpitaux de Paris	NULL	Recruiting	5 Years	N/A	All	104	N/A	France
2	NCT02589444 (ClinicalTrials.gov)	December 2015	20/10/2015	Vitamin D and Microbiota in Cystic Fibrosis	Pilot Study Evaluating the Role of Vitamin D Repletion on Gut and Lung Microbiota in Cystic Fibrosis	Vitamin D Deficiency;Cystic Fibrosis	Dietary Supplement: High-Dose Vitamin D3;Other: Stool Sample;Other: Sputum Sample;Other: Sham Comparator;Procedure: Blood draw	Emory University	NULL	Completed	18 Years	N/A	All	41	N/A	United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT03894657
(ClinicalTrials.gov)

December 18, 2019

12/2/2019

Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.

Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis

Cystic Fibrosis;Homozygous F508del Mutation

Diagnostic Test: Nasal brushing;Other: Sputum sample;Other: blood sample;Drug: Orkambi

Assistance Publique - Hôpitaux de Paris

NULL

Recruiting

5 Years

N/A

All

104

N/A

France

NCT02589444
(ClinicalTrials.gov)

December 2015

20/10/2015

Vitamin D and Microbiota in Cystic Fibrosis

Pilot Study Evaluating the Role of Vitamin D Repletion on Gut and Lung Microbiota in Cystic Fibrosis

Vitamin D Deficiency;Cystic Fibrosis

Dietary Supplement: High-Dose Vitamin D3;Other: Stool Sample;Other: Sputum Sample;Other: Sham Comparator;Procedure: Blood draw

Emory University

NULL

Completed

18 Years

N/A

All

N/A

United States