DDrare: Database of Drug Development for Rare Diseases

Pft (DrugBank: -)

3 diseases

ID	Disease name (Link within this page)	Number of trials
89	Lymphangioleiomyomatosis	1
113	Muscular dystrophy	1
299	Cystic fibrosis	1

89. Lymphangioleiomyomatosis [ 38 clinical trials, 42 drugs, (DrugBank: 19 drugs), 26 drug target genes, 134 drug target pathways]

Searched query = "Lymphangioleiomyomatosis", "LAM"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 38 trial found

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	NCT01799538 (ClinicalTrials.gov)	June 10, 2013	23/2/2013	Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis	13-H-0051 Bronchodilator Effects of Nebulized Versus Inhaled Albuterol in Subjects With Lymphangioleiomyomatosis	Lymphangioleiomyomatosis	Drug: albuterol inhaler;Drug: albuterol nebulizer;Procedure: PFT	National Heart, Lung, and Blood Institute (NHLBI)	NULL	Recruiting	18 Years	80 Years	Female	100	Phase 2	United States

113. Muscular dystrophy [ 567 clinical trials, 442 drugs, (DrugBank: 93 drugs), 55 drug target genes, 151 drug target pathways]

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 567 trial found

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	NCT02195999 (ClinicalTrials.gov)	December 2013	17/7/2014	Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy	Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy	Muscular Dystrophy, Duchenne	Other: Magnetic Resonance Imaging (MRI);Other: Pulmonary Function Testing (PFT);Other: Metabolic Exercise Testing using stationary bicycle;Other: Echocardiogram	University of Florida	CureDuchenne	Completed	5 Years	15 Years	Male	9		United States

299. Cystic fibrosis [ 1,592 clinical trials, 1,539 drugs, (DrugBank: 255 drugs), 81 drug target genes, 162 drug target pathways]

Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 1,592 trial found

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	NCT03579173 (ClinicalTrials.gov)	May 8, 2018	25/6/2018	Determinants of Early Cystic Fibrosis Lung Disease	Determinants of Early Cystic Fibrosis Lung Disease	Cystic Fibrosis in Children	Diagnostic Test: Infant PFT;Diagnostic Test: Lung Clearance Index;Diagnostic Test: Passive tidal breathing	Indiana University	NULL	Active, not recruiting	1 Month	5 Years	All	55		United States