DDrare: Database of Drug Development for Rare Diseases

Pft (DrugBank: -)

3 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
89	リンパ脈管筋腫症	1
113	筋ジストロフィー	1
299	嚢胞性線維症	1

89. リンパ脈管筋腫症　［臨床試験数：38，薬物数：42（DrugBank：19），標的遺伝子数：26，標的パスウェイ数：134］

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. 筋ジストロフィー　［臨床試験数：567，薬物数：442（DrugBank：93），標的遺伝子数：55，標的パスウェイ数：151］

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. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］

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