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Trials in Diseases and Abnormalities at or Before Birth

708 Studies Found

A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

Location: China
Status: Recruiting
Phase: Phase 1
Results: N/A
Start Date: 20 Mar, 2018
Completion Date: 30 Oct, 2018
Healthy VolunteersHemophilia A

Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."

Location: Italy
Status: Active, not recruiting
Results: N/A
Start Date: 14 Apr, 2016
Completion Date: 20 Mar, 2018
Hemophilia A

Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

Location: Canada, United States
Status: Recruiting
Phase: Phase 3
Results: N/A
Start Date: 01 Jun, 2015
Completion Date: 01 Nov, 2018
AnemiaSickle Cell

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Location: Argentina, Austria, Belgium, Bulgaria, Canada, Greece, Israel, Italy, Lithuania, Netherlands, New Zealand, Norway, Poland, Romania, Spain, United Kingdom, United States
Status: Active, not recruiting
Phase: Phase 3
Results: N/A
Start Date: 29 May, 2013
Completion Date: 17 Feb, 2020
Hemophilia A

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia

Location: United States
Status: Active, not recruiting
Phase: Phase 1/Phase 2
Results: N/A
Start Date: 09 Sep, 2009
Completion Date: 01 Aug, 2018
Peripheral Blood Stem Cell TransplantationAnemiaSickle CellGraft-Versus-Host DiseaseSirolimus (Rapamune )Alemtuzumab (Campath )

Clinical and Genetic Studies of VACTERL Association

Location: United States
Status: Completed
Results: N/A
Start Date: 29 Sep, 2008
Completion Date: 29 Jun, 2017
Congenital AbnormalitiesBirth DefectsCongenital Defects

Blood Sampling for Research Related to Sickle Cell Disease

Location: United States
Status: Enrolling by invitation
Results: N/A
Start Date: 09 Oct, 2007
Sickle Cell TraitSickle Cell DiseaseSickle Cell Anemia

Safety and Dose Escalation Study of an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia A Subjects

Location: United States
Status: Recruiting
Phase: Phase 1/Phase 2
Results: N/A
Start Date: 01 Mar, 2018
Completion Date: 30 Jun, 2022
Hemophilia A

National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A

Location: United Kingdom
Status: Completed
Results: N/A
Start Date: 01 Sep, 2015
Completion Date: 30 Nov, 2017
Hemophilia A

GCSF Adjunct Therapy for Biliary Atresia

Location: United States
Status: Enrolling by invitation
Phase: Early Phase 1
Results: N/A
Start Date: 15 Jan, 2018
Completion Date: 31 Jan, 2019
Biliary Atresia

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