Not applicableEnrolling By InvitationNCT06650358What this trial is testingClinical Outcomes of Patients with LAL+ in At Least One EyeWho this might be right forIOL, Cataract Frank A. Bucci, Jr., M.D. 25
Testing effectiveness (Phase 2)Study completedNCT02112994What this trial is testingSafety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc. 31
Not applicableActive Not RecruitingNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Not applicableStudy completedNCT04529616What this trial is testingSingle Center Exploratory Study To Evaluate The Use Of The RxSight Light Adjustable Lens (LAL) And The Light Delivery Device (LDD) To Improve Visual OutcomesWho this might be right forAphakiaCataract RxSight, Inc. 79
Not applicableStudy completedNCT04533191What this trial is testingBinocular Custom Vision Utilizing The Light Adjustable Lens (LAL)Who this might be right forCataractPseudophakia Vance Thompson Vision 25
Not applicableUnknownNCT02851550What this trial is testingAssessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant PatientsWho this might be right forLiver Post-transplant Patients Hospices Civils de Lyon 100
Post-approval studies (Phase 4)Study completedNCT02036489What this trial is testingPethema LAL-RI/2008: Treatment for Patients With Standard Risk Acute Lymphoblastic LeukemiaWho this might be right forAcute Lymphobkastic Leukemia PETHEMA Foundation 107
Early research (Phase 1)Study completedNCT01586455What this trial is testingHuman Placental-Derived Stem Cell TransplantationWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more New York Medical College 43
Not applicableLooking for participantsNCT06919393What this trial is testing"Triple Negative" Adult B-cell Acute Lymphoblastic Leukemia - TRINEG-ALLWho this might be right forLeukemia, Lymphoblastic Istituto Romagnolo per lo Studio dei Tumori Dino Amadori IRST S.r.l. IRCCS 60
Not applicableStudy completedNCT02372513What this trial is testingNational Lysosomal Acid Lipase Deficiency StudyWho this might be right forCholesteryl Ester Storage Disease Ankara University 810
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07522801What this trial is testingA Precision Medicine Trial for Patients With Relapsed or Refractory T Cell ALLWho this might be right forLAL Philippe ROUSSELOT 93
Not applicableStudy completedNCT01884220What this trial is testingWolman/CESD Natural History Chart Review and Longitudinal Follow-UpWho this might be right forWolman DiseaseCholesterol Ester Storage DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2 Children's Hospital Medical Center, Cincinnati 4
Not applicableNo Longer AvailableNCT02376751What this trial is testingAn Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc.
Early research (Phase 1)Looking for participantsNCT04532047What this trial is testingPEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)Who this might be right forMPS IMPS IIMPS IVA+6 more University of California, San Francisco 10
Not applicableStudy completedNCT01358370What this trial is testingA Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman PhenotypeWho this might be right forLysosomal Acid Lipase DeficiencyWolman Disease Alexion Pharmaceuticals, Inc. 40
Not applicableEnrolling By InvitationNCT07347379What this trial is testingLong-Term Clinical Outcomes Of Subjects Enrolled In The RxSight Light Adjustable Lens (LAL) And Light Delivery Device (LDD) New Enrollment StudyWho this might be right forAphakia Cataract RxSight, Inc. 108
Not applicableStudy completedNCT03660865What this trial is testingClinical Study to Evaluate the Safety and Effectiveness of the RxSight Light Adjustable Lens (LAL) in Subjects With Prior Corneal Refractive SurgeryWho this might be right forAphakiaCataract RxSight, Inc. 105
Not applicableUnknownNCT06287658What this trial is testingThe Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary IncontinenceWho this might be right forUrinary IncontinenceMenopauseWolman Disease+2 more Ondokuz Mayıs University 60
Testing effectiveness (Phase 2)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38
Not applicableEnded earlyNCT04177771What this trial is testingA Clinical Study to Evaluate The Safety and Effectiveness of The RxSight Light Adjustable Lens (LAL) In Subjects Desiring an Extended Depth of FocusWho this might be right forAphakiaCataractPresbyopia RxSight, Inc. 34