Not applicableStudy completedNCT04049877What this trial is testingRetrospective and Prospective Disease Progression and Quality of Life in XLHWho this might be right forX-linked Hypophosphatemia (XLH) Medialis Ltd. 11
Testing effectiveness (Phase 2)Looking for participantsNCT03579875What this trial is testingAlpha/Beta TCD HCT in Patients With Inherited BMF DisordersWho this might be right forFanconi AnemiaSevere Aplastic AnemiaMyelodysplastic Syndromes+5 more Masonic Cancer Center, University of Minnesota 48
Not applicableStudy completedNCT05209087What this trial is testingEffects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular DystrophyWho this might be right forMuscular Dystrophy, Duchenne Hacettepe University 30
Testing effectiveness (Phase 2)Study completedNCT02489344What this trial is testingEvaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry DiseaseWho this might be right forFabry Disease Genzyme, a Sanofi Company 8
Testing effectiveness (Phase 2)UnknownNCT03936894What this trial is testingSingle Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular DystrophyWho this might be right forDuchenne Muscular Dystrophy Children's National Research Institute 3
Not applicableStudy completedNCT04281537What this trial is testingDescribe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.Who this might be right forFabry Disease Amicus Therapeutics 82
Testing effectiveness (Phase 2)Active Not RecruitingNCT01821781What this trial is testingImmune Disorder HSCT ProtocolWho this might be right forImmune Deficiency DisordersSevere Combined ImmunodeficiencyChronic Granulomatous Disease+11 more Washington University School of Medicine 20
Not applicableStudy completedNCT04708301What this trial is testingCharacterisation of Heart Involvement in Fabry Disease With T1 MappingWho this might be right forFabry Disease Manchester University NHS Foundation Trust 200
Not applicableStudy completedNCT00414583What this trial is testingStroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke PatientsWho this might be right forCerebrovascular Accident CENTOGENE GmbH Rostock 5,000
Not applicableUnknownNCT05641805What this trial is testingPhysical Activity Level and Cognitive Functions in Children With Duchenne Muscular DystrophyWho this might be right forDuchenne Muscular Dystrophy Muş Alparslan University 40
Not applicableUnknownNCT04698551What this trial is testingNIPD on cffDNA for Triplet Repeat DiseasesWho this might be right forMyotonic Dystrophy 1Huntington DiseaseFragile X Syndrome University Hospital, Montpellier 36
Not applicableLooking for participantsNCT06809972What this trial is testingSynovial Proliferation on Routine Ultrasound: Active or Inactive?Who this might be right forHaemophiliaHemophilia AHemophilia B Van Creveldkliniek 46
Testing effectiveness (Phase 2)Study completedNCT01304277What this trial is testingThis Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.Who this might be right forFabry Disease Shire 17
Testing effectiveness (Phase 2)Study completedNCT00176865What this trial is testingStem Cell Transplant for Immunologic or Histiocytic DisordersWho this might be right forHemophagocytic LymphohistiocytosisX-Linked Lymphoproliferative DisordersChediak-Higashi Syndrome+3 more Masonic Cancer Center, University of Minnesota 19
Large-scale testing (Phase 3)Study completedNCT00168077What this trial is testingProthrombin Complex Concentrate for Anticoagulant ReversalWho this might be right forAcquired Coagulation Factor Deficiency CSL Behring 40
Testing effectiveness (Phase 2)Study completedNCT04322357What this trial is testingTwice Weekly Steroids and Exercise as Therapy for DMDWho this might be right forDuchenne Muscular Dystrophy (DMD) University of Florida 21
Not applicableUnknownNCT04172181What this trial is testingMulti-center Clinical Study of Cord Blood Stem Cell Transplantation for SCIDWho this might be right forSevere Combined Immunodeficiency Disease Children's Hospital of Fudan University 50
Testing effectiveness (Phase 2)Enrolling By InvitationNCT06867107What this trial is testingAn Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101Who this might be right forDuchenne Muscular Dystrophy (DMD) Satellos Bioscience, Inc. 30
Not applicableUnknownNCT00871611What this trial is testingViennese Prevalence Study of Anderson-Fabry DiseaseWho this might be right forFabry DiseaseLeft Ventricular Hypertrophy Medical University of Vienna 4,000
Not applicableLooking for participantsNCT04398628What this trial is testingATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic DisordersWho this might be right forHematologic DisorderBleeding DisorderConnective Tissue Disorder+10 more American Thrombosis and Hemostasis Network 3,000