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Results for “Genetics Disease”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT04058353
What this trial is testing

VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Who this might be right for
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated 271
Large-scale testing (Phase 3)Study completedNCT05176639
What this trial is testing

A Safety, Tolerability, and Efficacy Study of Veligrotug (VRDN 001) in Participants With Thyroid Eye Disease (TED)

Who this might be right for
Thyroid Eye Disease
Viridian Therapeutics, Inc. 113
Not applicableLooking for participantsNCT05178277
What this trial is testing

Czech AATD Registry

Who this might be right for
Alpha-1-antitrypsin Deficiency
Thomayer University Hospital 300
Testing effectiveness (Phase 2)Study completedNCT04259281
What this trial is testing

The Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Who this might be right for
Angelman Syndrome
Ultragenyx Pharmaceutical Inc 74
Testing effectiveness (Phase 2)UnknownNCT01883219
What this trial is testing

TKI Therapy Based on Molecular Monitoring in Allogeneic-HSCT Recipients With Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia

Who this might be right for
Philadelphia Chromosome Positive Acute Lymphocytic LeukemiaStem Cell TransplantationMinimal Residual Disease
Nanfang Hospital, Southern Medical University 80
Large-scale testing (Phase 3)Study completedNCT01747330
What this trial is testing

Evaluation of Safety and Tolerability of Creon Micro in Children Younger Than Four Years With Pancreatic Exocrine Insufficiency

Who this might be right for
Pancreatic Exocrine Insufficiency
Abbott Products 40
Large-scale testing (Phase 3)UnknownNCT01824030
What this trial is testing

FFR or OCT Guidance to RevasculariZe Intermediate Coronary Stenosis Using Angioplasty

Who this might be right for
Ischemic Heart Disease
Catholic University of the Sacred Heart 400
Not applicableStudy completedNCT02825667
What this trial is testing

Effectiveness of the Myofascial Therapy in the Hemophilic Arthropathy of Ankle

Who this might be right for
Haemophilia
Real Fundación Victoria Eugenia 65
Testing effectiveness (Phase 2)Ended earlyNCT01349192
What this trial is testing

Early Methicillin-resistant Staphylococcus Aureus (MRSA) Therapy in Cystic Fibrosis (CF)

Who this might be right for
Cystic FibrosisMethicillin-resistant Staphylococcus Aureus
University of North Carolina, Chapel Hill 47
Not applicableEnrolling By InvitationNCT05238909
What this trial is testing

Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1

Who this might be right for
Neurofibromatosis 1NF1Neurofibromatosis Type 1
Ann & Robert H Lurie Children's Hospital of Chicago 200
Not applicableUnknownNCT03859531
What this trial is testing

Gastrointestinal Study at Orkambi Therapy in CF Patients

Who this might be right for
Cystic Fibrosis
Karolinska University Hospital 20
Not applicableLooking for participantsNCT03185702
What this trial is testing

UTHealth Turner Syndrome Research Registry

Who this might be right for
Turner Syndrome
The University of Texas Health Science Center, Houston 200
Not applicableStudy completedNCT01842919
What this trial is testing

Dance and Huntington Disease

Who this might be right for
Huntington Disease
Institut National de la Santé Et de la Recherche Médicale, France 53
Not applicableUnknownNCT05987319
What this trial is testing

Feasibility Study to Collect and Assess Safety Data for a Radiofrequency Microneedling Device for Electrocoagulation and Hemostasis of Soft Tissues for Dermatologic Conditions

Who this might be right for
WrinkleFine LinesCrepey Skin+5 more
Cynosure, Inc. 20
Not applicableLooking for participantsNCT07229430
What this trial is testing

Safety and Effectiveness Evaluation of the High-intensity Focused Ultrasound Device for Lifting Lax Submental and Neck Tissue

Who this might be right for
Skin Laxity (Submental and Neck)Facial Skin LaxitySubmental Tissue Laxity
CLASSYS Inc. 273
Large-scale testing (Phase 3)Active Not RecruitingNCT06224907
What this trial is testing

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Who this might be right for
Hemophilia A
BioMarin Pharmaceutical 6
Not applicableStudy completedNCT04923464
What this trial is testing

A Decentralized Study to Evaluate Physical Activity and Cough Frequency Using Wearable Technology in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated 51
Not applicableStudy completedNCT01647399
What this trial is testing

Vitamin D, Steroids, and Asthma in African American Youth

Who this might be right for
AsthmaAllergy
Robert J. Freishtat 273
Testing effectiveness (Phase 2)Study completedNCT05421429
What this trial is testing

KN057 Multiple Dose Study in Moderately Severe to Severe Hemophilia

Who this might be right for
Hemophilia
Suzhou Alphamab Co., Ltd. 24
Testing effectiveness (Phase 2)Study completedNCT03796637
What this trial is testing

Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 6
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