Clinical Trials on Limb Deformities, Congenital in New Zealand

Total 955 results

Proteon Therapeutics

Completed

A Phase 1-2 Study of PRT-201 Administered in Chronic Kidney Disease (CKD) Patients

Chronic Kidney Disease

United States
Flex Pharma, Inc.

Terminated

A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps. (COMMIT)

Charcot-Marie-Tooth Disease

United States
Seaside Therapeutics, Inc.

Completed

Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A (guardian™4)

Congenital Bleeding Disorder | Haemophilia A

United States, Denmark, Austria, Spain, Japan, Brazil, Algeria, Lithuania, Portugal, Russian Federation, Serbia, Turkey, Greece, Hong Kong, Puerto Rico, Hungary, China, Poland
Michael Shy
Johns Hopkins University; University of Colorado, Denver; King's College Hospital... and other collaborators

Recruiting

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others (INC-6601)

Charcot Marie Tooth Disease

United States, Australia, Italy, United Kingdom, Canada
Gedeon Richter Plc.

Completed

Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

Prader-Willi Syndrome

United States, Spain, France, Italy, Czechia
Novartis Pharmaceuticals

Completed

A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism (SPARTAN)

Priapism

United States
Global Healthy Living Foundation
University of Alabama at Birmingham

Recruiting

PatientSpot Formerly Known as ArthritisPower (GHLF)

Heart Failure | Stroke | Epilepsy | Multiple Sclerosis | Obesity | Diabetes | Chronic Obstructive Pulmonary Disease | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Peripheral Arterial Disease | Inflammatory Bowel Diseases | Amyotrophic Lateral Sclerosis | Psoriasis | Asthma | Muscular Dystrophy | Fibromyalgia | Alzheimer... and other conditions

United States
University of Iowa
Johns Hopkins University; University of Colorado, Denver; King's College Hospital... and other collaborators

Recruiting

Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2 (INC-6602)

Charcot-Marie-Tooth Disease, Type Ia (Disorder) | HMSN

United States, Italy, United Kingdom, Australia
HedgePath Pharmaceuticals, Inc.

Completed

Open-label Trial of SUBA™-Itraconazole (SUBA-Cap) in Subjects With Basal Cell Carcinoma Nevus Syndrome (BCCNS)

Basal Cell Carcinoma in Basal Cell Nevus Syndrome

United States
Calcilytix Therapeutics, Inc., a BridgeBio company

Recruiting

Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1 (CALIBRATE)

Autosomal Dominant Hypocalcemia (ADH)

United States, Australia, United Kingdom, Denmark, Italy, France, Japan, Taiwan, Canada, Czechia, Netherlands
National Hospital Organization Nagoya Medical Center

Recruiting

ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016

Acute Lymphoblastic Leukemia | Down Syndrome | Childhood Cancer

Taiwan, Thailand, Hong Kong, Singapore, Malaysia, Japan
Proteon Therapeutics

Completed

A Study of PRT-201 Administered Immediately After Radiocephalic Arteriovenous Fistula(AVF) Creation in Patients With Chronic Kidney Disease (CKD) (PATENCY-1)

Chronic Kidney Disease

United States
AstraZeneca
Iqvia Pty Ltd

Terminated

Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease (HESTIA3)

Sickle Cell Disease

Italy, Spain, United Kingdom, Belgium, United States, India, South Africa, Brazil, Turkey, Egypt, Ghana, Greece, Kenya, Lebanon, Tanzania, Uganda
Novo Nordisk A/S

Completed

A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A (pathfinder8)

Congenital Bleeding Disorder | Haemophilia A

United States, Netherlands, Spain, Switzerland, Taiwan, Turkey, United Kingdom, Croatia, Germany, Brazil, France, Japan, Australia, Hungary, Israel, Korea, Republic of, Lithuania, Portugal, Ukraine, Greece, Italy, Denmark, Norway, Canada and more
Tetra Discovery Partners

Recruiting

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

Fragile X Syndrome

United States
Tetra Discovery Partners

Enrolling by invitation

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Dana-Farber Cancer Institute
National Cancer Institute (NCI); Wyeth is now a wholly owned subsidiary of... and other collaborators

Completed

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

Tuberous Sclerosis | Nonmalignant Neoplasm | Lymphangioleimyomatosis | Kidney Angiomyolipoma

United States
Celgene

Completed

Lenalidomide Safety/Efficacy in Myelodysplastic Syndromes (MDS) Associated With a Deletion (Del)(5q) Cytogenetic Abnormality

Myelodysplastic Syndromes

United States, Germany
ASH Research Collaborative
University of Pennsylvania; American Society of Hematology

Active, not recruiting

COVID-19 Vaccine Response in Sickle Cell Disease

COVID-19 | Sickle Cell Disease

United States
HealthCore-NERI
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Preventing Acute Chest Syndrome by Transfusion Feasibility Study (PROACTIVE)

Sickle Cell Disease

United States
Novo Nordisk A/S

Completed

A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A (pathfinder™5)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Switzerland, Malaysia, Brazil, Canada, Japan, United Kingdom, Israel, Portugal, Ukraine, Greece, Lithuania, Puerto Rico, Turkey, Italy
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
Novo Nordisk A/S

Completed

Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects

Congenital Bleeding Disorder | Haemophilia A

United States, Netherlands, Austria, Spain, Poland, United Kingdom, Croatia, Germany, Malaysia, Australia, France, Israel, Thailand, Ukraine, Turkey
Applied Genetics

Unknown

T4N5 Liposome Lotion Compared With Placebo Lotion for Preventing Actinic Keratoses in Patients With Xeroderma Pigmentosum

Precancerous Condition

United States, Germany, United Kingdom
Eli Lilly and Company
Daiichi Sankyo, Inc.

Terminated

A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)

Sickle Cell Disease

United Arab Emirates, Italy, United States, Egypt, United Kingdom, Belgium, Turkey, Brazil, Canada, Ghana, Kenya, Lebanon, Oman, Saudi Arabia
Pharnext S.C.A.
Worldwide Clinical Trials

Active, not recruiting

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PREMIER)

Charcot-Marie-Tooth Disease

United States, Spain, Belgium, Italy, Israel, Canada, Denmark, France, Germany
Zafgen, Inc.

Terminated

Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome (bestPWS)

Obesity | Prader-Willi Syndrome

United States
University of California, San Francisco
University of Chicago; National Institute of Neurological Disorders and Stroke... and other collaborators

Recruiting

Modifiers of Disease Severity in Cerebral Cavernous Malformations

Cerebral Cavernous Malformations | Cavernous Angioma, Familial | Cerebral Cavernous Hemangioma

United States
The University of Texas Health Science Center,...

Completed

Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

Tuberous Sclerosis | Angiofibromas

United States, Australia
Johns Hopkins University
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Renal Anhydramnios Fetal Therapy (RAFT)

Anhydramnios | Potter Syndrome | Lung Hypoplasia | Multicystic Dysplastic Kidney | Multicystic Renal Dysplasia, Bilateral | Lower Urinary Tract Obstructive Syndrome | Fetal Renal Anomaly

United States
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
Sol-Gel Technologies, Ltd.
Premier Research Group plc

Recruiting

Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome

Gorlin Syndrome

United States
Neuren Pharmaceuticals Limited

Completed

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

Fragile X Syndrome

United States
Terumo Medical Corporation

Completed

An Observational Study of Radial Access Embolization Procedures Using HydroPearl Microspheres (RAVI Registry)

Benign Prostatic Hyperplasia | Arteriovenous Malformations | Uterine Fibroid | Hypervascular Tumors

United States
Novo Nordisk A/S

Recruiting

Research Study Investigating How Well NDec Works in People With Sickle Cell Disease (ASCENT1)

Sickle Cell Disease

Spain, United States, United Kingdom, Canada, Italy, India, Turkey, Greece, South Africa, France, Lebanon, Oman
Neurona Therapeutics
California Institute for Regenerative Medicine (CIRM)

Recruiting

FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis

United States
Janssen Research & Development, LLC

Active, not recruiting

An Efficacy and Safety Study of Niraparib in Men With Metastatic Castration-Resistant Prostate Cancer and DNA-Repair Anomalies (Galahad)

Prostatic Neoplasms

United States, Australia, France, United Kingdom, Belgium, Taiwan, Spain, Israel, Netherlands, Sweden, Korea, Republic of, Canada, Russian Federation, Brazil, Denmark
Novo Nordisk A/S

Completed

Observational Registry of the Treatment of Glanzmann's Thrombasthenia

Congenital Bleeding Disorder | Glanzmann's Disease

Spain, Bulgaria, Pakistan, Belgium, Germany, France, United Kingdom, Italy, Hungary, Netherlands, United States, Algeria, Austria, Sweden, Switzerland
ApoPharma

Terminated

Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias (FIRST)

Iron Overload | Sickle Cell Disease | Other Anemias

United States, Turkey, United Kingdom, Egypt, Saudi Arabia, Brazil, Canada, Tunisia
Regulus Therapeutics Inc.

Completed

A Study of RGLS4326 in Patients With Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney Disease, Autosomal Dominant

United States
Centre hospitalier de l'Université de Montréal...
Centre Hospitalier Régional et Universitaire de Brest

Recruiting

Treatment of Brain AVMs (TOBAS) Study (TOBAS)

Arteriovenous Malformations | Unruptured Brain Arteriovenous Malformation | Ruptured Brain Arteriovenous Malformation | AVM | BAVM

France, United States, Canada, Brazil, Chile, Colombia
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States
HemaQuest Pharmaceuticals Inc.

Terminated

Effects of HQK-1001 in Patients With Sickle Cell Disease

Sickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin S

United States, Lebanon, Egypt, Canada, Jamaica
Palladio Biosciences

Completed

The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

Autosomal Dominant Polycystic Kidney Disease

United States
Hoffmann-La Roche

Completed

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Fragile X Syndrome

United States
Eisai Inc.
Pfizer

Terminated

Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial

Down Syndrome

United States
Novo Nordisk A/S

Completed

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A (pathfinder™2)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, Japan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Puerto Rico, Norway, Korea, Republic of, Bulgaria, Russian...
Enyo Pharma

Recruiting

Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1) (ALPESTRIA-1)

Alport Syndrome

Germany, Spain, United States, France
Novo Nordisk A/S

Completed

Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A. (pathfinder™3)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, United Kingdom, Croatia, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Italy, Japan, Norway, Bulgaria

Clinical Trials on Limb Deformities, Congenital in New Zealand

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