Clinical Trials on Phosphomannomutase II Congenital Disorder of Glycosylation in Canada

Total 36 results

Universidade Norte do Paraná
Hopital du Sacre-Coeur de Montreal

Completed

Effects of Dynamic Splinting on Knee Flexion Angle After Total Knee Arthroplasty: A Randomized Controlled Trial

Acquired Fixed Flexion Deformity of the Knee (Disorder)

Canada
Shriners Hospitals for Children

Completed

Botox Injection for Lower Extremity Lengthening and Deformity Correction Surgery (BOLLD)

Lower Extremity Deformities, Congenital | Unequal Length of Limbs; Congenital

Canada, United States
University of British Columbia
OrthoPediatrics

Recruiting

Pediatric Orthopaedic Implant Safety & Efficacy (Global POISE)

Hip Dysplasia | Hip Dislocation | Pediatric ALL | Orthopedic Disorder | Hip Disease | Fracture | Implant Complication | Deformity of Limb | Deformity; Bone | Lengthening; Leg | Implant Breakage

Canada
University Health Network, Toronto

Completed

Put a Face to a Name (Part A): The Effects of Photographic Aids on Patient Satisfaction, Clinician Communication, and Quality of Care (Face2Name)

Effects of Photographic Aids (Photos of Faces) on Patient Recall of Their Clinical Care Team | Effects of Photographic Aids (Photos of Faces) on Clinician-patient Communication | Effects of Photographic Aids (Photos of Faces) on Overall Patient Satisfaction

Canada
Jean-Patrice Baillargeon
Canadian Institutes of Health Research (CIHR)

Completed

Role of Insulin Action and Free Fatty Acids in Hyperandrogenism of Women With Polycystic Ovary Syndrome

Polycystic Ovary Syndrome

Canada
Clinique Ovo

Recruiting

Effect of Hyperandrogenism on IVF Outcomes in PCOS Patients (HIP)

PCOS (Polycystic Ovary Syndrome) of Bilateral Ovaries

Canada
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia (RADICHOL 1)

Metabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditions

United States, Taiwan, Brazil, Canada, South Africa, United Kingdom, Singapore
Novo Nordisk A/S

Completed

Survey Evaluating the Psychosocial Effects of Living With Haemophilia (HERO)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B | Relatives to/Carers of Patients

Spain, Canada, United Kingdom
Eli Lilly and Company

Completed

Somatropin Treatment to Final Height in Turner Syndrome (GDCT)

Turner Syndrome

Canada
Novo Nordisk A/S

Completed

Induction of Puberty With 17-beta Estradiol in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

Spain
Unity Health Toronto
Dartmouth College; Cure HHT

Active, not recruiting

Hereditary Hemorrhagic Telangiectasia (HHT) Research Outcomes Registry (HHT)

Hereditary Hemorrhagic Telangiectasia | HHT | Arteriovenous Malformation of Brain

Canada
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

Metabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditions

United States, Canada, Brazil, Taiwan, Singapore, South Africa, United Kingdom
Merck KGaA, Darmstadt, Germany
Merck Serono S.A., Geneva

Completed

First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study

Turner Syndrome | Idiopathic Growth Hormone Deficiency

Spain, France, Canada, United Kingdom, Czech Republic, Argentina, Germany, Italy, Sweden
Anagnostou, Evdokia, M.D.
The Hospital for Sick Children; Canadian Institutes of Health Research (CIHR); Alberta Health services and other collaborators

Not yet recruiting

Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders (CALM)

Anxiety Disorders | Generalized Anxiety Disorder | Anxiety | Tuberous Sclerosis | ADHD | Neurodevelopmental Disorders | Autism Spectrum Disorder | Fragile X Syndrome | Social Anxiety | Social Anxiety Disorder | Agoraphobia | Tourette Syndrome | Tic Disorders | Autism | ADHD Predominantly Inattentive Type | ADHD - Combined... and other conditions

Canada
Merck KGaA, Darmstadt, Germany

Completed

Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®

Growth Hormone Deficiency | Turner Syndrome

Argentina, Australia, Austria, Canada, France, Germany, Italy, Norway, Russian Federation, Singapore, Spain, Sweden, United Kingdom
University of British Columbia
Vancouver Coastal Health Research Institute; Genome British Columbia

Recruiting

Advancing Cardiac Care Unit-based Rapid Assessment and Treatment of hypErcholesterolemia (ACCURATE)

Acute Coronary Syndrome | Familial Hypercholesterolemia | STEMI | NSTEMI - Non-ST Segment Elevation MI | Familial Hypercholesterolemia - Heterozygous | Familial Hypercholesterolemia Due to Genetic Defect of Apolipoprotein B | Familial Hypercholesterolemia Due to Heterozygous LDL Receptor Mutation and other conditions

Canada
University Hospital, Geneva

Completed

Quality of Life in Patients With Congenital Afibrinogenemia (QualyAFIB)

Afibrinogenemia, Congenital

France, Turkey, Switzerland, Netherlands, Spain, India, Poland, Canada, United States, Algeria, Egypt, Germany, Italy, Japan, Kuwait, Lebanon, Morocco, Pakistan, Serbia, Slovakia, Tunisia
Regeneron Pharmaceuticals

Recruiting

A Study of DB-OTO, an Adeno-associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations (CHORD)

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Spain, United Kingdom, United States
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
University of California, Los Angeles
Université de Montréal

Withdrawn

Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery

Stroke | Epilepsy | Tuberous Sclerosis | Sturge-Weber Syndrome | Focal Cortical Dysplasia | Hemimegalencephaly | Polymicrogyria | Rasmussen Encephalitis | Gliosis | Tumor, Brain

United States, Canada
Neurocrine Biosciences

Active, not recruiting

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Congenital Adrenal Hyperplasia

United States, United Kingdom, France, Greece, Italy, Spain, Belgium, Canada, Germany, Poland
Spruce Biosciences

Active, not recruiting

A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH

Congenital Adrenal Hyperplasia

United States, Australia, Brazil, Estonia, Germany, Korea, Republic of, Latvia, Lithuania, Poland, Romania, Spain, Sweden, Turkey, Canada, Italy, United Kingdom
Washington University School of Medicine

Active, not recruiting

Spinraza in Adult Spinal Muscular Atrophy (SAS)

Spinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type II

United States, Canada
Neurocrine Biosciences

Active, not recruiting

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

Congenital Adrenal Hyperplasia

United States, Spain, Belgium, Austria, Bulgaria, Canada, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Serbia, Sweden, United Kingdom
Spruce Biosciences

Terminated

A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

Congenital Adrenal Hyperplasia

United States, Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Korea, Republic of, Latvia, Lithuania, Netherlands, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
Children's Oncology Group
National Cancer Institute (NCI)

Active, not recruiting

Combination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor

Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Adult Kidney Wilms Tumor | Beckwith-Wiedemann Syndrome | Childhood Kidney Wilms Tumor | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Stage V Kidney...

United States, Canada, Australia, New Zealand, Puerto Rico, Israel
National Tuberous Sclerosis Association

Recruiting

TSC Biosample Repository and Natural History Database

Tuberous Sclerosis | Lymphangioleiomyomatosis

United States, Canada
Novo Nordisk A/S

Recruiting

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8)

SGA, Turner Syndrome, Noonan Syndrome, ISS

Korea, Republic of, United States, Ireland, United Kingdom, Finland, France, Netherlands, Japan, Thailand, Belgium, China, Israel, Brazil, India, Portugal, Austria, Bulgaria, Canada, Croatia, Germany, Greece, Malaysia, Poland, Saudi Arabia and more
Marinus Pharmaceuticals

Enrolling by invitation

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, China, Italy, France, Israel, Germany, Canada, Australia
Novartis Pharmaceuticals

Active, not recruiting

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Tuberous Sclerosis Complex

United States, Spain, Belgium, Taiwan, Turkey, Australia, Korea, Republic of, Thailand, France, Hungary, United Kingdom, Italy, Japan, Canada, Russian Federation, Poland, Mexico, Colombia
Marinus Pharmaceuticals

Active, not recruiting

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (TrustTSC)

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, Israel, Italy, Germany, France, Canada
Novartis Pharmaceuticals

Completed

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) (EXIST-1)

Tuberous Sclerosis | Subependymal Giant Cell Astrocytoma

United States, Belgium, Canada, Germany, Italy, Russian Federation, Netherlands, Poland, Australia, United Kingdom
Novartis Pharmaceuticals

Completed

A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures (EXIST-3)

Tuberous Sclerosis Complex-associated Refractory Seizures

United States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
Novartis Gene Therapies

Active, not recruiting

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type III

United States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
Novartis Pharmaceuticals

Completed

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)

Lymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC)

United States, United Kingdom, Germany, Italy, Russian Federation, Netherlands, Japan, Canada, Poland, France, Spain
Children's Oncology Group
National Cancer Institute (NCI)

Active, not recruiting

Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma

Acute Lymphoblastic Leukemia | Down Syndrome | Philadelphia Chromosome Positive | Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 | Childhood B Acute Lymphoblastic Leukemia | Adult B Lymphoblastic Lymphoma | Ann Arbor Stage I B Lymphoblastic Lymphoma | Ann Arbor Stage... and other conditions

United States, Canada, Puerto Rico, Australia, New Zealand, Switzerland, Ireland

Clinical Trials on Phosphomannomutase II Congenital Disorder of Glycosylation in Canada

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Angola

Conditions

Rare Diseases

Drug Interventions

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