Clinical Trials on Myopathy Congenital in Colombia

Total 342 results

Emmaus Medical, Inc.

Completed

A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia

Sickle Cell Anemia | Sickle ß0-Thalassemia

United States
Amryt Research Limited

Completed

Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa (EASE)

Epidermolysis Bullosa

United States, Spain, Israel, Australia, France, Denmark, Switzerland, Italy, Hong Kong, Argentina, Austria, Brazil, Chile, Colombia, Czechia, Georgia, Germany, Greece, Hungary, Ireland, Romania, Russian Federation, Serbia, Singapore, U... and more
GlycoMimetics Incorporated

Completed

Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis

Sickle Cell Disease | Vaso-occlusive Crisis | Pain Crisis

United States, Canada
Merck Sharp & Dohme LLC

Completed

A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V114 in Children With Sickle Cell Disease (V114-023/PNEU-SICKLE)

Pneumococcal Infections

United States, Brazil, Colombia, Dominican Republic, Greece, Italy, Panama
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Angelman Syndrome

United States, United Kingdom, Canada, Australia, France, Germany, Spain, Israel
Elizabeth Berry-Kravis
National Institute of Neurological Disorders and Stroke (NINDS); Novartis Pharmaceuticals

Completed

AFQ056 for Language Learning in Children With FXS (FX-LEARN)

Fragile X Syndrome

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

Recruiting

A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 (REAL 9)

Turner Syndrome | Noonan Syndrome | SGA | ISS

Korea, Republic of, Malaysia, Netherlands, Spain, United States, Poland
Pfizer

Active, not recruiting

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease (HOPE Kids 2)

Sickle Cell Disease

United States, Oman, United Kingdom, France, Egypt, Ghana, Italy, Kenya, Nigeria, Saudi Arabia
Novartis Pharmaceuticals

Recruiting

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients

Sickle Cell Disease (SCD)

United States, Germany, Spain, Belgium, Italy, Lebanon, Brazil, Colombia, Oman, Switzerland, Turkey, United Kingdom, Canada, India, France
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
ADDMEDICA SASA

Completed

European Sickle Cell Disease Cohort - Hydroxyurea (ESCORT-HU)

Sickle Cell Disease

France, Germany, French Guiana, Greece, Guadeloupe, Italy, Martinique
Neuren Pharmaceuticals Limited

Completed

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

Fragile X Syndrome

United States
Parion Sciences
Vertex Pharmaceuticals Incorporated

Completed

Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia (CLEAN-PCD)

Primary Ciliary Dyskinesia

United States, United Kingdom, Netherlands, Denmark, Canada, Italy, Germany, Poland
CENTOGENE GmbH Rostock

Terminated

Biomarker for Alport Syndrome (BioAlport) (BioAlport)

Nephritis, Hereditary | Hematuria-Nephropathy-Deafness Syndrome

India, Sri Lanka, Albania, Georgia, Lithuania, Pakistan, Romania
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
Verinata Health, Inc.

Completed

Comparison of Aneuploidy Risk Evaluations (CARE)

Pregnancy | Down Syndrome | Edwards Syndrome | Patau Syndrome | Turners Syndrome

United States
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) (BioMaroteaux)

Lung Diseases | Eye Abnormalities | Lysosomal Storage Disease | Macroglossia | Obstructive Sleep Apnoea

Germany, India, Sri Lanka, Egypt
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
Hoffmann-La Roche

Completed

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Fragile X Syndrome

United States
Vesper Medical, Inc.

Active, not recruiting

Venous Stent for the Iliofemoral Vein Investigational Clinical Trial Using the DUO Venous Stent System (VIVID)

Deep Vein Thrombosis | Chronic Venous Insufficiency | May-Thurner Syndrome

United States, Poland
ADDMEDICA SASA

Recruiting

SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial) (SIKAMIC)

Sickle Cell Disease

France, Côte D'Ivoire, Guadeloupe, Mali, Martinique, Senegal
Icagen
McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Terminated

A Stratified Sickle Event Randomized Trial (ASSERT) (ASSERT)

Sickle Cell Disease

United States, France, United Kingdom, Brazil, Jamaica, Trinidad and Tobago
ADDMEDICA SASA
International Clinical Trials Association

Recruiting

ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study

Sickle Cell Disease

France, French Guiana, Germany, Greece, Guadeloupe, Italy, Martinique, Réunion
Cephalon

Completed

Extension Study of ACTIQ Treatment for Children and Adolescents With Breakthrough Pain

Pain | Cancer | Sickle Cell Anemia | Severe Burns

United States, Canada, Puerto Rico
Martina Bebin
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

Tuberous Sclerosis Complex

United States
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome (Harbor-A)

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Terminated

An Open Label Extension Study in Subjects With Fragile X Syndrome (209FX303)

Fragile X Syndrome

United States
Genzyme, a Sanofi Company

Completed

ATHENA: Natural History of Disease Study in Alport Syndrome Patients (RG012-01)

Alport Syndrome Patients With eGFR Between 45-90 ml/Min/1.73 m2

France, Germany, Australia, United States, United Kingdom, Canada
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
Women and Infants Hospital of Rhode Island
Sequenom, Inc.

Completed

A New Prenatal Blood Test for Down Syndrome (RNA)

Down Syndrome | Trisomy 21

United States, Canada, Israel, Hungary, Ireland, Argentina, Australia, Czech Republic, Italy, Spain
Novartis Pharmaceuticals

Active, not recruiting

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients (STAND)

Sickle Cell Disease (SCD)

United States, United Kingdom, Germany, Spain, Greece, Italy, Brazil, Lebanon, Turkey, Belgium, France, Jordan, India, Finland, Canada, Colombia, Netherlands, Oman, South Africa, Panama, Ghana
Forma Therapeutics, Inc.
Novo Nordisk A/S

Recruiting

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

Sickle Cell Disease

United States, Italy, Spain, United Kingdom, France, Lebanon, Germany, Oman, Canada, Greece
GlycoMimetics Incorporated

Terminated

Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease

Sickle Cell Disease | Sickle Cell Anemia | Vaso-occlusive Crisis | Pain Crisis | Sickle Cell Disorders

United States, Canada
Mast Therapeutics, Inc.

Completed

Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC) (EPIC)

Sickle Cell Disease | Vaso-occlusive Crisis

United States, Belgium, Brazil, Dominican Republic, Jamaica, Jordan, Lebanon, Oman, Panama, Spain, Turkey
Unity Health Toronto
National Institute of Neurological Disorders and Stroke (NINDS)

Recruiting

Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia (BVMC6203)

Hereditary Hemorrhagic Telangiectasia

Canada, United States, Netherlands
Zynerba Pharmaceuticals, Inc.

Recruiting

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome (RECONNECT)

Fragile X Syndrome

United States, Australia, United Kingdom, Ireland
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

Fragile X Syndrome

United States
New York State Institute for Basic Research
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Unknown

Vitamin E in Aging Persons With Down Syndrome

Alzheimer Disease | Down Syndrome

United States, Canada, Australia, United Kingdom
Actelion

Terminated

Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH) (ASSET-2)

Pulmonary Hypertension

United States, France, United Kingdom, Netherlands
Agios Pharmaceuticals, Inc.

Recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Sickle Cell Disease

United Kingdom, United States, Israel, Belgium, Netherlands, Germany, Canada, Brazil, France, Italy, Lebanon, Oman, Ghana, Kenya, Nigeria, Saudi Arabia, Turkey
Pfizer

Active, not recruiting

A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

Sickle Cell Disease | Vaso-occlusive Pain Episode in Sickle Cell Disease | Vaso-occlusive Crisis

United States, Oman, Colombia, Brazil, Lebanon, Nigeria, Germany, Italy, Kenya, Saudi Arabia, Tanzania, Turkey
Pfizer

Terminated

A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

Sickle Cell Disease | Vaso-occlusive Pain Episode in Sickle Cell Disease | Vaso-occlusive Crisis

United States, Oman, France, Brazil, Colombia, Lebanon, Nigeria, Germany, Italy, Kenya, Saudi Arabia, Turkey, United Kingdom, Zambia
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
Children's Oncology Group
National Cancer Institute (NCI)

Recruiting

Study of Kidney Tumors in Younger Patients

Renal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditions

United States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Study of Blood Samples From Newborns With Down Syndrome

Myeloid Proliferations Associated With Down Syndrome

United States, Canada, Australia, Puerto Rico
Reprixys Pharmaceutical Corporation
National Heart, Lung, and Blood Institute (NHLBI); Food and Drug Administration...

Completed

Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises (SUSTAIN)

Sickle Cell Disease

United States, Brazil, Jamaica
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
Otsuka Pharmaceutical Development & Commercialization...

Recruiting

A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

United States, Belgium, Spain, Germany, United Kingdom, Poland
Actelion

Terminated

Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH)

Pulmonary Hypertension

United States, France, United Kingdom, Netherlands

Clinical Trials on Myopathy Congenital in Colombia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Bahamas

Conditions

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Drug Interventions

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