Clinical Trials on Congenital Myotonic Dystrophy in Italy

Total 578 results

InMed Pharmaceuticals Inc.

Completed

INM-755 (Cannabinol) Cream for Treatment of Epidermolysis Bullosa

Epidermolysis Bullosa Dystrophica | Epidermolysis Bullosa Simplex | Epidermolysis Bullosa, Junctional | Kindler Syndrome

France, Germany, Greece, Israel, Italy
Merck Sharp & Dohme LLC

Active, not recruiting

A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)

United States, Australia, Brazil, Italy, United Kingdom
Boehringer Ingelheim

Recruiting

A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome

Netherton Syndrome

Belgium, United Kingdom, Australia, Germany, China, Malaysia, United States, Japan, France, Israel, Bulgaria, Italy, Finland, Switzerland, Netherlands, Portugal
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, United Kingdom, Canada, Germany, Italy
Shire

Completed

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

Beta-thalassemia | Transfusional Iron Overload

United States, United Kingdom, Italy, Thailand, Turkey
Sydney Children's Hospitals Network
Johns Hopkins University; Children's Hospital of Philadelphia; University of... and other collaborators

Unknown

Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)

Charcot-Marie-Tooth Disease

United States, Australia, Italy
Shire

Terminated

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

Beta-Thalassemia | Transfusional Iron Overload

United States, Lebanon, Canada, Italy, Turkey
Igenomix

Recruiting

RCT Study to Validate niPGT-A Clinical Benefit. (niPGT-A_RCT)

Infertility | Aneuploidy | Chromosome Abnormality

France, Argentina, Spain, United States, Brazil, Italy
Albireo

Completed

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)

Alagille Syndrome

United States, United Kingdom, France, Germany, Canada, Netherlands, Italy, Malaysia, Belgium, Israel, New Zealand, Poland, Turkey
Agios Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-Kids)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

United States, Italy, Germany, Saudi Arabia
SECRETO Study Consortium
Sahlgrenska University Hospital, Sweden; Academy of Finland; Hospital District... and other collaborators

Recruiting

Searching for Explanations for Cryptogenic Stroke in the Young: Revealing the Etiology, Triggers, and Outcome (SECRETO)

Ischemic Stroke | Thrombosis | Brain Infarction | Foramen Ovale, Patent

Italy, Estonia, Finland, Germany, Lithuania, Netherlands, Norway, Sweden, Turkey, United Kingdom, Greece, Portugal, Spain
Vifor (International) Inc.
Labcorp Corporation of America Holdings, Inc

Completed

Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia (VITHAL)

Beta-Thalassemia | Non-transfusion-dependent Thalassemia

Greece, Israel, Italy, Lebanon, Thailand
Società Italiana Talassemie ed Emoglobinopatie

Completed

Hydroxyurea in the Treatment of Sickle Cell Disease

Sickle Cell Disease

Italy
Novo Nordisk A/S

Completed

Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain, Italy, Greece, Germany, United Kingdom
BioMarin Pharmaceutical

Recruiting

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia

United States, Spain, Canada, France, Japan, Australia, Italy, Argentina, Brazil, Germany, United Kingdom
Zimmer Biomet

Recruiting

A Prospective, Clinical Investigation of the Vanguard 360 Revision Knee

Osteoarthritis | Rheumatoid Arthritis | Complications; Arthroplasty | Traumatic Arthritis | Deformity of Limb | Post Traumatic Deformity

Italy, United States, Belgium, Denmark, France, Germany, Spain, United Kingdom
Abbott Medical Devices

Recruiting

AMPLATZER PFO Occluder Post Approval Study (PFO PAS)

Stroke | Patent Foramen Ovale

United States, Germany, Canada, Switzerland, Italy, Portugal, United Kingdom
Albireo

Enrolling by invitation

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia (BOLD-EXT)

Biliary Atresia

Spain, United States, Netherlands, Malaysia, Canada, Germany, Turkey, Italy, France, Australia, Poland, New Zealand
Novartis Pharmaceuticals

Completed

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Myelofibrosis | Sickle Cell Disease | Thalassemia | Diamond Blackfan Anemia

United States, Netherlands, Taiwan, Thailand, Italy, Germany, Spain, Canada, Belgium, Greece, Turkey, United Kingdom
Ionis Pharmaceuticals, Inc.
Biogen

Active, not recruiting

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

Angelman Syndrome

United States, Israel, Italy, France, Australia, United Kingdom
Pfizer

Completed

Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease

Phase 1 Sickle Cell

United States, United Kingdom, Belgium, Netherlands, Italy
Abbott Medical Devices

Recruiting

Trevisio Post-Approval Study (TrevisioPAS)

PFO - Patent Foramen Ovale | VSD - Muscular Ventricular Septal Defect | PIVSD - Post Infarct Muscular Ventricular Septal Defect | ASD - Atrial Septal Defect

Spain, Germany, Poland, France, Italy, Netherlands, Switzerland
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Sickle Cell Disease | Hemoglobinopathies | Hematological Diseases

United States, Belgium, Canada, France, Germany, Italy, United Kingdom
Igenomix

Completed

Multi-center Study to Validate niPGT-A (niPGT-A)

Infertility | Aneuploidy | Chromosome Abnormality

Mexico, Turkey, United States, Argentina, Spain, Brazil, Italy, Peru
Agios Pharmaceuticals, Inc.

Active, not recruiting

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Pyruvate Kinase Deficiency

United States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
Hoffmann-La Roche

Active, not recruiting

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome

Angelman Syndrome

United States, Spain, Netherlands, Italy
Novartis Pharmaceuticals

Completed

A Dose Escalation/Expansion Study of LDK378 in Patients With Tumors Characterized by Genetic Abnormalities in Anaplastic Lymphoma Kinase

Tumors Characterized by Genetic Abnormalities of ALK

Australia, Belgium, Germany, Netherlands, Spain, Italy, Singapore, Korea, Republic of, United Kingdom, United States, Canada
AstraZeneca

Completed

A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease (Hestia2)

Sickle Cell Disease

United States, Italy, France, United Kingdom, Turkey, Egypt, Lebanon, Kenya
Agios Pharmaceuticals, Inc.

Completed

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
Pfizer

Completed

Study Evaluating 13-valent Pneumococcal Conjugate Vaccine (13vPnC) in Children With Sickle Cell Disease

Pneumococcal Conjugate Vaccine

France, United States, United Kingdom, Lebanon, Italy, Egypt, Saudi Arabia
Novo Nordisk A/S

Completed

Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

France, United States, Israel, Spain, Switzerland, Germany, Canada, Japan, United Kingdom, Italy, Finland, Austria
Translational Research Center for Medical Innovation...

Recruiting

International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases

Neutral Lipid Storage Disease

China, United States, France, Austria, Germany, Italy, Japan, Netherlands, United Kingdom
C. R. Bard

Completed

Lutonix® Global AV Registry Investigating Lutonix Drug Coated Balloon for Treatment of Native and Synthetic AV Fistulae (AVR)

Arteriovenous Fistula

United Kingdom, Singapore, France, Italy, Germany, Switzerland, Greece, Austria, Poland, Portugal, Saudi Arabia, Taiwan, Turkey
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
Dr. Rebecca Schule
German Federal Ministry of Education and Research; German Center for Neurodegenerative...

Recruiting

Phenotypes, Biomarkers and Pathophysiology in Hereditary Spastic Paraplegias and Related Disorders (HSP-PBP)

Hereditary Spastic Paraplegia

Austria, Germany, Italy
Edimer Pharmaceuticals

Completed

Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (ECP-002)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
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
bluebird bio

Active, not recruiting

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Transfusion-dependent Beta-Thalassemia

United States, France, United Kingdom, Australia, Thailand, Italy, Germany, Greece
rEVO Biologics

Completed

Assess Incidence of Deep Vein Thrombosis(DVT)Following Administration of Recombinant Human Antithrombin (rhAT) to Hereditary Antithrombin(AT) Deficient Patients in High Risk Situations. (rhAT)

Antithrombin Deficiency, Congenital

France, United Kingdom, Germany, United States, Sweden, Italy
Edimer Pharmaceuticals

Active, not recruiting

Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002 (ECP-002e)

X-linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
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
EspeRare Foundation
Iqvia Pty Ltd; Pierre Fabre Medicament

Recruiting

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (EDELIFE)

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

United States, Spain, France, Germany, Italy, United Kingdom
Edimer Pharmaceuticals

Completed

Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia (ECP-015)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
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
Agios Pharmaceuticals, Inc.

Recruiting

Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)

Pyruvate Kinase Deficiency

United States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
Zogenix, Inc.
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Active, not recruiting

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Dravet Syndrome | Epileptic Encephalopathy | Lennox Gastaut Syndrome

United States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Mexico, Netherlands, Poland, Spain, Sweden, United Kingdom
Bristol-Myers Squibb

Recruiting

A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (α)-Thalassemia

Anemia

Thailand, China, Canada, Turkey, Greece, Taiwan, Italy, Hong Kong
Hoffmann-La Roche

Recruiting

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD). (CROSSWALK-a)

Sickle Cell Disease

United States, Brazil, Italy, Spain, Kenya, Netherlands, United Kingdom, France, South Africa
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Enrolling by invitation

A Long-term Follow-up Study in Participants Who Received CTX001

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

United States, Canada, Italy, United Kingdom, Germany

Clinical Trials on Congenital Myotonic Dystrophy in Italy

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