Clinical Trials on Genetic Anticipation in Italy

Total 1216 results

University Hospital Padova

Recruiting

Water and Electrolytes Content in HYpertension (WHYSKI) in the SKIn (WHYSKI)

Hypertension | Essential Hypertension | Hyperaldosteronism | Aldosteronism | Conn Adenoma | Aldosterone Disorder | Aldosterone-producing Adenoma | Bilateral Adrenal Hyperplasia

Italy
Bellicum Pharmaceuticals

Terminated

Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Lymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Leukemia, Acute Myeloid (AML), Child

Italy
Mallinckrodt ARD LLC

Terminated

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy (BRAVE)

Muscular Dystrophy, Duchenne

Spain, Bulgaria, Italy, United States, Mexico, Israel, Serbia, Turkey
CSL Behring

Completed

A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

Hemophilia B

Germany, Australia, Italy, Israel, Czech Republic, Spain, France, Canada, Austria, Russian Federation
Dr. Reddy's Laboratories Limited
PharmaNet

Completed

A Safety and Efficacy Study of DRL-17822, a Cholesteryl Ester Transfer Protein (CETP) Inhibitor, in Patients With Abnormal Cholesterol Levels

Type II Hyperlipidemia

Ukraine, Poland, Italy
PTC Therapeutics
Genzyme, a Sanofi Company

Terminated

Study of Ataluren (PTC124) in Hemophilia A and B

Hemophilia A | Hemophilia B

United States, Canada, France, Italy
Bayer

Active, not recruiting

Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study) (LIFE-ACTIVE)

Hemophilia A

Canada, Spain, United States, Belgium, Italy, Taiwan, Slovenia
International Registry of Asymptomatic Brugada...

Withdrawn

Empiric Quinidine for Asymptomatic Brugada Syndrome

Arrhythmia

Netherlands, Israel, United States, Japan, Germany, Italy
Octapharma

Completed

A Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL Administered With Two Different Delivery Devices: Injections Using Pump or Rapid Push

Primary Immunodeficiency

United Kingdom, Australia, Germany, Italy
TWi Biotechnology, Inc.

Recruiting

An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)

Generalized Epidermolysis Bullosa Simplex

United States, Taiwan, Australia, Philippines, Austria, Italy, Poland
Azienda Socio Sanitaria Territoriale degli Spedali...

Completed

Transcranial Alternating Current Stimulation (tACS) in Patients With Ataxia (SCAtACS)

Ataxia | Multiple System Atrophy | Spinocerebellar Ataxias | Ataxia With Oculomotor Apraxia | CANVAS

Italy
Universitaire Ziekenhuizen KU Leuven
KU Leuven; Vertex Pharmaceuticals Incorporated; University of Lisbon

Withdrawn

Organoid Study R334W

Cystic Fibrosis

Spain, Italy, France, Belgium, Portugal
aTyr Pharma, Inc.

Completed

Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Netherlands, France, Italy
University of L'Aquila

Unknown

Evaluation of Menses in Congenital Bleeding Disorders (MEVA-3)

Von Willebrand Disease | Congenital Coagulation Factors Deficiency | Haemophilia Carriers

Italy
H. Lundbeck A/S

Completed

Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia

Friedreich's Ataxia

Germany, Austria, Italy
Celgene

Terminated

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.

Beta Thalassemia Intermedia | Beta Thalassemia Major

France, United Kingdom, Italy, Greece
Fondazione Telethon

Unknown

Natural History Study in Subjects With Usher Syndrome

Usher Syndrome, Type 1B

Italy, Netherlands, Spain
Grifols Therapeutics LLC
Instituto Grifols, S.A.

Completed

Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Participants With Congenital Afibrinogenemia (IG0902)

Congenital Afibrinogenemia

India, United States, Italy, Lebanon
Advice Pharma Group srl

Recruiting

KidneYou - Innovative Digital Therapy

Diabetes | Chronic Kidney Diseases | Tuberous Sclerosis | Albuminuria | Treatment Adherence | Arterial Hypertension | Treatment Adherence and Compliance | Nephronophthisis | Polycystic Kidney | Chronic Glomerulonephritis | Cystic Kidney Diseases | Cortical Cystic Disease | Cystic Diseases

Italy
Pfizer

Completed

Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B

Hemophilia B

United States, Spain, Croatia, Serbia, Russian Federation, Canada, Hungary, Italy, Romania
CSL Behring

Completed

Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency

Common Variable Immunodeficiency | Agammaglobulinemia

Canada, Germany, Italy, Spain
Cooperative International Neuromuscular Research...

Unknown

Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

Becker Muscular Dystrophy

United States, Canada, Italy, United Kingdom
Second Sight Medical Products

Terminated

Argus® II Retinal Prosthesis System Post-Market Surveillance Study

Retinitis Pigmentosa | Outer Retinal Degeneration

Germany, Italy
Northwestern University

Completed

Impact of the Severity of Pediatric Psoriasis on Childhood Body Mass Index

Psoriasis | Atopic Dermatitis

Chile, United States, Netherlands, Canada, Hong Kong, Italy
Azienda Socio Sanitaria Territoriale degli Spedali...

Completed

Cerebello-Spinal tDCS as Rehabilitative Intervention in Neurodegenerative Ataxia (SCA02)

Ataxia | Multiple System Atrophy | Spinocerebellar Ataxias | Spinocerebellar Ataxia 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Ataxia With Oculomotor Apraxia | CANVAS

Italy
BioMarin Pharmaceutical
Syneos Health

Recruiting

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding (PALomino)

Phenylketonuria, Maternal

Germany, United States, Italy
Ente Ospedaliero Ospedali Galliera

Completed

Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia (PAH2010)

Pulmonary Arterial Hypertension | Thalassemia Major | Thalassemia Intermedia

Italy
aTyr Pharma, Inc.

Completed

Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD) (FSHD)

Facioscapulohumeral Muscular Dystrophy | Limb Girdle Muscular Dystrophy

United States, Denmark, Italy
University Hospital Freiburg

Completed

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond Syndrome

Spain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
Univar BV

Unknown

Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Wilson Disease

United Kingdom, Germany, Greece, Italy
Newron Pharmaceuticals SPA

Terminated

Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms (STARS)

Rett Syndrome

United States, India, United Kingdom, Australia, Italy
Terumo BCTbio
United States Department of Defense; U.S. Army Medical Research and Development... and other collaborators

Terminated

Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE) (PRAISE)

Transfusion Dependent Thalassemia

United States, Israel, Italy, Turkey
Azienda Socio Sanitaria Territoriale degli Spedali...

Completed

Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia (CStDCSAtaxia)

Cerebellar Ataxia | Multiple System Atrophy | Spinocerebellar Ataxias | Spinocerebellar Degenerations | Spinocerebellar Ataxia 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Ataxia, Spinocerebellar | Ataxia, Cerebellar | Ataxia With Oculomotor Apraxia

Italy
Wave Life Sciences Ltd.

Terminated

Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

Duchenne Muscular Dystrophy

United States, Belgium, United Kingdom, Czechia, Canada, France, Italy, Sweden
Octapharma

Not yet recruiting

Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery

Hemophilia A

Spain, France, Finland, Germany, Italy, Serbia
Pfizer

Terminated

A Study of Crisaborole Ointment 2%; Crisaborole Vehicle; TCS and TCI in Subjects Aged ≥ 2 Years, With Mild-moderate AD

Atopic Dermatitis

United States, Poland, Germany, United Kingdom, Italy, Sweden, Switzerland
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sickle Cell Disease | Hemoglobinopathies | Hematological Diseases | Hydroxyurea Failure | Hydroxyurea Intolerance

United States, Italy, United Kingdom, Germany
Amicus Therapeutics

Available

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Pompe Disease Infantile-Onset

Taiwan, United States, Italy
Mario Negri Institute for Pharmacological Research

Completed

Somatostatin In Patients With Autosomal Dominant Polycystic Kidney Disease And Moderate To Severe Renal Insufficiency (ALADIN2)

Autosomal Dominant Polycystic Kidney Disease

Italy
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Beta-Thalassemia

France, United Kingdom, United States, Thailand, Italy, Germany
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors (PRESent-3)

Hemophilia B | Hemophilia B With Inhibitor

United States, Spain, Australia, Taiwan, France, Turkey, United Kingdom, Italy, Armenia, India, Germany
Sarepta Therapeutics, Inc.

Active, not recruiting

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)

Duchenne Muscular Dystrophy

United States, Belgium, Spain, United Kingdom, Netherlands, Germany, Canada, Italy
CSL Behring

Completed

A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

Hemophilia B

United States, Japan, Israel, Austria, Bulgaria, France, Germany, Italy, Russian Federation, Spain
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

Spain, United States, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
ApoPharma
Food and Drug Administration (FDA)

Completed

Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

Pantothenate Kinase-Associated Neurodegeneration

United States, Germany, Italy, United Kingdom
Virginia Commonwealth University

Recruiting

DMCRN-02-001: Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)

Congenital Myotonic Dystrophy | Childhood Myotonic Dystrophy | CDM | CHDM

United States, Italy
ApoPharma

Completed

Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration (TIRCON-EXT)

Pantothenate Kinase-Associated Neurodegeneration

United States, Germany, Italy, United Kingdom
Medexus Pharma, Inc.

Completed

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

Hemophilia B

United States, France, United Kingdom, India, Israel, Italy, Poland
PTC Therapeutics

Completed

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Dystrophinopathy

United Kingdom, Canada, Germany, Spain, Sweden, Italy, Australia, Belgium, France, Israel
BioMarin Pharmaceutical

Completed

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Batten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease

United States, Italy, Germany, United Kingdom

Clinical Trials on Genetic Anticipation in Italy

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Angola

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations