Clinical Trials on Hurler Syndrome (MPS I)

Total 799 results

Genentech, Inc.

Completed

A Study to Evaluate Subjects With Turner Syndrome Treated With Growth Hormone

Turner Syndrome
Seattle Children's Hospital
Eli Lilly and Company

Unknown

Turner Syndrome Prenatal Diagnosis Study

Turner Syndrome

United States
Thomas Jefferson University
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

The Effect of Androgen and Growth Hormone on Height and Learning in Girls With Turner Syndrome

Turner Syndrome

United States
Ferring Pharmaceuticals

Completed

An Open-Label, Multi-centre, Phase III Study of Local Tolerability of ZOMACTON 10MG

Turner's Syndrome

Czech Republic, France, Netherlands
University of Aarhus

Unknown

Long Term Follow-up of Bone Mineral Density in Hormone Treated Turner Syndrome

Turner Syndrome
Eli Lilly and Company

Completed

The Effect of Growth Hormone in Very Young Girls With Turner Syndrome

Turner Syndrome

United States
Noema Pharma AG

Recruiting

Basimglurant in Children, Adolescents, and Young Adults With TSC

Tuberous Sclerosis Complex

United States, India, Israel, Turkey, Italy, Poland, Spain, United Kingdom
Nobelpharma

Active, not recruiting

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC

Tuberous Sclerosis Complex

Japan
Eunice Kennedy Shriver National Institute of Child...

Terminated

Studies of Autistic Patients: Gene Networks and Clinical Subtypes

Tuberous Sclerosis | Autism

United States
Katarzyna Kotulska

Recruiting

Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants (ViRap)

Tuberous Sclerosis Complex

Poland
University Hospitals Bristol and Weston NHS Foundation...
University College, London; The Tuberous Sclerosis Association

Active, not recruiting

Acceptance and Commitment Therapy in Tuberous Sclerosis Complex (ACT in TSC)

Tuberous Sclerosis Complex

United Kingdom
University of California, Los Angeles
Boston Children's Hospital

Completed

Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis Complex

Tuberous Sclerosis Complex

United States
University of Pennsylvania
H. Lundbeck A/S

Terminated

Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients (STARS)

Complex Partial Seizures

United States
Children's Hospital Medical Center, Cincinnati
Massachusetts General Hospital; The University of Texas Health Science Center... and other collaborators

Completed

Tuberous Sclerosis Complex Natural History Study: Renal Manifestations

Tuberous Sclerosis Complex

United States
Case Comprehensive Cancer Center
National Cancer Institute (NCI)

Completed

Dose Monitoring of Busulfan and Combination Chemotherapy in Hodgkin or Non-Hodgkin Lymphoma Undergoing Stem Cell Transplant

Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma | Recurrent Adult Diffuse Small Cleaved Cell Lymphoma and other conditions

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

Completed

Stem Cell Transplantation for Patients With Cancers of the Blood

Acute Lymphocytic Leukemia | MDS (Myelodysplastic Syndrome) | CLL (Chronic Lymphocytic Leukemia) | CML (Chronic Myelogenous Leukemia) | AML (Acute Myelogenous Leukemia)

United States
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
Shire

Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
Green Cross Corporation

Completed

A Study of GC1111 in Hunter Syndrom Patients

Hunter Syndrome

Korea, Republic of
Shire

Completed

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Novo Nordisk A/S

Completed

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

Turner Syndrome | Genetic Disorder

Spain
Green Cross Corporation

Unknown

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Hunter Syndrome

Korea, Republic of
Centre Hospitalier Universitaire Dijon
Novartis Pharmaceuticals

Recruiting

Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP) (SESAM)

Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)

France
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
CENTOGENE GmbH Rostock

Completed

Biomarker for Hereditary AngioEdema Disease (BioHAE)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | C1 Esterase Inhibitor Deficiency | HAE | Angio Edema | C4 Deficiency | Hereditary Angioedema Type III

Turkey, Armenia, Georgia, India, Peru, Poland, Romania
National Cancer Institute (NCI)

Enrolling by invitation

Biology Studies of Hematologic Cancers

Acute Lymphoblastic Leukemia | Acute Myelogenous Leukemia | Non-Hodgkins Lymphoma | Myelodysplastic Syndrome (MDS) | Hodgkins Lymphoma

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Withdrawn

HIV-Resistant Gene Modified Stem Cells and Chemotherapy in Treating Patients With Lymphoma With HIV Infection

Stage III Adult Hodgkin Lymphoma | Stage IV Adult Hodgkin Lymphoma | Human Immunodeficiency Virus 1 Positive | Stage I Adult Hodgkin Lymphoma | Stage II Adult Hodgkin Lymphoma | Stage I Adult Non-Hodgkin Lymphoma | Stage II Adult Non-Hodgkin Lymphoma | Stage III Adult Non-Hodgkin Lymphoma | Stage IV...

United States
Sidney Kimmel Comprehensive Cancer Center at Johns...
Janssen Research & Development, LLC

Not yet recruiting

Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

Lymphoma | Multiple Myeloma | Hodgkin Lymphoma | Myelofibrosis | Non Hodgkin Lymphoma | Chronic Myeloid Leukemia | Hematologic Malignancy | Aplastic Anemia | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML) | Bone Marrow Transplant Rejection | Acute Lymphoblastic Leukemia (ALL), Adult
Takeda

Recruiting

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

Hunter Syndrome

India
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Novo Nordisk A/S

Completed

Safety of Somatropin and Induction of Puberty With 17-beta-oestradiol in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

Netherlands
Ferring Pharmaceuticals

Terminated

Evaluation of the Adherence and the Patient Acceptability of Zomacton® Treatment With the Zomajet® Vision X Device (ZOMAXEPT)

Growth Hormone Deficiency | Turner's Syndrome

France
Green Cross Corporation

Completed

Safety and Efficacy of Hunterase (GC1111)

Hunter Syndrome

Korea, Republic of
Eli Lilly and Company

Completed

Somatropin Treatment to Final Height in Turner Syndrome (GDCT)

Turner Syndrome

Canada
Jazz Pharmaceuticals

Recruiting

A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex (EpiCom)

Tuberous Sclerosis Complex Associated Neuropsychiatric Disease

United States, United Kingdom, Poland
Universiteit Antwerpen
University Hospital, Antwerp

Recruiting

Gait in Rare Diseases (GAGA)

Tuberous Sclerosis | STXBP1 Encephalopathy With Epilepsy

Belgium
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Not yet recruiting

A Clinical Trial to Evaluate the Tolerability and Pharmacokinetics of TQ-B3234 in Patients With Type I Neurofibromatosis

Type I Neurofibromatosis

China
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
Boston Children's Hospital
National Institute of Neurological Disorders and Stroke (NINDS); Tuberous Sclerosis...

Completed

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

Tuberous Sclerosis Complex

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

Completed

Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

Tuberous Sclerosis | Angiofibromas

United States, Australia
Dana-Farber Cancer Institute
Novartis Pharmaceuticals

Completed

Everolimus for Cancer With TSC1 or TSC2 Mutation

Tuberous Sclerosis Complex | TSC1 | TSC2 | MTOR

United States
Shire

Completed

Hunter Outcome Survey (HOS) (HOS)

Hunter Syndrome

United States
Shire

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Hunter Syndrome

United States, United Kingdom
Assistance Publique - Hôpitaux de Paris
Groupe Hospitalier Pitie-Salpetriere

Active, not recruiting

Cryopreservation of Ovarian Cortex in Girls With Turner Syndrome (CRYO-X0)

Turner Syndrome | Ovarian Insufficiency

France
Children's Hospital Medical Center, Cincinnati
National Center for Advancing Translational Sciences (NCATS)

Completed

Risk of Diabetes in Young Turner Syndrome Patients

Turner Syndrome

United States
Novo Nordisk A/S

Completed

Growth Response in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

France
Novo Nordisk A/S

Completed

Use of Somatropin in Turner Syndrome

Turner Syndrome | Genetic Disorder

United Kingdom
University of Aarhus

Unknown

Aortic Dimensions in Turner Syndrome

Turner Syndrome
University of California, Los Angeles

Recruiting

Remote Assessment and Intervention for Behavior Problems in Kids With TSC (RAINBOW)

Tuberous Sclerosis

United States

Clinical Trials on Hurler Syndrome (MPS I)

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Mali

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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