Clinical Trials on Hereditary Spastic Paraplegia

Total 3536 results

Novartis Pharmaceuticals

Recruiting

A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)

Renal Angiomyolipoma

Taiwan
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Scleral Buckling for Retinal Detachment Prevention in Genetically Confirmed Stickler Syndrome (STL-DR-PREV)

Stickler Syndrome

France
Russian Academy of Medical Sciences
Blokhin's Russian Cancer Research Center

Recruiting

Atypical MOLes and Melanoma Early Detection Study (MoleMed) (MoleMed)

Melanoma | Nevus | Nevus, Pigmented | Melanoma (Skin) | Mucosal Melanoma | Moles | Dysplastic Nevus Syndrome | Nevus, Blue | Nevus, Spitz | Nevi, Spindle Cell | Nevi, Dysplastic | Mucosal Melanosis

Russian Federation
M.D. Anderson Cancer Center

Recruiting

Variables That Are Correlated to Developing Multiple Endocrine Neoplasia (MEN) and Pancreatic Neuroendocrine Tumors (PNET)

Hyperparathyroidism | Multiple Endocrine Neoplasia | Pancreatic Neuroendocrine Tumors

United States
National Cancer Institute (NCI)

Enrolling by invitation

Analysis of Data Collected From Individuals Administered Neurobehavioral Assessments

HIV | Neurofibromatosis Type 1 | Brain Tumors

United States
Assistance Publique - Hôpitaux de Paris

Completed

Vascular Thrombus Involvement in Nephroblastoma (VIN)

Nephroblastoma With Vena Cava Thrombosis

France
HedgePath Pharmaceuticals, Inc.

Completed

Open-label Trial of SUBA™-Itraconazole (SUBA-Cap) in Subjects With Basal Cell Carcinoma Nevus Syndrome (BCCNS)

Basal Cell Carcinoma in Basal Cell Nevus Syndrome

United States
University Hospital, Clermont-Ferrand

Completed

Correlation Between Clinical and Electrophysiological Phenotypes in a Population of Patients With Neuropathy Charcot-Marie-Tooth Disease Type 1A (CCA1)

Charcot-Marie-Tooth Type 1A Neuropathy

France
Bursa Yüksek İhtisas Education and Research Hospital

Completed

Assessment of Central Sensitization, Neuropathic Pain, Sleep Quality and Daily Life Activities in Behcet's Disease

Central Sensitisation | Behcet's Disease

Turkey
M.D. Anderson Cancer Center

Active, not recruiting

Overall and Disease Specific Survival in Patients With Confirmed MEN1 With or Without PNET (Pancreatic Neuroendocrine Tumors)

Genetic Mutation | MEN1

United States
National Cancer Institute (NCI)

Withdrawn

Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)

Neoplasms, Nerve Tissue | Peripheral Nervous System Diseases | Heredodegenerative Disorders, Nervous System | Neurofibromatosis 1
University of California, Davis

Completed

Potential Research Study Participant Registry

Psoriasis | Eczema | Rosacea | Actinic Keratosis | Skin Cancer | Atopic Dermatitis | Acne | Leg Ulcers | Foot Ulcers | Dysplastic Nevi

United States
AstraZeneca

Completed

A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male Participants

Healthy Participants | Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)

United States
M.D. Anderson Cancer Center
Inex Pharmaceuticals

Completed

Liposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies

Soft Tissue Sarcoma | Lymphoma | Leukemia | Osteosarcoma | Wilms' Tumor

United States
Connecticut Children's Medical Center
DuPont Nutrition and Health

Withdrawn

Probiotics and Corticosteroids for Treating Periodic Fever, Aphthous Stomatitis, Pharyngitis, Cervical Adenitis (PFAPA) (PFAPA)

Pharyngitis | Aphthous Stomatitis | Periodic Fever | Cervical Adenitis
Novartis Pharmaceuticals

Withdrawn

Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

Basal Cell Carcinoma

Greece, Switzerland, Germany, United Kingdom, Belgium, Canada, Czechia, Finland, Italy, Spain
Pamela L. Kunz
Genentech, Inc.

Terminated

A Phase II Study of Pertuzumab and Erlotinib for Metastatic or Unresectable Neuroendocrine Tumors

Neuroendocrine Tumors | Neuroblastoma | Multiple Endocrine Neoplasia | Pancreatic Neuroendocrine Tumors | Carcinoid Tumors | Adrenal Gland Tumors

United States
Istituto Ortopedico Rizzoli

Completed

Postoperative Alpha Lipoic Acid in the Carpal Tunnel Syndrome: a Randomized Controlled Trial.

Entrapment Neuropathy, Carpal Tunnel | Compression Neuropathy, Carpal Tunnel | Median Neuropathy, Carpal Tunnel

Italy
Universitaire Ziekenhuizen KU Leuven

Unknown

Ophthalmological Screening and Follow-up of Optic Pathway Gliomas in Children With Neurofibromatosis Type 1.

Neurofibromatosis Type 1

Belgium
QLT Inc.
Novartis

Terminated

Two Studies to Determine if Verteporfin PDT is Effective & Safe in Treating Multiple Basal Cell Carcinoma of the Skin.

Nevoid Basal Cell Carcinoma Syndrome | Basal Cell Carcinoma | Gorlin Syndrome
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Terminated

Therapeutic Allogeneic Lymphocytes and Aldesleukin in Treating Patients With High-Risk or Recurrent Myeloid Leukemia After Undergoing Donor Stem Cell Transplant

Recurrent Adult Acute Myeloid Leukemia | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult... and other conditions

United States
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease (HAPLO-FANCONI)

Fanconi Syndrome
Porteus, Matthew, MD

Recruiting

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

Fanconi Anemia

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

Recruiting

Eltrombopag for People With Fanconi Anemia

Fanconi Anemia

United States
Fred Hutchinson Cancer Center
National Heart, Lung, and Blood Institute (NHLBI); Rocket Pharma Limited

Terminated

Gene Therapy for Fanconi Anemia

Fanconi Anemia

United States
Sanguine Biosciences

Terminated

Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia

Fanconi Anemia

United States
Children's Hospital Medical Center, Cincinnati
Food and Drug Administration (FDA)

Completed

Quercetin in Children With Fanconi Anemia; a Pilot Study

Fanconi Anemia

United States
Julian Sevilla

Completed

Eltrombopag for Treatment of Fanconi Anemia

Fanconi Anemia

Spain
Assistance Publique - Hôpitaux de Paris

Completed

Microcephaly Genetic Deficiency in Neural Progenitors (MICROFANC)

Microcephaly

France
OHSU Knight Cancer Institute
National Cancer Institute (NCI)

Completed

Tissue Sample Collection From Patients With Fanconi Anemia

Unspecified Childhood Solid Tumor, Protocol Specific | Unspecified Adult Solid Tumor, Protocol Specific | Fanconi Anemia

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

Completed

Cord Blood Stem Cell Transplantation Study (COBLT)

Neoplasms | Myelodysplastic Syndromes | Hematologic Diseases | Leukemia | Severe Combined Immunodeficiency | Fanconi Anemia | Anemia, Aplastic | Hematopoietic Stem Cell Transplantation
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Medical Treatment for Diamond Blackfan Anemia

Hematologic Disease | Fanconi's Anemia

United States
Masonic Cancer Center, University of Minnesota

Recruiting

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditions

United States
Gilead Sciences

Completed

Fanconi Syndrome Due to ARVs in HIV-Infected Persons

HIV Infections | Renal Impairment | Fanconi Syndrome | Kidney Disease

Canada, United States
Boston Children's Hospital
National Eye Institute (NEI); Howard Hughes Medical Institute

Recruiting

Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies

Ocular Motility Disorders | Facial Palsy | Synkinesis | Sixth Nerve Palsy | Mobius Syndrome | Congenital Fibrosis of Extraocular Muscles | Duane Retraction Syndrome | Duane Radial Ray Syndrome | Brown Syndrome | Marcus Gunn Syndrome | Strabismus Congenital | Horizontal Gaze Palsy | Horizontal Gaze Palsy With Progressive... and other conditions

United States
Seattle Children's Hospital

Recruiting

EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults

Soft Tissue Sarcoma | Carcinoma | Osteosarcoma | Ewing Sarcoma | Rhabdoid Tumor | Neuroblastoma | Retinoblastoma | Rhabdomyosarcoma | Wilms Tumor | Hepatoblastoma | Synovial Sarcoma | Desmoplastic Small Round Cell Tumor | Germ Cell Tumor | Pediatric Solid Tumor | Clear Cell Sarcoma | Malignant Peripheral Nerve Sheath Tumors

United States
National Cancer Institute (NCI)

Withdrawn

Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study

Hematopoietic and Lymphoid Cell Neoplasm | Lynch Syndrome | Refractory Lymphoma | Refractory Malignant Solid Neoplasm | Recurrent Lymphoma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma | Refractory Neuroblastoma | Recurrent Primary Central Nervous System Neoplasm | Refractory Primary Central... and other conditions

United States, Canada
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

Cerebral Adrenoleukodystrophy (CALD)

United States
University Hospital, Antwerp
Kom Op Tegen Kanker; Stichting tegen Kanker

Recruiting

First-in-human Interleukin-15-transpresenting Wilms' Tumor Protein 1-targeting Autologous Dendritic Cell Vaccination in Cancer Patients (IL15-TransDC)

Esophageal Cancer | Ovarian Cancer | Pancreas Cancer | Liver Cancer

Belgium
National Tuberous Sclerosis Association

Recruiting

TSC Biosample Repository and Natural History Database

Tuberous Sclerosis | Lymphangioleiomyomatosis

United States, Canada
Brigham and Women's Hospital
Massachusetts General Hospital

Recruiting

Feasibility of [11C]Acetate-PET in LAM and TSC

Tuberous Sclerosis Complex | Lymphangioleiomyomatosis

United States
Medical University of Bialystok
University of Bialystok

Recruiting

Diffusion Weighted Magnetic Resonance Imaging and the Optic Nerve Neuropathy.

Optic Neuritis | Ischemic Optic Neuropathy | Traumatic Optic Neuropathy | Toxic Optic Neuropathy | Compressive Optic Neuropathy | Hereditary Optic Neuropathies

Poland
Poxel SA

Not yet recruiting

Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Adrenomyeloneuropathy
Zarife Sahenk

Recruiting

NT-3 Levels and Function in Individuals With CMT

Charcot-Marie-Tooth Disease | Peripheral Neuropathy | Chronic Inflammatory Demyelinating Polyneuropathy

United States
Academisch Medisch Centrum - Universiteit van Amsterdam...
IRCCS Azienda Ospedaliero-Universitaria di Bologna; Hospital Clinic of Barcelona and other collaborators

Recruiting

A Personalized Surveillance and Intervention Protocol for Patients With Familial Adenomatous Polyposis That Have Undergone (Procto)Colectomy

Familial Adenomatous Polyposis

Netherlands
George Zogopoulos

Recruiting

Quebec Pancreas Cancer Study (QPCS)

Pancreatic Cancer | Bile Duct Cancer | Pancreas Cancer | Gallbladder Cancer | Pancreas Neoplasm | Familial Pancreatic Cancer | Duodenal Cancer | Hereditary Cancer | Pancreas Cyst | Ampullary Cancer | Pancreatic Precancerous Condition

Canada
Masonic Cancer Center, University of Minnesota

Recruiting

Adrenoleukodystrophy National Registry Study

Cerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)

United States
National Cancer Institute (NCI)

Completed

Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1

Neurofibromatosis | MPNST

United States
National Cancer Institute (NCI)

Completed

Validating Pain Scales in Children and Young Adults

Leukemia | Solid Tumor | Sickle Cell Disease | Neurofibromatosis

United States
Cellaion SA

Terminated

HepaStem Long-Term Safety Registry (PROLONGSTEM)

Urea Cycle Disorder | Crigler-Najjar Syndrome | Acute on Chronic Liver Failure

Belgium, Spain, France, Bulgaria, Poland

Clinical Trials on Hereditary Spastic Paraplegia

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CROs in Finland

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