Clinical Trials on Genetic Diseases, X-Linked in United States

Total 7967 results

Takeda

Completed

Efficacy and Safety of Oral Febuxostat in Participants With Gout (CONFIRMS)

Gout

United States
Novartis Pharmaceuticals

Active, not recruiting

A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP

Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive

United States, China, Hungary, Japan, Belgium, Germany, Canada, Singapore, Australia, Israel, Netherlands, Spain, Italy, Korea, Republic of, Bulgaria, France, United Kingdom, Czechia, Sweden, India, Malaysia, Denmark, Taiwan, Norway, Fi... and more
VA Office of Research and Development

Completed

CSP594 Comparative Effectiveness in Gout: Allopurinol vs. Febuxostat

Chronic Kidney Diseases | Gout

United States
University of Nottingham

Completed

Doxycycline In Lymphangioleiomyomatosis (LAM)

Tuberous Sclerosis | Lymphangioleiomyomatosis

United Kingdom
Otsuka Pharmaceutical Development & Commercialization...

Completed

Tolvaptan Extension Study in Participants With ADPKD (TEMPO 4/4)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Australia, Belgium, Germany, Italy, Poland, Romania, United Kingdom, Argentina, Canada, France, Netherlands, Russian Federation
KalVista Pharmaceuticals, Ltd.

Completed

A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, Romania, Slovakia, Spain, United Kingdom
Takeda
Takeda Development Center Americas, Inc.

Completed

A Study of Lanadelumab in Teenagers and Adults to Prevent Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)

Angioedema

United States, Spain, Netherlands, Hungary, Italy, Canada, Germany, France, Japan, Poland
University of Alabama at Birmingham
Pacific Pediatric Neuro-Oncology Consortium; Array BioPharma

Active, not recruiting

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas (NF108-BINI)

Neurofibromatosis Type 1 | Plexiform Neurofibroma

United States
Biohaven Pharmaceuticals, Inc.

Active, not recruiting

Troriluzole in Adult Subjects With Spinocerebellar Ataxia

Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8

United States, China
LeMaitre Vascular
Factory CRO for Medical Devices B.V.

Recruiting

Prospective and Non-randomized Registry of CardioCel 3D

Intracardiac and Septal Defects | Valve and Anulus Repair | Great Vessel Reconstruction | Peripheral Vessel Reconstruction | Suture Line Buttressing

Germany, Italy, Spain, United Kingdom
Cancer Prevention Pharmaceuticals, Inc.

Completed

Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)

Familial Adenomatous Polyposis

United States, Belgium, Germany, Canada, Netherlands, Spain, United Kingdom
Jazz Pharmaceuticals

Completed

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Seizures | Tuberous Sclerosis Complex

United States, Spain, Australia, Poland, United Kingdom, Netherlands
Pfizer
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Compare Bosutinib To Imatinib In Subjects With Newly Diagnosed Chronic Phase Philadelphia Chromosome Positive CML

Chronic Myeloid Leukemia

France, United States, Spain, Belgium, Singapore, Poland, Canada, Japan, United Kingdom, Taiwan, Hungary, Germany, Argentina, Russian Federation, Turkey, Colombia, Italy, South Africa, Brazil, Chile, China, Hong Kong, India, Korea, Republic... and more
Novartis Pharmaceuticals

Completed

A Phase I Study of Oral Asciminib (ABL001) in Patients With CML or Ph+ ALL

Chronic Myelogenous Leukemia | Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia

Germany, Australia, Netherlands, Spain, Korea, Republic of, France, Japan, Italy, Singapore, United States
KalVista Pharmaceuticals, Ltd.

Terminated

A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II (KVD824-201)

Angioedema, Hereditary, Types I and II

United States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico
Bristol-Myers Squibb

Completed

Study of BMS-354825 (Dasatinib) in Patients With Chronic Myeloid Leukemia Who Are Either Resistant or Intolerant to Imatinib

Chronic Myeloid Leukemia | Philadelphia-Positive Myeloid Leukemia

Belgium, Netherlands, Spain, United States, Italy, Canada, France, Germany, Peru, Singapore, United Kingdom, Australia, Sweden, Finland, Israel, Ireland, Denmark, Korea, Republic of, Norway, Austria, South Africa, Switzerland
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Bristol-Myers Squibb

Completed

Dasatinib (BMS-354835) Versus Imatinib Mesylate in Subjects With Chronic Myeloid Leukemia

Chronic Myeloid Leukemia | Philadelphia-Positive Myeloid Leukemia

Belgium, Spain, United States, Italy, Argentina, Brazil, Canada, Germany, Peru, Puerto Rico, Singapore, South Africa, Taiwan, United Kingdom, Australia, France, Philippines, Sweden, Thailand, Russian Federation, China, Poland, Finland, Isra... and more
Pfizer

Completed

Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)

Transthyretin Amyloidosis | Transthyretin Gene Mutations

United States, France, Spain, Taiwan, Israel, Korea, Republic of, Denmark, Canada, Netherlands, Japan, Sweden, Brazil, Mexico, Argentina, Portugal, Belgium, Bulgaria, Cyprus, Germany, Italy, Malaysia, Romania, Saudi Arabia, Turkey, United...
Intellia Therapeutics

Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

United Kingdom, New Zealand, Sweden
KalVista Pharmaceuticals, Ltd.

Recruiting

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Bulgaria, France, Greece, Hungary, Israel, Italy, Netherlands, New Zealand, North Macedonia, Poland, Spain, United Kingdom, Australia, Germany, South Africa, Austria, Portugal, Slovakia, Romania, Canada, Saudi Arabia, Japa...
CHDI Foundation, Inc.

Recruiting

Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort

Huntington's Disease

United States, Canada, Belgium, Spain, Australia, Germany, France, Netherlands, United Kingdom, New Zealand, Argentina, Austria, Chile, Colombia, Denmark, Ireland, Italy, Poland, Portugal, Switzerland
City of Hope Medical Center

Active, not recruiting

Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor

Unspecified Childhood Solid Tumor, Protocol Specific | Solid Tumor | Ewing Sarcoma | Unspecified Adult Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Neuroblastoma | Recurrent Adult Soft Tissue Sarcoma | Recurrent Adult Brain Tumor | Recurrent... and other conditions

United States
Northwestern University

Completed

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

Neurofibromatosis Type 1 | Neurofibromatosis Type 2 | Adult Ependymoma | Recurrent Adult Brain Tumor | Acoustic Schwannoma | Adult Anaplastic Meningioma | Adult Grade I Meningioma | Adult Grade II Meningioma | Adult Meningeal Hemangiopericytoma | Adult Papillary Meningioma

United States
Sponsor GmbH
GlaxoSmithKline; iOMEDICO AG; Arbeitsgemeinschaft fur Internistische Onkologie; Arbeitskreis Klinische Studien

Terminated

Dose Finding Study for Combination of Capecitabine, Lapatinib and Vinorelbine in Metastatic Breast Cancer (CELAVIE)

Metastatic Breast Cancer | HER2 Positive | First or Second Line Therapy | Failure or Contraindication of Trastuzumab Therapy

Germany
Orphalan
Ergomed

Recruiting

International Wilson's Disease Patient Registry (iWilson Registry)

Wilson's Disease

Spain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
University of Alabama at Birmingham
Medtronic; The Children's Tumor Foundation

Terminated

A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1) (NF107-BMP2)

NF1 | Congenital Pseudarthrosis of Tibia

United States, Australia
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
National Cancer Institute (NCI)

Completed

Flavopiridol in Treating Children With Relapsed or Refractory Solid Tumors or Lymphomas

Unspecified Childhood Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Recurrent Childhood Soft... and other conditions

United States
Imperial College London
Royal Brompton & Harefield NHS Foundation Trust; St George's, University of... and other collaborators

Recruiting

Low QRS Voltages in Young Healthy Individuals and Athletes

Dilated Cardiomyopathy | Sudden Cardiac Death | Sudden Cardiac Death Due to Cardiac Arrhythmia | Sudden Cardiac Arrest | Arrhythmogenic Right Ventricular Cardiomyopathy | Arrhythmogenic Left Ventricular Cardiomyopathy

United Kingdom
Novartis Pharmaceuticals

Completed

Safety and Efficacy of Imatinib Versus Interferon-α Plus Cytarabine in Patients With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia

Chronic Myelogenous Leukemia

Canada, Italy, United States, United Kingdom, Belgium, Germany, France, Switzerland, Austria, Denmark, Spain, Norway, Sweden, Netherlands, New Zealand, Australia
Imperial College London

Recruiting

Multiple Breath Washout, a Clinimetric Dataset

Bronchiectasis | Sleep Disorder | Asthma | Cystic Fibrosis | Bronchitis | Primary Ciliary Dyskinesia

United Kingdom
Scholar Rock, Inc.

Active, not recruiting

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX (ONYX)

Neuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Anti-myostatin | Spinal Muscular Atrophy Type II

United States, Spain, Belgium, Germany, Netherlands, Poland, Italy, France, United Kingdom
National Cancer Institute (NCI)

Completed

Sorafenib Tosylate in Treating Patients With Metastatic, Locally Advanced, or Recurrent Medullary Thyroid Cancer

Multiple Endocrine Neoplasia Type 2A | Multiple Endocrine Neoplasia Type 2B | Recurrent Thyroid Gland Medullary Carcinoma | Hereditary Thyroid Gland Medullary Carcinoma | Locally Advanced Thyroid Gland Medullary Carcinoma | Sporadic Thyroid Gland Medullary Carcinoma | Stage III Thyroid Gland Medullary... and other conditions

United States
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Germany, Switzerland, Macedonia, The Former Yugoslav Republic of, Serbia, Taiwan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Israel, Russian Federation, Turkey, Lithuania, Poland, Japan, Latvia, Puerto...
Shire

Completed

A European Post-Authorisation Observational Study Of Patients With Hereditary Angioedema

Hereditary Angioedema (HAE)

Germany, Spain, United Kingdom, France, Italy, Belgium
National Cancer Institute (NCI)
Multicenter Hemophilia Cohort Study

Completed

The Second Multicenter Hemophilia Cohort Study

Hepatocellular Carcinoma | Non-Hodgkin Lymphoma | Liver Decompensation

United States, Germany, Italy, Brazil, Canada, France, Greece, Sweden
University of Washington, the Collaborative Health...
Cystic Fibrosis Foundation

Recruiting

Colorectal Cancer Screening in Cystic Fibrosis (NICE-CF)

Colorectal Cancer | Adenoma | Cystic Fibrosis

United States
Immunovia, Inc.

Completed

A Study of IMMray™ PanCan-d Test for Early Detection of Pancreatic Cancer in High-risk Groups

Pancreatic Ductal Adenocarcinoma | Familial Pancreatic Cancer | FAMMM - Familial Atypical Mole Malignant Melanoma Syndrome

United States, Sweden, Canada, Spain, United Kingdom
Gilead Sciences

Completed

Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF (AZLI CAT)

Cystic Fibrosis

United States
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects (guardian™1)

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Germany, Switzerland, Serbia, Taiwan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Japan, Denmark, Hungary, Israel, Turkey, Russian Federation, Former Serbia and Montenegro
Sanofi

Completed

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Sphingomyelin Lipidosis

Italy, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
Otsuka Pharmaceutical Development & Commercialization...

Completed

Long Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant

Belgium, Germany, Italy, Spain, Sweden, United States, Canada, Australia, Netherlands, Czechia, Poland, Denmark, Israel, Hungary, Russian Federation, South Africa, United Kingdom, Romania, Argentina, Norway
Emalex Biosciences Inc.

Completed

Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension (D1AMOND)

Tourette Syndrome in Children | Tourette Syndrome in Adolescence

United States, Poland, France, Canada, Germany
Guy's and St Thomas' NHS Foundation Trust

Completed

Reliability of Functional Outcome Measures in Neurofibromatosis 2

Neurofibromatosis 2

United Kingdom
NewAmsterdam Pharma

Active, not recruiting

Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies (BROADWAY)

Dyslipidemias | Hypercholesterolemia | Familial Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | High Cholesterol

United States, Poland, Denmark, Czechia, China, Netherlands, Japan, Georgia
Genzyme, a Sanofi Company

Recruiting

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I)

Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
Alnylam Pharmaceuticals

Active, not recruiting

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
CSL Behring
Chiltern International Inc.

Completed

Patient Registry Study of Berinert® in Normal Clinical Practice

Includes: Hereditary Angioedema

United States, Denmark, Germany, Switzerland
Progenity, Inc.

Completed

Expanded Noninvasive Genomic Medical Assessment: The Enigma Study

Down Syndrome | Aneuploidy | DiGeorge Syndrome | Turner Syndrome | Klinefelter Syndrome | Chromosome Deletion | Edwards Syndrome | Patau Syndrome

United States

Clinical Trials on Genetic Diseases, X-Linked in United States

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Iran

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations