Search clinical trials for: DTX401

Total 47 results

UNION therapeutics

Completed

Study to Evaluate Safety & Efficacy of Topical ATx201 OINTMENT in Adolescents and Adults With Mild to Moderate AD

Atopic Dermatitis

Bulgaria, Denmark, Poland
UNION therapeutics

Completed

Study Evaluating the Safety, Tolerability, PK, and Activity of Topical ATx201 Gel in Outpatients With Impetigo

Impetigo

South Africa
UNION therapeutics

Completed

A Study to Evaluate the Irritation Potential of ATx201 in Healthy Volunteers

Irritation Potential of Topic Agent

United States
UNION therapeutics

Completed

Sensitization Study of ATx201 in Healthy Volunteers

Sensitivity, Contact

United States
Bayer
Ultragenix pharmaceutical

Active, not recruiting

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".

Hemophilia A

Netherlands, United States, Germany, United Kingdom, France, Bulgaria
UNION therapeutics

Completed

A Study to Evaluate the Safety and Efficacy of ATx201 in Subjects With Moderate Atopic Dermatitis

Atopic Dermatitis

Canada
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)

Dravet Syndrome

Australia
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome (ENDEAVOR)

Dravet Syndrome

United States
Coeptis Therapeutics
Fred Hutchinson Cancer Center

Completed

Allogeneic Natural Killer (NK) Cell Therapy in Subjects Hospitalized for COVID-19

COVID-19 Pneumonia

United States
Alexion Pharmaceuticals

Completed

Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

Wilson Disease

United States, Germany, Austria, Poland, United Kingdom
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (UK Only)

Dravet Syndrome

United Kingdom
Xilio Development, Inc.

Recruiting

XTX301 in Patients With Advanced Solid Tumors

Advanced Solid Tumor

United States
Daewon Pharmaceutical Co., Ltd.

Completed

To Evaluate the Efficacy and Safety of DW-1401 in Acute and Chronic Gastritis Patients (2)

Gastritis Acute | Gastritis Chronic
Ultragenyx Pharmaceutical Inc

Completed

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

GSD1

Netherlands, United States, Canada, Spain
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
AlgoTherapeutix

Recruiting

Efficacy and Safety of ATX01 in Adult Patients With CIPN (Chemotherapy-induced Peripheral Neuropathy) (ACT)

Chemotherapy-induced Peripheral Neuropathy

United States, Spain, Belgium, France, Czechia, Poland, Italy
Ultragenyx Pharmaceutical Inc

Terminated

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Hemophilia B

United States, United Kingdom, Bulgaria
Coeptis Therapeutics
Duke University

Recruiting

Safety and Efficacy of Allogeneic NK Cell Infusions in Patients With Relapsed/Refractory AML and High Risk MDS

MDS | AML, Adult Recurrent

United States
Alexion Pharmaceuticals

Completed

Copper Balance in Healthy Participants Administered ALXN1840

Healthy

United Kingdom
Alexion

Completed

Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

Wilson Disease

United States, New Zealand, United Kingdom
Centre of Clinical Pharmacology, Hanoi Medical...

Not yet recruiting

Efficacy and Safety of DT01 Tablets in Patients With Irritable Bowel Syndrome With Diarrhea

Irritable Bowel Syndrome With Diarrhea

Vietnam
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
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
UNION therapeutics

Completed

Evaluation of ATx201 as a Topical Antibiotic Agent

Infection, Bacterial

Austria
Vertex Pharmaceuticals Incorporated

Not yet recruiting

Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Sickle Cell Disease
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
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

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

United States, Germany, Italy, United Kingdom, Canada
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
Alexion Pharmaceuticals, Inc.

Terminated

Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Wilson Disease

Spain, Korea, Republic of, France, Germany, Japan, Australia, Poland, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency (CAPtivate)

Ornithine Transcarbamylase (OTC) Deficiency

United Kingdom, United States, Canada, Spain
Xilio Development, Inc.
Hoffmann-La Roche

Recruiting

XTX101 Monotherapy and XTX101 and Atezolizumab Combination Therapy in Patients With Advanced Solid Tumors

Advanced Solid Tumor

United States
Clinica Universidad de Navarra, Universidad de...
DNAtrix, Inc.

Completed

Virus DNX2401 and Temozolomide in Recurrent Glioblastoma (D24GBM)

Glioblastoma Multiforme | Recurrent Tumor

Spain
Tigenix S.A.U.

Completed

Efficacy and Safety of Adipose Stem Cells to Treat Complex Perianal Fistulas Not Associated to Crohn´s Disease (FATT1)

Anal Fistula

Spain, Germany, United Kingdom
M.D. Anderson Cancer Center
DNAtrix, Inc.

Recruiting

MSC-DNX-2401 in Treating Patients With Recurrent High-Grade Glioma

Recurrent Glioblastoma | Recurrent Malignant Glioma | Recurrent Gliosarcoma | Recurrent Anaplastic Astrocytoma | IDH1 wt Allele

United States
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Glycogen Storage Disease Type IA

United States, Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain
Tigenix S.A.U.

Completed

Long-term Safety and Efficacy of Adipose-derived Stem Cells to Treat Complex Perianal Fistulas in Patients Participating in the FATT-1 Randomized Controlled Trial (LTE)

Complex Perianal Fistula

Spain
Tigenix S.A.U.

Terminated

Safety and Efficacy of Adipose-Derived Stem Cells to Treat Complex Perianal Fistulas Patients With Crohn's Disease (FATT)

Crohn Disease | Complex Perianal Fistula

Spain, Netherlands, Austria
DNA Therapeutics

Completed

DNA Repair Inhibitor & Irradiation on Melanoma (DRIIM)

Local Metastatic Melanoma

France
Ultragenyx Pharmaceutical Inc

Recruiting

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

OTC Deficiency

United States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
Alexion

No longer available

Early Access Program for ALXN1840 in Patients With Wilson Disease

Wilson Disease
Alexion

Completed

Study of Oral ALXN1840 at 2 Dose Strengths in Healthy Adults

Healthy

United Kingdom
Alexion

Terminated

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

Wilson Disease

United States, France, Germany, Spain, Canada, Singapore, Poland, Denmark, Hungary, Australia, Austria, Czechia, Hong Kong, Israel, Japan, Korea, Republic of, New Zealand, Russian Federation, Serbia, Taiwan, Turkey, United Kingdom
Alexion

Completed

A Study of ALXN1840 (Non-coated) Administered With And Without Omeprazole In Healthy Adults

Healthy

United States
Alexion
PPD; ERT: Clinical Trial Technology Solutions

Completed

A Study of the Cardiac Effects of ALXN1840 in Healthy Adults

Healthy

United States
Alexion

Completed

A Study of ALXN1840 (Coated and Non-coated) Administered With And Without Omeprazole In Healthy Adults

Healthy

United States
Alexion

Completed

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

Wilson Disease

United States
Alexion

Completed

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

Wilson Disease

United States

Search clinical trials for: DTX401

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