Clinical Trials Sponsored by ProQR Therapeutics

Total 6330 results

ProQR Therapeutics

Active, not recruiting

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Netherlands, United Kingdom, Germany
ProQR Therapeutics

Terminated

Extension Study to Study PQ-110-001 (NCT03140969) (INSIGHT)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium
ProQR Therapeutics

Withdrawn

Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3 (Fuchs Focus)

Corneal Diseases | Fuchs Endothelial Corneal Dystrophy | FECD3 | LRS | Descemet's Membrane Disorder

United Kingdom
ProQR Therapeutics

Completed

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Stellar)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Canada, France
ProQR Therapeutics

Recruiting

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN)

Eye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

Belgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
ProQR Therapeutics

Terminated

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, United Kingdom
ProQR Therapeutics

Terminated

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (HELIA)

Retinitis Pigmentosa | Usher Syndrome Type 2

United States, Canada, France
ProQR Therapeutics

Completed

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Leber's Congenital Amaurosis

United States, Belgium
ProQR Therapeutics

Active, not recruiting

A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene (AURORA)

Eye Diseases | Retinitis | Eye Diseases, Hereditary | Retinal Dystrophies | Vision Disorders | Retinal Disease | Autosomal Dominant Retinitis Pigmentosa | Vision Tunnel

United States
ProQR Therapeutics

Active, not recruiting

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) (ILLUMINATE)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom
ProQR Therapeutics
European Commission

Completed

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Cystic Fibrosis

United States, France, Belgium
ProQR Therapeutics
European Commission

Completed

Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

Cystic Fibrosis

United States, Spain, United Kingdom, Germany, Italy, Belgium, Canada, Czechia, Denmark, France
Ranok Therapeutics (Hangzhou) Co., Ltd.

Recruiting

A Study of RNK05047 in Subjects With Advanced Solid Tumors/Diffuse Large B-cell Lymphoma (CHAMP-1)

Advanced Solid Tumor | DLBCL

United States
Akebia Therapeutics
Cyclerion Therapeutics

Completed

A Study to Evaluate the Soluble Guanylate Cyclase (sGC) Stimulator IW-1973 in Diabetic Nephropathy / Diabetic Kidney Disease as Measured by Albuminuria

Type 2 Diabetes Mellitus With Diabetic Nephropathy

United States
Akebia Therapeutics
Cyclerion Therapeutics

Completed

A Study of the Effect of IW-1973 on the Exercise Capacity of Patients With Heart Failure With Preserved Ejection Fraction (HFpEF) (CAPACITY-HFpEF)

Heart Failure With Preserved Ejection Fraction

United States, Canada
Eikon Therapeutics
Impact Therapeutics, Inc.

Recruiting

A First-in-human Study of PARP1 Selective Inhibitor, IMP1734, in Participants With Advanced Solid Tumors

Advanced Solid Tumor

Australia, United States
CRISPR Therapeutics AG

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC)

Renal Cell Carcinoma

United States, Australia, Canada, Netherlands
APRINOIA Therapeutics, LLC

Active, not recruiting

A First-in-Human Study to Assess Single Doses of APNmAb005 in Healthy Participants

Alzheimer Disease | Tauopathies | Healthy Volunteers

United States
CRISPR Therapeutics AG

Recruiting

A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed or Refractory Solid Tumors

Clear Cell Renal Cell Carcinoma | Pancreatic Adenocarcinoma | Cervical Carcinoma | Malignant Pleural Mesothelioma | Esophageal Carcinoma

United States
CRISPR Therapeutics AG

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory T or B Cell Malignancies (COBALT-LYM)

T Cell Lymphoma

United States, Australia, Canada
CRISPR Therapeutics AG

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)

B-cell Lymphoma | Non-Hodgkin Lymphoma | B-cell Malignancy | Adult B Cell ALL

United States, Spain, Canada, France, Germany, Australia
Bacainn Therapeutics, Inc.

Completed

A Phase 1, Single-Ascending-Dose Study of BT051 in Healthy Subjects

Healthy Volunteers

United States
Celsus Therapeutics PLC

Terminated

Double-Blind, Trial to Evaluate the Safety and Efficacy of MRX-6 Cream 2% (C012013)

Atopic Dermatitis

Israel
CRISPR Therapeutics AG

Recruiting

A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies

Follicular Lymphoma | B-cell Lymphoma | Mantle Cell Lymphoma | Marginal Zone Lymphoma | Non-Hodgkin Lymphoma | B-cell Malignancy | Large B-cell Lymphoma | Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL)

United States
CRISPR Therapeutics AG

Enrolling by invitation

A Long-term Follow-up Study of Subjects Who Received CRISPR CAR T Cellular Therapies

Hematologic Malignancy | Solid Malignancy

United States, Australia
CRISPR Therapeutics AG

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma

Multiple Myeloma

United States, Spain, Australia, Canada
APRINOIA Therapeutics, LLC

Active, not recruiting

Evaluation of [18F]APN-1607 PET Uptake in Alzheimer's Disease Patients Compared With Healthy Subjects

Healthy Volunteers | Alzheimer's Disease | Mild Cognitive Impairment Due to Alzheimer's Disease

United States
Skyline Therapeutics (US) Inc.

Recruiting

Phase I/II Study of SKG0106 Intravitreal Injection in Patients With Neovascular Age-related Macular Degeneration (nAMD)

Neovascular Age-related Macular Degeneration

China, United States
iCell Gene Therapeutics
iCar Bio Therapeutics

Recruiting

CD7 CAR-T Cell Treatment of Relapsed/Refractory CD7+ T -Acute Lymphoblastic Leukemia/ Lymphoma

Relapsed/Refractory, High Risk Hematologic Malignancies | T-ALL/Lymphoma

China
Arthrosi Therapeutics

Recruiting

Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients

Hyperuricemia | Gout | Arthritis, Gouty | Gout Chronic

United States, Australia, New Zealand
Quanta Therapeutics

Recruiting

QTX3046 in Patients With KRAS G12D Mutations

Advanced Solid Tumor

United States
Highlight Therapeutics

Recruiting

Clinical Trial to Evaluate BO-112 in Patients With Basal Cell Carcinoma (BCC) (SPOTLIGHT204)

Basal Cell Carcinoma

Israel
Neurona Therapeutics

Not yet recruiting

Effect of NRTX-1001, a Cellular Therapy Product, for the Treatment of Refractory Bilateral TLE

Epilepsy, Temporal Lobe
Aliada Therapeutics

Recruiting

A Phase 1 Trial to Assess Safety, Tolerability and Pharmacokinetics of ALIA-1758 in Healthy Participants

Alzheimer Disease

United States
Tisento Therapeutics

Not yet recruiting

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

United States, Australia, Italy, Germany, Canada, United Kingdom
Kyverna Therapeutics

Not yet recruiting

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Systemic Sclerosis

Systemic Sclerosis | Systemic Sclerosis - Diffuse Cutaneous | Systemic Sclerosis - 2013 ACR/EULAR Classification Criteria
Eilean Therapeutics

Recruiting

Study of Single and Multiple Ascending Doses of ZE46-0134 in Healthy Volunteers

AML

Australia
United Therapeutics

Not yet recruiting

DeciPHer-ILD: A Real-world Patient Registry in Group 3 Pulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)

Pulmonary Hypertension | Interstitial Lung Disease | Pulmonary Hypertension Due to Lung Diseases and Hypoxia
Kyverna Therapeutics

Not yet recruiting

A Study of Anti-CD19 CAR T-Cell Therapy, in Subjects With Refractory Primary and Secondary Progressive Multiple Sclerosis

Multiple Sclerosis | Multiple Sclerosis, Secondary Progressive | Multiple Sclerosis, Primary Progressive
Eilean Therapeutics

Not yet recruiting

Dose Escalation and Expansion Study to Evaluate the Safety, PK, PD and Efficacy of ZE46-0134 in Adults With FLT3 Mutated Relapsed or Refractory Acute Myeloid Leukemia

AML With Gene Mutations

Australia
Skyline Therapeutics

Recruiting

A Long-term Follow-up Study to Evaluate SKG0106 in the Treatment of Patients With nAMD

Neovascular (Wet) Age-related Macular Degeneration

China, United States
Starna Therapeutics

Not yet recruiting

A Study to Describe the Safety and Immunogenicity of a Respiratory Syncytial Virus Vaccine STR-V003 in Healthy Adults (STR-V003)

Respiratory Syncytial Virus Infections
Kyverna Therapeutics

Recruiting

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Refractory Lupus Nephritis (KYSA-3)

Lupus Nephritis | Lupus Nephritis - WHO Class IV | Lupus Nephritis - WHO Class III

Germany
Nektar Therapeutics

Recruiting

A Phase 2b Study to Evaluate Rezpegaldesleukin (Rezpeg) in the Treatment of Severe to Very Severe Alopecia Areata in Adult Patients (Rezolve AA)

Alopecia Areata

United States, Canada
Cybrexa Therapeutics

Not yet recruiting

A Study of CBX 12 in Subjects With Platinum Resistant or Refractory Ovarian Cancer

Platinum-resistant Ovarian Cancer | Refractory Ovarian Carcinoma
Cartesian Therapeutics

Recruiting

Descartes-15 for Patients With Relapsed/Refractory Multiple Myeloma (DC15-MM-01)

Refractory Multiple Myeloma | Relapsed Multiple Myeloma

United States
TransCode Therapeutics

Not yet recruiting

Study of TTX-MC138 in Subjects With Advanced Solid Tumors

Advanced Solid Tumor

United States
Valerio Therapeutics

Recruiting

A Phase 1/2 Study of VIO-01 in Participants With Recurrent Solid Tumors

Breast Cancer | Prostate Cancer | Advanced or Metastatic Solid Tumors | Recurrent Ovarian Cancer

United States
Eikon Therapeutics

Recruiting

A Phase 2 Study of EIK1001 in Combination With Pembrolizumab and Chemotherapy in Patients With Stage 4 Non-Small Cell Lung Cancer

NSCLC

United States
Kyverna Therapeutics

Not yet recruiting

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Subjects With Refractory Generalized Myasthenia Gravis

Generalized Myasthenia Gravis | Myasthenia Gravis

Clinical Trials Sponsored by ProQR Therapeutics

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