Clinical Trials Sponsored by ProQR Therapeutics

Total 6223 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Withdrawn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
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
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
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

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
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
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
CRISPR Therapeutics AG
ViaCyte

Recruiting

An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D

Glucose Metabolism Disorders | Immune System Diseases | Autoimmune Diseases | Diabetes Mellitus | Endocrine System Diseases | Diabetes Mellitus, Type 1 | Metabolic Disease

Canada
iCell Gene Therapeutics
iCar Bio Therapeutics

Not yet recruiting

Anti-CD33 CAR-T Cells for the Treatment of Relapsed/Refractory CD33+ Acute Myeloid Leukemia

High Risk Hematologic Malignancies | Relapsed and/or Refractory Acute Myeloid Leukemia

China
Melinta Therapeutics, LLC

Recruiting

Open-Label, Dose-Finding, Pharmacokinetics, Safety and Tolerability Study of Oritavancin in Pediatric Patients With Suspected or Confirmed Bacterial Infections

Gram Positive Bacterial Infections

United States
TenNor Therapeutics Limited

Completed

TNP-2092 to Treat Acute Bacterial Skin and Skin Structure Infection (P2_ABSSSI)

Gram-Positive Bacterial Infections | Skin and Subcutaneous Tissue Bacterial Infections

United States
CRISPR Therapeutics AG
ViaCyte

Completed

An Open-Label, FIH Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With T1D

Glucose Metabolism Disorders | Immune System Diseases | Autoimmune Diseases | Diabetes Mellitus | Endocrine System Diseases | Diabetes Mellitus, Type 1 | Metabolic Disease

Canada
BlackThorn Therapeutics, Inc.
Neumora Therapeutics, Inc.

Completed

Study in Major Depressive Disorder With BTRX-335140 (NMRA-335140) vs Placebo

Major Depressive Disorder

United States
Sirion Therapeutics, Inc.
ReVision Therapeutics, Inc.

Completed

Study of Fenretinide in the Treatment of Geographic Atrophy Associated With Dry Age-Related Macular Degeneration

Geographic Atrophy | Dry Age Related Macular Degeneration
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

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
Verismo Therapeutics

Recruiting

Long-Term Follow-Up Safety Study of Subjects Treated With SynKIR-110 in Cancer Studies

Cancer

United States
Ractigen Therapeutics.

Recruiting

A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy

Non-Muscle-Invasive Bladder Cancer (NMIBC)

Australia
NeuShen Therapeutics

Not yet recruiting

First-into-human Study of NS-136 in Healthy Subjects

Healthy Subjects

Australia
Monopar Therapeutics

Recruiting

Diagnostic Efficacy and Dosimetry of MNPR-101-DFO*-89Zr in Patients With Solid Tumors

Gastric Cancer | Colorectal Cancer | Pancreatic Cancer | Ovarian Cancer | Lung Cancer | Bladder Cancer | Urothelial Carcinoma | Triple-negative Breast Cancer | Solid Tumor, Adult

Australia
vTv Therapeutics

Not yet recruiting

Cadisegliatin as Adjunctive Therapy in Type 1 Diabetes (CATT1)

Diabetes Mellitus, Type 1
Enliven Therapeutics

Recruiting

ELVN-002 With Trastuzumab +/- Chemotherapy in HER2+ Solid Tumors, Colorectal and Breast Cancer

Colorectal Cancer | HER2-positive Breast Cancer | HER2-positive Gastric Cancer | HER2 Amplification | HER2 Positive Solid Tumors

United States
OX2 Therapeutics

Recruiting

CD200AR-L and Allogeneic Tumor Lysate Vaccine Immunotherapy for Recurrent HGG and Newly Diagnosed DMG/DIPG in Children and Young Adults

Diffuse Midline Glioma, H3 K27M-Mutant | Recurrent High Grade Glioma

United States
Transcend Therapeutics

Recruiting

A Single, Ascending Dose Evaluation of the Safety, Pharmacokinetics of Methylone in Healthy Subjects

Post Traumatic Stress Disorder

Australia
Remix Therapeutics

Not yet recruiting

Study of REM-422 in Patients With AML or Higher Risk MDS

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Acute Myeloid Leukemia Refractory | Higher Risk Myelodysplastic Syndromes

United States
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
Dianthus Therapeutics

Recruiting

A Phase 2 Study to Evaluate DNTH103 in Adults With Generalized Myasthenia Gravis (MAGIC) (MAGIC)

Myasthenia Gravis, Generalized

United States
Intellia Therapeutics

Not yet recruiting

Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002

Hereditary Angioedema

France, New Zealand, United Kingdom
Aprea Therapeutics

Not yet recruiting

Study of APR-1051 in Patients With Advanced Solid Tumors

Advanced Solid Tumor
ReCode Therapeutics

Recruiting

A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants

Cystic Fibrosis

New Zealand

Clinical Trials Sponsored by ProQR Therapeutics

Sponsors and Collaborators

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CROs in Indonesia

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