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Results for “Adeno-Associated Virus (AAV)”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Ended earlyNCT00076557
What this trial is testing

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

Who this might be right for
Hemophilia B
Avigen 15
Testing effectiveness (Phase 2)Study completedNCT02053064
What this trial is testing

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
Testing effectiveness (Phase 2)Study completedNCT02341807
What this trial is testing

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Who this might be right for
ChoroideremiaCHM (Choroideremia) Gene Mutations
Spark Therapeutics, Inc. 15
Testing effectiveness (Phase 2)Looking for participantsNCT07630649
What this trial is testing

Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration

Who this might be right for
Neovascular Age-Related Macular Degeneration (nAMD) Wet AMD
Adverum Biotechnologies, Inc. 15
Testing effectiveness (Phase 2)Looking for participantsNCT05836259
What this trial is testing

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

Who this might be right for
Hypertrophic Cardiomyopathy
Tenaya Therapeutics 30
Not applicableStudy completedNCT04560933
What this trial is testing

Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A

Who this might be right for
Hemophilia A
BioMarin Pharmaceutical 186
Not applicableStudy completedNCT02971969
What this trial is testing

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Who this might be right for
Hemophilia B
Ultragenyx Pharmaceutical Inc 6
Large-scale testing (Phase 3)Study completedNCT05139316
What this trial is testing

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

Who this might be right for
Glycogen Storage Disease Type IA
Ultragenyx Pharmaceutical Inc 49
Testing effectiveness (Phase 2)Active Not RecruitingNCT07174726
What this trial is testing

Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

Who this might be right for
X-Linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics 10
Testing effectiveness (Phase 2)Study completedNCT03734588
What this trial is testing

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Who this might be right for
Adeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, Inherited+14 more
Spark Therapeutics, Inc. 4
Early research (Phase 1)Active Not RecruitingNCT02496273
What this trial is testing

Phase I Trial of CEA Specific AAV-DC-CTL Treatment in Stage IV Gastric Cancer

Who this might be right for
Gastric Cancer
The First People's Hospital of Changzhou 60
Early research (Phase 1)Ended earlyNCT01620801
What this trial is testing

Hemophilia B Gene Therapy With AAV8 Vector

Who this might be right for
Hemophilia B
Spark Therapeutics, Inc. 4
Testing effectiveness (Phase 2)Ended earlyNCT00643890
What this trial is testing

Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease

Who this might be right for
Parkinson's Disease
Neurologix, Inc. 44
Early research (Phase 1)Study completedNCT00430768
What this trial is testing

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

Who this might be right for
Alpha 1-Antitrypsin Deficiency
University of Massachusetts, Worcester 9
Not applicableStudy completedNCT02302690
What this trial is testing

Immunity Against AAV in Crigler Najjar Patient

Who this might be right for
Crigler Najjar Syndrome
Genethon 49
Testing effectiveness (Phase 2)UnknownNCT02935517
What this trial is testing

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

Who this might be right for
Achromatopsia
Beacon Therapeutics 24
Large-scale testing (Phase 3)Active Not RecruitingNCT05345171
What this trial is testing

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

Who this might be right for
OTC Deficiency
Ultragenyx Pharmaceutical Inc 37
Testing effectiveness (Phase 2)Ended earlyNCT02618915
What this trial is testing

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

Who this might be right for
Hemophilia B
Ultragenyx Pharmaceutical Inc 6
Very early researchStudy completedNCT00377416
What this trial is testing

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

Who this might be right for
Alpha 1-Antitrypsin Deficiency
University of Massachusetts, Worcester 12
Not applicableLooking for participantsNCT07629115
What this trial is testing

A Long-Term Follow-up Study of the Severe Parkinson's Disease Patients Administered the IPS101A Gene Therapy Product.

Who this might be right for
Parkinson DiseaseParkinson's Disease
Innopeutics Corporation 6
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