Phase 2 N=9 Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Alpha-1 Antitrypsin Deficiency
Primary: Frequency of Grade 3 or 4 Adverse Events — 0; 0; 1 participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=3 AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Duchenne Muscular Dystrophy
Primary: Number of Participants With Unacceptable Toxicity. — 0 Participants
Phase 2 N=11 Gene Therapy for Achromatopsia (CNGA3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Phase 2 N=12 Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Leber Congenital Amaurosis
Primary: Number of Participants Experiencing Ocular or Non-ocular Adverse Events — 6; 6 participants
Phase 2 N=10 MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
Phase 2 N=12 ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Phase 2 N=36 Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
Adenosine Deaminase Deficiency · Severe Combined Immunodeficiencies (SCID)
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 100; 100; 100 percentage of participants
Phase 2 N=13 Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Pompe Disease
Primary: Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. — 40,031; 29,638; 5,509,882; 1,907,161 mU/mL
Phase 2 N=6 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
Alpha 1-Antitrypsin Deficiency
Primary: Treatment-emergent Adverse Events Related to ADVM-043 — 0; 1; 2 Participants
Phase 1 N=12 Safety Study of Adenovirus/PNP Coupled With Fludarabine Phosphate to Treat Solid Tumors
Head and Neck Cancer
Primary: Number of Participants With Side Effects After Ad/PNP-F-araAMP Treatment — 12; 8; 8; 5 participants
Phase 2 N=15 Gene Therapy Study in Severe Haemophilia A Patients (270-201)
Severe Haemophilia A
Primary: Number of Participants With Treatment Emergent Adverse Events — 1; 1; 6; 7 Participants
Phase 2 N=46 Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
ADA-SCID
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 85.71; 100; 92.31 percentage of participants
Phase 1 N=9 Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Alpha 1-Antitrypsin Deficiency
Primary: Adverse Events Possibly, Probably or Definitely Related to Study Drug — 2; 2; 3; 2 participants
Phase 1 N=6 Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Retinal Disease · Retinitis Pigmentosa
Primary: Systemic and Ocular Safety — 0; 0; 1; 0 Participants
Phase 2 N=15 Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Leber Congenital Amaurosis
Primary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
Phase 2 N=2 Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Number of Unanticipated Grade III or Higher Treatment-Related Toxicities — 0; 0 events
Phase 2 N=33 Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
Fabry Disease
Primary: Incidence of Treatment-emergent Adverse Events (TEAEs) - All — 2; 2; 3; 2 Participants
Phase 2 N=15 Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
Choroideremia · CHM (Choroideremia) Gene Mutations
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 5; 5; 5 Participants
Phase 2 N=14 A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
Parkinson's Disease
Primary: Incidence of Adverse Events Related to the Treatment — 0; 1; 0 Participants
Phase 2 N=42 Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial
Neovascular Age-related Macular Degeneration · Wet Age-related Macular Degeneration
Primary: Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)) — 6; 6; 6; 12 Participants
Phase 2 N=3 Dose Confirmation Trial of AAV5-hFIXco-Padua
Hemophilia B
Primary: Factor IX Activity Levels — 30.6 Factor IX activity (%)
Phase 2 N=23 Gene Therapy for Achromatopsia (CNGB3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Phase 3 N=201 Study to Assess the Safety and Efficacy of Brincidofovir in Treatment of Early Versus Late Adenovirus Infection
Adenovirus Infection
Primary: Number of Participants With All-Cause Mortality — 9; 27; 8 Participants
Phase 2 N=3 Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Number of Dose Limiting Toxicity (DLT) Adverse Events as Assessed by 21 CFR 312.32. — 0 Number of Events
Phase 2 N=45 Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
X-Linked Retinitis Pigmentosa
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to…
Phase 2 N=10 Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
Hemophilia B
Primary: Number of Participants With Adverse Events — 5; 5 participants
Phase 1 N=28 Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
Leber's Hereditary Optic Neuropathy
Primary: Number of Treatment Related Adverse Events — 1; 1; 1; 5 Participants
Phase 2 N=27 Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked Retinoschisis
Primary: Number of Participants Experiencing Adverse Events — 6; 7; 12 Participants
Phase 3 N=67 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Hemophilia B
Primary: Annualized Bleeding Rate (ABR) for All Bleeding Episodes — 4.19; 1.51 bleeds/year/participants
Phase 3 N=126 Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
Type 2 Spinal Muscular Atrophy
Primary: Change From Baseline at the End of Period 1 in the Hammersmith Functional Motor Scale Expanded - Total Score - in the ≥ 2 to < 18 Years Age Group — 2.39; 0.51 Scores on…