Search clinical trials for: voretigene neparvovec-rzyl

Total 37 results

Spark Therapeutics

Active, not recruiting

Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Inherited Retinal Dystrophy Due to RPE65 Mutations
Spark Therapeutics, Inc.
Children's Hospital of Philadelphia; University of Iowa

Active, not recruiting

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Leber Congenital Amaurosis | Inherited Retinal Dystrophy Due to RPE65 Mutations

United States
Spark Therapeutics

Active, not recruiting

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Leber Congenital Amaurosis

United States
Spark Therapeutics

Completed

Safety Study in Subjects With Leber Congenital Amaurosis

Leber Congenital Amaurosis

United States
Spark Therapeutics

Active, not recruiting

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

United States
Novartis Pharmaceuticals

Active, not recruiting

Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Japan
Freeline Therapeutics

Terminated

Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

Hemophilia B

United States, United Kingdom
Novartis Gene Therapies
PRA Health Sciences

Completed

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Spinal Muscular Atrophy Type I

Korea, Republic of, Taiwan, Japan
Novartis Gene Therapies

Terminated

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy (STRONG)

Spinal Muscular Atrophy

United States
Novartis Gene Therapies
PRA Health Sciences

Completed

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)

Spinal Muscular Atrophy

United States, United Kingdom, Australia, Belgium, Canada, Japan
Novartis Gene Therapies

Completed

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 (STR1VE)

Gene Therapy | SMA - Spinal Muscular Atrophy

United States
Novartis Pharmaceuticals

Recruiting

A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma (ARISER)

Spinal Muscular Atrophies

Brazil
Novartis Gene Therapies

Active, not recruiting

Long-Term Follow-up Study for Patients From AVXS-101-CL-101 (START)

Spinal Muscular Atrophy 1

United States
Novartis Gene Therapies

Active, not recruiting

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type III

United States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
Novartis Gene Therapies

Completed

Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 (STRIVE-EU)

SMA

France, United Kingdom, Italy, Belgium
Raziel Therapeutics Ltd.

Completed

An Open Label Study That Will Test Safety and Efficacy of RZL-012 Injection Into the Submental Fat in Chinese vs. Non-Chinese Subjects.

Submental Fat

United States
Raziel Therapeutics Ltd.

Completed

An Open-label Study That Will Test a Second Treatment Session of RZL-012.

Submental Fat

United States
Raziel Therapeutics Ltd.

Completed

Treatment of Women With Lipedema Involving Substantial Fat Above the Knee or of Women and Men With Nodular Dercum's Disease

Dercum Disease | Lipedema

United States
Raziel Therapeutics Ltd.
University of Arizona

Completed

Treatment of Women With Lipedema Involving Substantial Fat Knee of Women and Men With Nodular Dercum's Disease

Dercum Disease | Lipedema

United States
Raziel Therapeutics Ltd.
Spaulding Clinical Research LLC

Completed

Evaluation of Safety, Efficacy and Thermogenesis-induction of RZL-012 in Overweight and Obese Volunteers

Obesity | Weight Loss | Overweight and Obesity

United States
Raziel Therapeutics Ltd.

Completed

A Single Blind, Randomized, Placebo-controlled, Phase 2a, 2-cohort Study for the Evaluation of Safety and Efficacy of RZL-012 for Submental Fat Reduction in Healthy Volunteers.

Submental Fat

United States
Raziel Therapeutics Ltd.

Completed

Evaluation of Safety and Efficacy of RZL-012 in Subjects Seeking Fat Reduction in the Flanks

Flank Mass

United States
Raziel Therapeutics Ltd.

Completed

Efficacy and Safety of RZL-012 on Submental Fat Reduction

Submental Fat

United States
Raziel Therapeutics Ltd.

Completed

RZL-012 for Dercum's Disease Lipomas

Dercum's Disease Lipomas

United States
Novartis Pharmaceuticals

Recruiting

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)

Spinal Muscular Atrophy (SMA)

Spain, Canada, Taiwan, Belgium, Malaysia, China, Japan, Italy, Singapore, United Kingdom, France, Australia, Thailand, Denmark, United States
GenSight Biologics

Active, not recruiting

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT)

Leber Hereditary Optic Neuropathy

Belgium, Spain, United States, France, United Kingdom, Taiwan, Italy
GenSight Biologics

Completed

RESCUE and REVERSE Long-term Follow-up (RESTORE)

Leber Hereditary Optic Neuropathy

United States, France, Italy, United Kingdom, Germany
Raziel Therapeutics Ltd.

Completed

Evaluation the Effects of Various Treatments on RZL-012-Induced Adverse Events

Submental Fat

United States
GenSight Biologics

Completed

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, France, Germany, Italy
GenSight Biologics

Completed

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) (REVERSE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, Germany, Italy, France
Active Biotech AB

Completed

ABR-217620/Naptumomab Estafenatox With Interferon-alpha (IFN-alpha) Compared to IFN-alpha Alone in Patients With Advanced Renal Cell Carcinoma

Renal Cell Carcinoma

Romania, Russian Federation, United Kingdom, Ukraine, Bulgaria
Amsterdam Molecular Therapeutics
International Antiviral Therapy Evaluation Center

Unknown

Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X]

Familial Lipoprotein Lipase Deficiency

Canada
Ajou University School of Medicine
Ilsung Pharmaceuticals CO.,LTD.

Completed

Comparison of the Effects of Dexmedetomidine and Propofol on the Cardiovascular Autonomic Nervous System During Spinal Anesthesia (ANS)

Spinal Anesthesia | Autonomic Nervous System

Korea, Republic of
UniQure Biopharma B.V.
Chiesi Farmaceutici S.p.A.

Withdrawn

Alipogene Tiparvovec for the Treatment of LPLD Patients

LPL Deficiency

Canada, United States
Mercy Health Ohio

Terminated

Noninferiority Comparison of Prophylactic Open Fracture Antimicrobial Regimens

Open Fracture | Post-Op Wound Infection

United States
Amsterdam Molecular Therapeutics
The Clinical Trial Company

Completed

Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects

Familial Lipoprotein Lipase Deficiency

Canada
University of Washington
National Cancer Institute (NCI)

Completed

FLARE RT for Patients With Stage IIB-IIIB Non-small Cell Lung Cancer: Personalizing Radiation Therapy Using PET/CT and SPECT/CT Imaging

Stage IIB Lung Non-Small Cell Carcinoma AJCC v7 | Stage IIIA Lung Non-Small Cell Cancer AJCC v7 | Stage IIIB Lung Non-Small Cell Cancer AJCC v7

United States

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