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Results for “Single-gene Disorders”

419 trials

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Showing 20 of 419 results

Early research (Phase 1)Study completedNCT02286817
What this trial is testing

Phase I Single Dose, Open-Label Pharmacokinetic Study and Single-Blind, Placebo-Controlled Dose Escalation Study of NFC-1 in Adolescents With Attention-Deficit Hyperactivity Disorder

Who this might be right for
Attention-deficit Hyperactivity Disorder (ADHD)
Aevi Genomic Medicine, LLC, a Cerecor company 30
Testing effectiveness (Phase 2)Ended earlyNCT06716853
What this trial is testing

A Clinical Gene Therapy Study With Hematopoietic Stem Cells for the Treatment, With Single Dose of Temferon, of Patients Suffering From Metastatic Renal Cell Carcinoma

Who this might be right for
Clear Cell RCC
Genenta Science 10
Testing effectiveness (Phase 2)Study completedNCT01828021
What this trial is testing

Phase 2 Study of the Monoclonal Antibody MGAH22 (Margetuximab) in Patients With Relapsed or Refractory Advanced Breast Cancer

Who this might be right for
Breast Cancer
MacroGenics 25
Large-scale testing (Phase 3)Active Not RecruitingNCT05606614
What this trial is testing

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

Who this might be right for
Rett Syndrome
Taysha Gene Therapies, Inc. 17
Not applicableStudy completedNCT01199393
What this trial is testing

Genetic Polymorphisms Predict Chemotherapeutic Outcomes in Patients With Metastatic Breast Cancer

Who this might be right for
Breast NeoplasmDrug TherapyPolymorphism,Single Nucleotide
Peking University Cancer Hospital & Institute 153
Testing effectiveness (Phase 2)Looking for participantsNCT05747261
What this trial is testing

Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

Who this might be right for
Spinal Muscular Atrophy (SMA)
Biocad 40
Large-scale testing (Phase 3)WithdrawnNCT06297486
What this trial is testing

Study of a Gene Therapy Treatment for Hemophilia A

Who this might be right for
Hemophilia A
Spark Therapeutics, Inc.
Testing effectiveness (Phase 2)Looking for participantsNCT05824169
What this trial is testing

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Who this might be right for
Spinal Muscular Atrophy
GeneCradle Inc 18
Early research (Phase 1)Study completedNCT01579383
What this trial is testing

Safety Tolerability and Pharmacokinetics of ALD403

Who this might be right for
Migraine Disorders
Alder Biopharmaceuticals, Inc. 104
Not applicableUnknownNCT05523128
What this trial is testing

The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.

Who this might be right for
Hemophilia A
Institute of Hematology & Blood Diseases Hospital, China 6
Very early researchNot Yet RecruitingNCT07685444
What this trial is testing

A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Injection of LY-N001 Injection for the Treatment of Moderate to Advanced Parkinson's Disease With GBA1 Mutations

Who this might be right for
Parkinson's Disease (PD)
Lingyi Biotech Co., Ltd. 18
Testing effectiveness (Phase 2)Looking for participantsNCT06980948
What this trial is testing

Safety and Tolerability Study of ST-503 for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)

Who this might be right for
Chronic Neuropathic Pain
Sangamo Therapeutics 27
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07818304
What this trial is testing

SRT Combined With Envafolimab and Endostar in the First-line Treatment of Advanced NSCLC

Who this might be right for
Non Small Cell Lung CancerLung Neoplasms
The First People's Hospital of Lianyungang 61
Not applicableLooking for participantsNCT06840717
What this trial is testing

Association Between Genetic Polymorphisms and Type 2 Asthma in Children

Who this might be right for
Asthma in ChildrenGenetic DiseaseInflammation
Guangzhou Institute of Respiratory Disease 300
Testing effectiveness (Phase 2)Looking for participantsNCT07053462
What this trial is testing

CRISPR-Edited HLA Donor Kidney Transplant to Reduce Rejection Risk

Who this might be right for
End-Stage Renal DiseaseEnd Stage Renal Disease on DialysisEnd Stage Renal Disease With Renal Transplant+4 more
AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC 90
Not applicableStudy completedNCT07750197
What this trial is testing

Exercise and Diet Responses in Adult Men With Obesity by MC4R Genotype and Metabolic Health

Who this might be right for
ObesityMetabolically Healthy ObesityMetabolically Unhealthy Obesity
Harbin Sport University 40
Large-scale testing (Phase 3)Study completedNCT03370913
What this trial is testing

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Who this might be right for
Hemophilia A
BioMarin Pharmaceutical 144
Testing effectiveness (Phase 2)Study completedNCT01867333
What this trial is testing

Enzalutamide With or Without Vaccine Therapy for Advanced Prostate Cancer

Who this might be right for
Prostate Cancer
National Cancer Institute (NCI) 57
Testing effectiveness (Phase 2)Looking for participantsNCT06220487
What this trial is testing

A Single-arm, Open-label Study of Olverembatinib, CD3/CD19 Bispecific T-cell Engager, and Chidamide in Patients With Newly Diagnosed Ph+ALL

Who this might be right for
Acute Lymphoblastic LeukemiaPhiladelphia ChromosomePhiladelphia-Positive ALL+2 more
Nanfang Hospital, Southern Medical University 67
Testing effectiveness (Phase 2)Looking for participantsNCT07673809
What this trial is testing

Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
AO GENERIUM 32
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