Duplication Clinical Trials

1 recruiting

Frequently Asked Questions

Common questions about Duplication clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 112 of 12 trials

Recruiting

Defining the Brain Phenotype of Children With Williams Syndrome

Williams SyndromeDuplication
National Institute of Mental Health (NIMH)415 enrolled1 locationNCT01132885
Recruiting
Phase 1Phase 2

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Ionis Pharmaceuticals, Inc.48 enrolled11 locationsNCT06430385
Recruiting
Phase 2

Gilteritinib Plus VA Followed By Consolidation Chemotherapy in Newly Diagnosed FLT3-ITD+ AML

Acute Myeloid LeukemiaIntermediate Risk Acute Myeloid LeukemiaFLT3 Internal Tandem Duplication Positive+1 more
First Affiliated Hospital of Zhejiang University25 enrolled1 locationNCT07259707
Recruiting

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

Mitochondrial DiseasesMitochondrial DisordersMitochondrial Genetic Disorders+2 more
Columbia University1,000 enrolled17 locationsNCT01694940
Recruiting

The Myelin Disorders Biorepository Project

AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369
Recruiting

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

SYNGAP1-Related Intellectual DisabilityDLG4FOXP1+181 more
Simons Searchlight100,000 enrolled2 locationsNCT01238250
Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+188 more
Sanford Health20,000 enrolled2 locationsNCT01793168
Recruiting

Clinical and Molecular Biomarker Studies in RAI1 (Retinoic Acid-Induced 1) -Related Disorders

RAI1 Gene 17P11.2 Deletion+Duplication
Baylor College of Medicine90 enrolled1 locationNCT06274164
Recruiting

Characterization and Natural History of Williams Syndrome and Other Chromosome 7q11.23 Variants

Williams SyndromeWilliams Beuren SyndromeWilliams Beuren Region Duplication+1 more
University of Pennsylvania2,000 enrolled1 locationNCT06930417
Recruiting

The 3q29 Deletion and 3q29 Duplication: Architecture of Behavioral Phenotypes

Microdeletion 3q29 SyndromeMicroduplication 3q29 Syndrome
Rutgers, The State University of New Jersey800 enrolled1 locationNCT02447861
Recruiting

Lynch Syndrome X-Talk of Enteral Mucosa With Immune System

Hereditary Cancer SyndromeHereditary CancerLynch Syndrome+19 more
San Raffaele University300 enrolled5 locationsNCT06708429
Recruiting
Not Applicable

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

MECP2 Duplication Syndrome
HuidaGene Therapeutics Co., Ltd.6 enrolled1 locationNCT06615206