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Results for “MECP2 Duplication Syndrome”

7 trials

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Showing 7 of 7 results

Not applicableLooking for participantsNCT06615206
What this trial is testing

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)

Who this might be right for
MECP2 Duplication Syndrome
HuidaGene Therapeutics Co., Ltd. 6
Testing effectiveness (Phase 2)Looking for participantsNCT06430385
What this trial is testing

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)

Who this might be right for
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Ionis Pharmaceuticals, Inc. 48
Not applicableStudy completedNCT00299312
What this trial is testing

Genetic and Physical Characteristics of Rett Syndrome

Who this might be right for
Rett SyndromeMECP2 Duplication DisorderRett-related Disorder
University of Alabama at Birmingham 10
Not applicableActive Not RecruitingNCT06014541
What this trial is testing

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Who this might be right for
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Ionis Pharmaceuticals, Inc. 29
Not applicableStudy completedNCT03077308
What this trial is testing

Rare Diseases Clinical Research Network: Neurophysiological Correlates

Who this might be right for
Rett Syndrome, Preserved Speech VariantMecp2 Duplication SyndromeRett-related Disorders
University of Alabama at Birmingham 185
Not applicableStudy completedNCT02705677
What this trial is testing

Biobanking of Rett Syndrome and Related Disorders

Who this might be right for
Rett SyndromeMECP2 DuplicationCDKL5+1 more
University of Alabama at Birmingham 752
Not applicableStudy completedNCT02738281
What this trial is testing

Natural History of Rett Syndrome & Related Disorders

Who this might be right for
Rett SyndromeMECP2 Duplication dIsorderCDKL5 Disorder+1 more
University of Alabama at Birmingham 1,044