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Showing 1–24 of 42 matching trials from the live ClinicalTrials.gov search.
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Recruiting Not applicable Interventional

Intrauterine Stent Placement Following Hysteroscopic Septum Resection

NCT07032506
View directory record Official record
Conditions
Intrauterine Adhesion
Interventions
Intraoperative placement of an inflated intrauterine pediatric foley catheter, No Catheter placement, Hormonal Therapy Agent, Oral antibiotics
Procedure · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older · Female only
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
3
States / cities
Little Rock, Arkansas • New York, New York • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Rare Diseases Clinical Research Network: Neurophysiological Correlates

NCT03077308
View directory record Official record
Conditions
Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome, Rett-related Disorders
Interventions
Auditory and Visual Event-related Potentials and EEG
Procedure
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
2 Years to 65 Years
Enrollment
185 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
5
States / cities
Denver, Colorado • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Development of Non-invasive Prenatal Test for Microdeletion and Other Genetic Syndromes Based on Cell Free DNA

NCT02109770
View directory record Official record
Conditions
Microdeletion Syndromes, Trisomy 21, Trisomy 18, Trisomy 13, Sex Chromosome Abnormalities
Interventions
Not listed
Lead sponsor
Natera, Inc.
Industry
Eligibility
Not listed
Enrollment
216 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
2
States / cities
San Carlos, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 25, 2019 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Clinical Evaluation of the SEQureDx T21 Test In High Risk Pregnancies

NCT01555346
View directory record Official record
Conditions
Down Syndrome, Fetal Aneuploidy
Interventions
Not listed
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years to 60 Years · Female only
Enrollment
3,062 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
27
States / cities
Mobile, Alabama • Tucson, Arizona • Campbell, California + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2016 · Synced Jul 21, 2026, 9:30 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration

NCT04201457
View directory record Official record
Conditions
Low Grade Glioma (LGG) of Brain With BRAF Aberration, High Grade Glioma (HGG) of the Brain With BRAF Aberration, Low Grade Glioma of Brain With Neurofibromatosis Type 1
Interventions
Dabrafenib, Trametinib, Hydroxychloroquine
Drug
Lead sponsor
Pediatric Brain Tumor Consortium
Network
Eligibility
1 Year to 30 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
15
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Generating Advancements Through Longitudinal Analysis in X and Y Variations (GALAXY)

NCT06373861
View directory record Official record
Conditions
Sex Chromosome Aneuploidy
Interventions
no intervention
Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
0 Days to 100 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2037
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational Results available

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

NCT02699190
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Biobanking of Rett Syndrome and Related Disorders

NCT02705677
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
752 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 21, 2026, 9:30 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial

NCT01545674
View directory record Official record
Conditions
Trisomy 13, Trisomy 18, Trisomy 21, Aneuploidy
Interventions
Blood Draw
Procedure
Lead sponsor
Natera, Inc.
Industry
Eligibility
Female only
Enrollment
937 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2023
U.S. locations
24
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2023 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Myotubular Myopathy Genetic Testing Study

NCT01817946
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Genetic Testing
Other
Lead sponsor
Cure CMD
Other
Eligibility
30 Days and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2018 · Synced Jul 21, 2026, 9:30 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

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

NCT06274164
View directory record Official record
Conditions
RAI1 Gene 17P11.2 Deletion+Duplication
Interventions
Electroencephalography/Polysomnography (EEG/PSG), Skin Biopsy, Blood draw
Diagnostic Test · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Month to 80 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

NCT04240002
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase 3 (FLT3) Mutation / Internal Tandem Duplication (ITD)
Interventions
gilteritinib, fludarabine, cytarabine, granulocyte colony-stimulating factor (G-CSF)
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
6 Months to 21 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 4, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Program for Revumenib

NCT05918913
View directory record Official record
Conditions
Relapsed/Refractory Acute Leukemia
Interventions
Revumenib
Drug
Lead sponsor
Syndax Pharmaceuticals
Industry
Eligibility
6 Months and older
U.S. locations
35
States / cities
Birmingham, Alabama • Phoenix, Arizona • Duarte, California + 26 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Jul 21, 2026, 9:30 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Quizartinib With Azacitidine or Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

NCT01892371
View directory record Official record
Conditions
FLT3 Gene Mutation Negative, FLT3 Internal Tandem Duplication Positive, Recurrent Acute Myeloid Leukemia, Recurrent Chronic Myelomonocytic Leukemia, Recurrent Myelodysplastic Syndrome, Refractory Acute Myeloid Leukemia, Refractory Chronic Myelomonocytic Leukemia, Refractory Myelodysplastic Syndrome
Interventions
Azacitidine, Cytarabine, Quizartinib
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2025 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Diagnostic Study of Gene Alterations in Patients With Acute Myeloid Leukemia

NCT00003931
View directory record Official record
Conditions
Leukemia
Interventions
reverse transcriptase-polymerase chain reaction
Genetic
Lead sponsor
Alliance for Clinical Trials in Oncology
Other
Eligibility
15 Years and older
Enrollment
9,769 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2006
U.S. locations
46
States / cities
Birmingham, Alabama • La Jolla, California • San Francisco, California + 28 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2016 · Synced Jul 21, 2026, 9:30 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

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

NCT06930417
View directory record Official record
Conditions
Williams Beuren Syndrome, Williams Syndrome, Williams Beuren Region Duplication, Dup7
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2045
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Genetic and Physical Characteristics of Rett Syndrome

NCT00299312
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication Disorder, Rett-related Disorder
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2017 · Synced Jul 21, 2026, 9:30 PM EDT
Recruiting Phase 1Phase 2 Interventional

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)

NCT06430385
View directory record Official record
Conditions
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
ION440, Sham procedure
Drug · Procedure
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
8
States / cities
San Diego, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
Completed Phase 1Phase 2 Interventional Results available

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

NCT04240314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
scAAV9.U7.ACCA
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
6 Months to 13 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Jul 21, 2026, 9:30 PM EDT
Completed Phase 3 Interventional Results available

(QuANTUM-R): An Open-label Study of Quizartinib Monotherapy vs. Salvage Chemotherapy in Acute Myeloid Leukemia (AML) Subjects Who Are FLT3-ITD Positive

NCT02039726
View directory record Official record
Conditions
AML
Interventions
Quizartinib, Salvage Chemotherapy
Drug
Lead sponsor
Daiichi Sankyo
Industry
Eligibility
18 Years and older
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
36
States / cities
Duarte, California • Los Angeles, California • Sacramento, California + 31 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2021 · Synced Jul 21, 2026, 9:30 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Defining the Brain Phenotype of Children With Williams Syndrome

NCT01132885
View directory record Official record
Conditions
Williams Syndrome, Duplication
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
5 Years and older
Enrollment
415 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 21, 2026, 9:30 PM EDT
Completed Phase 2 Interventional Results available

A Study of ASP2215 (Gilteritinib), Administered as Maintenance Therapy Following Induction/Consolidation Therapy for Subjects With FMS-like Tyrosine Kinase 3 (FLT3/ITD) Acute Myeloid Leukemia (AML) in First Complete Remission

NCT02927262
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation
Interventions
Gilteritinib, Placebo
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
6
States / cities
Gainesville, Florida • Jacksonville, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2024 · Synced Jul 21, 2026, 9:30 PM EDT
Completed No phase listed Observational

Natural History of Rett Syndrome & Related Disorders

NCT02738281
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
1,044 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 21, 2026, 9:30 PM EDT
Terminated Phase 2 Interventional Results available

A Study of Soticlestat in Adults and Children With Rare Epilepsies

NCT03635073
View directory record Official record
Conditions
Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
Interventions
Soticlestat
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years and older
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
27
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 18 more
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Jul 21, 2026, 9:30 PM EDT