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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:08 AM EDT

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Showing 433–456 of 559 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Chromosomal Abnormalities Clear all
Completed Phase 1 Interventional Results available

CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory CD19/CD22-expressing B Cell Malignancies

NCT03448393
View directory record Official record
Conditions
Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, B Cell, B-Cell Lymphoma, B-Cell Leukemia, Acute Lymphoid Leukemia, B-Non Hodgkin Lymphoma, B-NHL, B-All, Acute Lymphoblastic Leukemia, Acute Lymphocytic Leukemia
Interventions
CD19/CD22 CAR T-Cells, Fludarabine, Cyclophosphamide, Apheresis, Anti-emetic, Diphenhydramine, Acetaminophen, ECG, ECHO, MRI Brain, Bone marrow biopsy, Cardiac MRI
Biological · Drug · Procedure + 2 more
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 39 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 4, 2025 · Synced Oct 22, 2026, 5:08 AM EDT
Recruiting Phase 2 Interventional

A Study of CSTI-500 in Patients With Prader-Willi Syndrome

NCT07348601
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
CSTI-500
Drug
Lead sponsor
ConSynance Therapeutics
Industry
Eligibility
13 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 22, 2026, 5:08 AM EDT
Recruiting Phase 3 Interventional

Compassionate Use Study of Epi-ON Corneal Collagen Crosslinking Performed Using UVA Exposure on Eyes With Ectatic Corneal Diseases for Subjects With Down Syndrome

NCT07135167
View directory record Official record
Conditions
Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
Interventions
Epi-ON corneal cross-linking (CXL)
Combination Product
Lead sponsor
Woolfson Eye Institute
Other
Eligibility
8 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
12
States / cities
Atlanta, Georgia • Canton, Georgia • Cumming, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2026 · Synced Oct 22, 2026, 5:08 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome

NCT03790865
View directory record Official record
Conditions
Prader-Willi Syndrome, Hyperphagia
Interventions
Livoletide, Placebo
Drug
Lead sponsor
Millendo Therapeutics SAS
Industry
Eligibility
4 Years to 65 Years
Enrollment
158 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 22, 2026, 5:08 AM EDT
Completed No phase listed Observational

Studying Chromosomes in Samples From Younger Patients With Neuroblastoma

NCT01589341
View directory record Official record
Conditions
Neuroblastoma
Interventions
laboratory biomarker analysis
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 18 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Monrovia, California
Source: ClinicalTrials.gov public record
Updated May 18, 2016 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Assessing Malleability of Spatial Abilities in Down Syndrome

NCT05332912
View directory record Official record
Conditions
Down Syndrome
Interventions
Spatial Ability Experience
Behavioral
Lead sponsor
University of Alabama, Tuscaloosa
Other
Eligibility
4 Years to 25 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Tuscaloosa, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 22, 2026, 5:08 AM 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 to 99 Years
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 Oct 16, 2026 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Phase 1 Interventional

Vorinostat and Decitabine in Treating Patients With Relapsed, Refractory, or Poor-Prognosis Hematologic Cancer or Other Diseases

NCT00357708
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Chronic Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Philadelphia Chromosome Negative Chronic Myelogenous Leukemia, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Relapsing Chronic Myelogenous Leukemia
Interventions
decitabine, vorinostat, laboratory biomarker analysis, pharmacological study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 7, 2013 · Synced Oct 22, 2026, 5:08 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

Validation of Sleep Questionnaires in the Down Syndrome Population

NCT03771469
View directory record Official record
Conditions
Obstructive Sleep Apnea, Down Syndrome
Interventions
Sleep study
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years to 17 Years
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2027
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Sep 24, 2025 · Synced Oct 22, 2026, 5:08 AM EDT
Active, not recruiting Phase 2 Interventional

Medications for Obstructive Sleep Apnea to Improve Cognition in Children With Down Syndrome

NCT05933603
View directory record Official record
Conditions
Obstructive Sleep Apnea, Down Syndrome
Interventions
Atomoxetine and Oxybutynin (ato-oxy)
Drug
Lead sponsor
University of Arizona
Other
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Not applicable Interventional

Intensive Dysarthria Sessions in Adults and Children With Down Syndrome

NCT01684670
View directory record Official record
Conditions
Dysarthria, Down Syndrome
Interventions
Speech sessions
Behavioral
Lead sponsor
Duke University
Other
Eligibility
8 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 23, 2016 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Phase 2 Interventional Results available

Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy

NCT01869114
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), de Novo Myelodysplastic Syndromes, Myelodysplastic Syndrome With Isolated Del(5q), Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia
Interventions
Sirolimus, Azacitidine
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2024
U.S. locations
5
States / cities
Sewell, New Jersey • Abington, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Oct 22, 2026, 5:08 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Vascular Function in Adults With Down Syndrome

NCT07630207
View directory record Official record
Conditions
Down Syndrome (Trisomy 21)
Interventions
Not listed
Lead sponsor
University of Nevada, Las Vegas
Other
Eligibility
18 Years to 35 Years
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2027
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 22, 2026, 5:08 AM EDT
Not listed Phase 2 Interventional

Combination Chemotherapy Followed by Donor Bone Marrow Transplant or Peripheral Stem Cell Transplant in Treating Patients With Hematologic Cancer or Genetic Disorders

NCT00008307
View directory record Official record
Conditions
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, cyclosporine, fludarabine phosphate, melphalan, methylprednisolone, mycophenolate mofetil, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, syngeneic bone marrow transplantation
Biological · Drug · Procedure
Lead sponsor
Herbert Irving Comprehensive Cancer Center
Other
Eligibility
1 Year to 80 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 6, 2014 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Screening of Low Responders for Aneuploidy to Improve Reproductive Efficiency

NCT01977144
View directory record Official record
Conditions
Infertility
Interventions
Comprehensive Chromosomal Screening
Other
Lead sponsor
Reproductive Medicine Associates of New Jersey
Other
Eligibility
18 Years to 43 Years · Female only
Enrollment
206 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Basking Ridge, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 14, 2022 · Synced Oct 22, 2026, 5:08 AM EDT
Recruiting No phase listed Observational

Angelman Syndrome Natural History Study

NCT04507997
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2031
U.S. locations
8
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2025 · Synced Oct 22, 2026, 5:08 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Angelman Syndrome Video Assessment (ASVA) Source Material Study

NCT05637697
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
1 Year and older
Enrollment
73 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Phase 3 Interventional

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome

NCT01325220
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
arbaclofen, Placebo
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
5 Years to 11 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
28
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 24 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2013 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Not applicable Interventional

Ultraviolet-B Light Therapy and Allogeneic Stem Cell Transplantation in Treating Patients With Hematologic Malignancies

NCT00068523
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, fludarabine phosphate, methylprednisolone, UV light therapy, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2020 · Synced Oct 22, 2026, 5:08 AM EDT
Withdrawn Phase 2 Interventional

Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40

NCT04823052
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2, Placebo, Gaboxadol (HLX-0206)
Drug
Lead sponsor
Healx Limited
Industry
Eligibility
13 Years to 40 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
7
States / cities
Aurora, Colorado • Miami, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2022 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Phase 3 Interventional Accepts healthy volunteers

Blood Flow and Blood Pressure Investigation in Down Syndrome

NCT05528549
View directory record Official record
Conditions
Down Syndrome
Interventions
Exercise
Other
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
18 Years to 45 Years
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 22, 2026, 5:08 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT05127226
View directory record Official record
Conditions
Angelman Syndrome
Interventions
ION582
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
San Diego, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Not applicable Interventional Results available

A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea

NCT02344108
View directory record Official record
Conditions
Sleep Apnea, Obstructive, Down Syndrome
Interventions
Inspire® Upper Airway Simulation System (Model 3028 IPG )
Device
Lead sponsor
Christopher Hartnick, M.D.
Other
Eligibility
10 Years to 21 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
5
States / cities
Atlanta, Georgia • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2022 · Synced Oct 22, 2026, 5:08 AM EDT
Completed Phase 2 Interventional

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

NCT05630066
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Alogabat
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
5 Years to 17 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 22, 2026, 5:08 AM EDT