Dell Children's Medical Center
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 10 Trials 
21 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Chang, Alicia Hsin-Ming
PROSPECT, NCT04930445: Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD

Recruiting
N/A
1000
US
Oxbryta® (voxelotor) 500mg Tablets, Voxelotor, Oxbryta®
Pfizer, Pfizer
Sickle Cell Disease
01/30
01/30
Stromberg, Daniel
pediRES-Q, NCT02708134: Quality of Pediatric Resuscitation in a Multicenter Collaborative

Recruiting
N/A
5000
Europe, Canada, Japan, US, RoW
Children's Hospital of Philadelphia, Zoll Medical Corporation
Cardiac Arrest, Cardiopulmonary Arrest
01/28
01/30
Berry-Kravis, Elizabeth
NCT05358886: A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

Recruiting
3
150
US
BPN14770/ zatolmilast, Placebo
Tetra Discovery Partners
Fragile X Syndrome
06/25
09/25
NCT05367960: An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Enrolling by invitation
3
300
US
Zatolmilast/ BPN14770
Tetra Discovery Partners
Fragile X Syndrome
09/27
12/27
RECONNECT, NCT04977986: Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults with Fragile X Syndrome

Recruiting
3
250
Europe, US, RoW
ZYN002 - transdermal gel, cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery), Placebo, Placebo Comparator, Matching Placebo
Zynerba Pharmaceuticals, Inc.
Fragile X Syndrome
05/25
05/25
NCT05163808: A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

Recruiting
2/3
150
US
zatolmilast, BPN14770, Placebo
Tetra Discovery Partners
Fragile X Syndrome
09/25
12/25
REVEAL, NCT05606614: Safety and Efficacy of TSHA-102 in Adolescent and Adult Females with Rett Syndrome ( Adult Study)

Recruiting
1/2
18
Canada, US
TSHA-102
Taysha Gene Therapies, Inc.
Rett Syndrome
01/25
01/32
NCT06152237: Safety and Efficacy of TSHA-102 in Pediatric Females with Rett Syndrome (REVEAL Pediatric Study)

Recruiting
1/2
20
Europe, Canada, US
TSHA-102
Taysha Gene Therapies, Inc.
Rett Syndrome
11/28
11/31
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
NCT04476862: Cerliponase Alfa Observational Study in the US

Active, not recruiting
N/A
35
US
Cerliponase Alfa, Brineura, Administration Kit
BioMarin Pharmaceutical
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
08/30
08/30
Robinson, Rhea
NCT04301843: Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

Recruiting
2
131
Canada, US
Eflornithine, DFMO, difluoromethylornithine
Giselle Sholler, K C Pharmaceuticals Inc., Beat NB Cancer Foundation, Team Parker for Life
Neuroblastoma
10/28
10/33
PEDS-PLAN, NCT02559778: Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

Recruiting
2
500
Canada, US
Ceritinib, Zykadia, dasatinib, Sprycel, sorafenib, Nexavar, vorinostat, ZOLINZA, DFMO, Eflornithine, α-difluoromethylornithine
Giselle Sholler, Dell, Inc., Beat NB Cancer Foundation, K C Pharmaceuticals Inc., Team Parker for Life
Neuroblastoma
09/30
09/35
PROSPECT, NCT04930445: Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD

Recruiting
N/A
1000
US
Oxbryta® (voxelotor) 500mg Tablets, Voxelotor, Oxbryta®
Pfizer, Pfizer
Sickle Cell Disease
01/30
01/30
ATHN Transcends, NCT04398628: A Natural History Study of Non-Neoplastic Hematologic Disorders

Recruiting
N/A
3000
US
American Thrombosis and Hemostasis Network, Pfizer, Hemophilia of Georgia, Inc., Genentech, Inc., Hemab Therapeutics, CSL Behring, Sanofi, Novo Nordisk A/S
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
06/35
12/35
Varshney, Pooja
adored, NCT05003804: Allergic Disease Onset Prevention Study

Active, not recruiting
1/2
264
US, RoW
STMC-103H, Placebo
Siolta Therapeutics, Inc.
Atopic Dermatitis, Type 1 Hypersensitivity
11/24
10/25
Hedequist, Daniel
SPARTAN, NCT05071144: Spine Procedures Assisted With RoboTics And Navigation

Recruiting
N/A
600
US
Boston Children's Hospital, Pediatric Orthopaedic Society of North America, Medtronic, Children's Hospital Colorado, Hospital for Special Surgery, New York, Children's Healthcare of Atlanta, Rainbow Babies and Children's Hospital, Dell Children's Medical Center of Central Texas, Cedars-Sinai Medical Center, Medical University of South Carolina Shawn Jenkins Children's Hospital, St. Louis Children's Hospital
Spine Deformity, Idiopathic Scoliosis, Adolescent Idiopathic Scoliosis, Spondylolisthesis, Congenital Scoliosis, Neuromuscular Scoliosis
12/30
12/31
Julich, Kristina
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Koch, Kendra
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Albert, Orsi
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Kaufman, Brian
SPARTAN, NCT05071144: Spine Procedures Assisted With RoboTics And Navigation

Recruiting
N/A
600
US
Boston Children's Hospital, Pediatric Orthopaedic Society of North America, Medtronic, Children's Hospital Colorado, Hospital for Special Surgery, New York, Children's Healthcare of Atlanta, Rainbow Babies and Children's Hospital, Dell Children's Medical Center of Central Texas, Cedars-Sinai Medical Center, Medical University of South Carolina Shawn Jenkins Children's Hospital, St. Louis Children's Hospital
Spine Deformity, Idiopathic Scoliosis, Adolescent Idiopathic Scoliosis, Spondylolisthesis, Congenital Scoliosis, Neuromuscular Scoliosis
12/30
12/31
Ross, Kirsten
SPARTAN, NCT05071144: Spine Procedures Assisted With RoboTics And Navigation

Recruiting
N/A
600
US
Boston Children's Hospital, Pediatric Orthopaedic Society of North America, Medtronic, Children's Hospital Colorado, Hospital for Special Surgery, New York, Children's Healthcare of Atlanta, Rainbow Babies and Children's Hospital, Dell Children's Medical Center of Central Texas, Cedars-Sinai Medical Center, Medical University of South Carolina Shawn Jenkins Children's Hospital, St. Louis Children's Hospital
Spine Deformity, Idiopathic Scoliosis, Adolescent Idiopathic Scoliosis, Spondylolisthesis, Congenital Scoliosis, Neuromuscular Scoliosis
12/30
12/31

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