Phase 2 N=37 NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
22q11 Deletion Syndrome
Primary: Safety and Tolerability of NB-001 — 18; 24; 14; 20 Participants
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=19 Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
22q13 Deletion Syndrome · Phelan-McDermid Syndrome
Primary: Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1 — 15.78; 11.22; 7.61; 9.70 score on a scale
N/A N=110 Mindfulness Program for Adolescents With 22q11DS
22Q11 Deletion Syndrome
Primary: Change From Baseline in Adolescents' Coping Skills — 3.10; 2.68; 3.18; 2.79 units on a scale — p=0.04
Phase 2 N=20 Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
22Q11.2 Deletion Syndrome
Primary: Overall Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) — 12; 3 Participants
Phase 2 N=26 Thymus Transplantation in DiGeorge Syndrome #668
DiGeorge Syndrome · Complete Typical DiGeorge Anomaly
Primary: Survival at 1 Year Post-Cultured Thymus Tissue Implantation (CTTI) — 72 % of participants who survive to 1 year
Phase 2 N=6 Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Phase 2 N=7 Thymus Transplantation Dose in DiGeorge #932
DiGeorge Anomaly · DiGeorge Syndrome · Complete DiGeorge Anomaly
Primary: Survival at 1 Year Post-CTTI — 83 % of participants who survive to 1 year
Phase 2 N=14 Phase I/II Thymus Transplantation With Immunosuppression #950
DiGeorge Anomaly · Complete DiGeorge Anomaly · Complete Atypical DiGeorge Anomaly
Primary: Survival at 1 Year Post-CTTI — 71 % of participants who survive to 1 year
N/A N=29 Thymus Transplantation Safety-Efficacy
Complete DiGeorge Anomaly · DiGeorge Syndrome · DiGeorge Anomaly
Primary: Survival Rate at End of 1 Year — 79.3 percentage of participants
Phase 2 N=6 AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
Phelan-McDermid Syndrome · Epilepsy
Primary: Number of Adverse Events — 19 events
N/A N=165 Influence of Genetic Polymorphisms on Ventricular Structure and Function in Patients With Single Ventricle Anatomy
Congenital Heart Disease
Primary: To Evaluate the Incidence of the Pre-proendothelin SNP at Nucleotide 5665 — 10 participants
N/A N=23 Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome
Down Syndrome · Obstructive Sleep Apnea Syndrome
Primary: Change in Epworth Sleepiness Scale From Baseline to End of Study — -1; -0.5; 1 units on a scale