Phase 4 N=20 Buspirone Treatment of Anxiety in Williams Syndrome
Williams Syndrome · Anxiety
Primary: Mean 16-Week Change in Pediatric Anxiety Rating Scale 5-Item Total Score — -7.9 score on a scale — p=<0.001
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=21 Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
Williams Beuren Syndrome
Primary: Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography — 0.028; 0.012 mm
Phase 2 N=21 A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram Syndrome · Diabetes Mellitus · Optic Nerve Atrophy
Primary: Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests — 0; 0; 0; 2 Participants
Phase 2 N=8 Gene Therapy for Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS)
Primary: Safety of Reduced Conditioning Regimen — 8 Participants
Phase 3 N=12 Betaine and Peroxisome Biogenesis Disorders
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
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