Early Phase 1 N=10 Study of Modified Atkins Diet in Kabuki Syndrome
Kabuki Syndrome
Primary: Benton Judgement of Line Orientation — 8.6; 11.6 score on a scale
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=10 Low-Dose Ketamine in Children With ADNP Syndrome
ADNP Syndrome
Primary: Number of Participants With an Adverse Event — 9 Participants
Phase 2 N=47 Tofacitinib for Immune Skin Conditions in Down Syndrome
Down Syndrome · Alopecia Areata · Atopic Dermatitis / Eczema
Primary: Number of Serious Adverse Events (SAE) Definitely Related to Tofacitinib Treatment. — 0 Serious Adverse Events
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
N/A N=10 Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
Autism Spectrum Disorder
Primary: Preschool Language Scales — 7.6 Raw score units / 16 weeks
Phase 2 N=22 Anakinra in Infants and Children With Coronary Artery Abnormalities in Acute Kawasaki Disease
Kawasaki Disease
Primary: Number of Participants With Adverse Events — 2; 3; 13 participants