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Adjunctive Everolimus (RAD 001) Therapy for Epilepsy in Children With Sturge-Weber Syndrome (SWS) (External Link)
The main study part has 3 phases. The first phase is called the baseline phase and consists of 1-2 study site visit(s). During this phase, the investigators will gather information …
Baylor Role:
Lead Sponsor
Completed
A Safety Study of NNZ-2566 in Patients With Rett Syndrome (External Link)
Rett Syndrome is a developmental disorder primarily if not exclusively affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of …
Baylor Role:
Collaborator
Completed
Efficacy of a Brief Behavioral Intervention to Treat ADHD and Disruptive Behaviors In Preschoolers (External Link)
This pilot study will employ a randomized controlled clinical trial design comparing symptoms of children who complete the Brief Behavioral Intervention to a wait-list control. Children in the wait-list control …
Baylor Role:
Lead Sponsor
Completed
Calmer Life: Testing the Effectiveness of a Treatment for Anxiety (External Link)
Cognitive behavioral treatment (CBT) produces positive outcomes for late-life worry/GAD in academic and primary care settings, but minorities are significantly underrepresented in clinical trials, and standard CBT poorly addresses the …
Baylor Role:
Lead Sponsor
Active, Not Recruiting
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita (External Link)
Dyskeratosis congenita (DC) is an inherited multisystem disorder, which classically presents with a clinical triad of skin pigment abnormalities, nail dystrophy, and oral leukoplakia. DC is part of a spectrum …
Baylor Role:
Collaborator
Terminated
Intravenous Ketamine in the Treatment of Obsessive-Compulsive Disorder (External Link)
This study will test the safety and efficacy of a single intravenous (IV) dose of the N-methyl-D-aspartate (NMDA) glutamate receptor antagonist, ketamine, in treatment-resistant OCD.
Active, Not Recruiting
Administration of Donor T Cells With the Caspase-9 Suicide Gene (External Link)
If the patient is doing well after the stem cell transplant, and does not have severe GvHD, s/he will be eligible to receive the special "iCasp9" T cells from Day …
Baylor Role:
Lead Sponsor
Completed
Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW (External Link)
Subjects with AS, RTT or PWS and normal siblings (controls) will be recruited for study participation. Subjects will be recruited from the Rare Disease Clinical Research Network (RDCRN) consortium registries …
Baylor Role:
Lead Sponsor
Completed
Calcium Absorption in Patients With Rothmund-Thomson Syndrome (External Link)
This is an evaluation of bone calcium deposition in patients with Rothmund-Thomson Syndrome (RTS). This study includes one study visit during which subjects will be admitted to the inpatient unit …
Baylor Role:
Lead Sponsor
Completed
A Trial of Levodopa in Angelman Syndrome (External Link)
Levodopa is a prodrug that "delivers" dopamine to the brain. It is usually given with carbidopa, a peripheral decarboxylase inhibitor, to increase the bioavailability of levodopa. Animal studies have suggested …
Baylor Role:
Collaborator