1538 | Term | Boston Children's Hospital

AZithromycin Therapy in Preschoolers With a Severe Wheezing Episode Diagnosed at the Emergency Department

AZ-SWED is a parallel group, double blind, placebo control efficacy clinical trial with two separate hypotheses. The trial will compare the 5-day outcome of preschool children presenting to an ...

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Pragmatic Pediatric Trial of Balanced Versus Normal Saline Fluid in Sepsis

The objectives of this multicenter pragmatic clinical trial are to compare the effectiveness and relative safety of balanced fluid resuscitation versus 0.9% "normal" saline in children with septic ...

Nitrous Oxide for Pain Management During IUD Insertion in Nulliparous Adolescent Women

This pilot study aims to assess the feasibility, acceptability, and effectiveness of using nitrous oxide (N2O) sedation for intrauterine device (IUD) insertions for nulliparous adolescent and young ...

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll ...

Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.

Perinatal Arterial Stroke: A Multi-site RCT of Intensive Infant Rehabilitation (I-ACQUIRE)

This is a Phase III clinical trial to compare the efficacy of two dosages of a new infant rehabilitation protocol - I-ACQUIRE - to usual and customary forms of infant rehabilitation in infants who ...

Interventions for Convergence Insufficiency in Concussed Children

The ICONICC Study is a randomized controlled clinical trial designed to compare the proportion of successful treatment outcomes between children assigned to standard concussion care only, standard ...

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

This is a multicenter, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with AG-348 in participants who were previously enrolled in Study AG348-C ...

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative ...

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