To evaluate the safety and tolerability of VAX-31 at 3 dose levels administered to healthy infants at 2, 4, 6, and 12-15 months of age alone and compared to PCV20.
We are recruiting young people with bipolar disorder to determine whether a 16-week keto diet therapy, coupled with standard psychiatric care, is well-tolerated and can lead to improvements in the mood and health of young people with bipolar spectrum disor
Evaluate the Safety, Pharmacokinetics, and Efficacy of XYOSTED® for Testosterone Replacement in Male Adolescents (ages: 12 to <18 years) with Conditions Associated with deficiency or Absence of Endogenous Testosterone Due to Primary or Secondary Hypogonadi
This study wants to collect health and growth information about children with hypochondroplasia. This information will help us understand their basic health and see if they can join future studies about hypochondroplasia.
We are studying adolescents who may be at higher risk of getting type 2 diabetes, as they go through puberty. Our goal is to identify early signs that might help predict who will develop the disease.
This study wants to learn if breathing tests (called spirometry) performed in an outpatient clinic give results that are as reliable and accurate as spirometry that is done in a specialized lung function lab.
In this study we want to find out the safest dose of the oral drug revumenib to give young children with KMT2A acute lymphoblastic leukemia that has come back or not responded to other treatment. We want to see if this treatment in combination with chemoth
This study will evaluate the effectiveness and safety of risdiplam (Evrysdi®) administered to patients under 2 years of age genetically diagnosed with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec a
This study wants to learn about how effective and safe the study drug Debio 4326 (triptorelin) is for treating children who have central precocious puberty (CPP).
This study wants to learn more about how blood sugar levels in healthy children without chronic disease compare to the blood sugar levels of children with Cystic Fibrosis (CF). We are only recruiting children without chronic disease to join the study.
Treatment of Duchenne Muscular Dystrophy (DMD) with dystrophin deletion amenable to exon 50 skipping
In this study we want to gather more information about the safety and tolerability of a drug called ferric citrate for treating children with chronic kidney disease who have hyperphosphatemia.
We are studying teens who feel angry a little, sometimes, or a lot to see if they think about social situations in different ways. Teens who often feel very angry might have more problems when they grow up, so we want to learn more about how their brains work.
This study wants to learn new ways to prevent and respond to pandemics by studying current infections, like colds. We will collect and store biological samples and health data from children to use in research to make drugs, tests, and vaccines for these ty
To evaluate the safety and tolerability of multiple-ascending dose (MAD) levels, 40 mg/kg, 60 mg/kg, and 80 mg/kg, of NS-089/NCNP-02 in ambulant boys with DMD after 4 weeks of once weekly intravenous (IV) treatment; and o evaluate the pharmacokinetics (PK)
In this study, we want to learn more about how doctors use a special type of dialysis machine called CARPEDIEM to treat babies that have kidney problems. We want to collect medical information from babies who were treated with this machine to help improve
This study wants to look at how endocannabinoids are different in children with and without autism. We are only looking for children who do not have autism to join our study.
An Open-Label multinational, multicenter study to evaluate the long-term safety, tolerability, and efficacy of subcutaneous amlitelimab in participants aged 12 years and older with moderate to severe atopic dermatitis
A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome with an Inadequate Response to Human Growth Hormone
To evaluate the safety and tolerability of benralizumab