23 results
At least 90% of the subjects, with 95% confidence, will tolerate the test formula.
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…
Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…
To evaluate the effect of prolonged duration of (fos)aprepitant prophylaxis on the prevention of delayed CINV (complete remission in the 24-72 hours after the final dose of chemotherapy) in children. The current 3-day regimen is compared to a…
To evaluate (reduction of) intraoperative blood-loss with and without pre-operative TXA administration, in children undergoing a proximal femoral and/or pelvic osteotomy (PFPO).
Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.
Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI for the total group (both prenatal and postnatal diagnosis) and the prenatal diagnosis group, which is needed to…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in pediatric subjects with pyruvate kinase deficiency (PK deficiency) who are regularly transfused.
Part A: PrimaryTo evaluate the safety and tolerability of single ascending dose of BIIB115 administered via IT bolus injection to HVsSecondaryTo evaluate the single dose PK of BIIB115 administered via IT bolus injection to HVsPart B:PrimaryTo…
The main goal of this study is to find out whether finerenone in combination with either an ACE inhibitor or an ARB can reduce the amount of protein in the participants' urine more than a placebo. A placebo looks like a drug, but contains no…
In a randomized control trial (RCT) with mothers with postpartum depressive symptoms the following main hypotheses will be tested:• Oxytocin will promote more sensitive caregiving during mother-infant interactions and while interacting with a life-…
Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander disease Secondary Objectives: To further evaluate the efficacy of ION373 in improving or stabilizing disease…
Primary: To determine whether three months of prophylactic treatment with co-trimoxazole causes a reduction in the number of days a child experiences at least two RTI symptoms in children aged 6 months to <=10 years with recurrent RTIs, when…
In the present randomised double blind controlled study, we will study the effects of GOS or FOS vs a placebo on stool consistency. Other parameters to be investigated include stool frequency, stool consistency in number of cases (%), painful…
Primary objectives:Evaluation of efficacy of multispecific T-cell transfer in patients with chemo-refractory viral infections after allogeneic stem cell transplantationSecondary objectives:- Incidence and severity of newly occurring GvHD- Incidence…
In the present randomised double blind controlled study, we will study the effects of GOS vs a placebo on stool consistency in children with hard or lumpy stools. Other parameters to be investigated include stool frequency, stool consistency in…
Primary Objective:To investigate change in fat mass index between 6 and 12 months of age in infants receiving Nuturis® formula feeding versus standard formula feeding during the first 6 months of life.Secondary Objectives:- To investigate change in…
The primary objective is to demonstrate the superiority versus placebo of a 4 weeks intranasal OT administration on oral skills assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) in infants with PWS aged less than or equal to 3 months at…
The main objective is to study the effect of the nutritional intervention versus the control product on white matter development (as assessed using tract based spatial statistics of fractional anisotropy) using a DTI scan at the corrected age of…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused