Clinical trials: CHILD Syndrome
Fragile X Syndrome, Fragile X Syndrome (FXS)
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with…
- Trial name
- RECONNECT (NCT04977986)
- Testing
- ZYN002
- Where
- 27 sites in United States, Australia and 3 more
- Volunteers
- 257 people
- Sponsor
- Harmony Biosciences Management, Inc.
Generalized Myasthenia Gravis
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
- Trial name
- ADAPT Jr (NCT04833894)
- Testing
- Efgartigimod IV
- Where
- 25 sites in United States, Austria and 10 more
- Volunteers
- 15 people
- Sponsor
- argenx
Dravet Syndrome
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with…
- Trial name
- ARGUS (NCT04462770)
- Testing
- Clemizole HCl
- Where
- 86 sites in United States, Argentina and 9 more
- Volunteers
- 150 people
- Sponsor
- Epygenix
HIV Infections
T-20 Plus a Selected Anti-HIV Treatment in HIV-Infected Children and Adolescents
This study will evaluate T-20 in children.
- Trial name
- NCT00022763
- Testing
- Enfuvirtide
- Where
- 7 sites in United States
- Volunteers
- 52 people
- Sponsor
- Hoffmann-La Roche
Hyperphagia in Prader-Willi Syndrome
Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome
The purpose of this study is to evaluate the safety and effectiveness of intranasal FE 992097 in children and adults with Prader-Willi Syndrome.
- Trial name
- NCT01968187
- Testing
- FE 992097
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- Ferring Pharmaceuticals
HIV Infections
Safety and Effectiveness of Giving Two Nucleoside Reverse Transcriptase Inhibitors Alone or in Combination With 141W94 to HIV-Infected Children Who Have Never Received Protease Inhibitors
The purpose of this study is to see if it is safe and effective to add a new protease inhibitor (PI), 141W94, to an anti-HIV regimen that includes 2 nucleoside reverse transcriptase inhibitors (NRTIs).
- Trial name
- NCT00002193
- Testing
- Amprenavir
- Where
- 2 sites in United States
- Volunteers
- 210 people
- Sponsor
- Glaxo Wellcome
Solid Tumors, CNS Tumors
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain…
- Trial name
- STARTRK-NG (NCT02650401)
- Testing
- Entrectinib
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 69 people
- Sponsor
- Hoffmann-La Roche
Rett Syndrome
Analysis of the Glutathione Cycle in Children With Rett Syndrome
Analyis of the Glutathione Cycle in Children with Rett Syndrome
- Trial name
- NCT02360436
- Where
- United States
- Sponsor
- Edison Pharmaceuticals Inc
Neuroblastoma, Rhabdomyosarcoma
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
This study is a Phase 1, open-label, dose escalation and cohort expansion trial designed to characterize the safety, tolerability, PK, PD, immunogenicity and preliminary antitumor activity of enoblituzumab administered IV on a…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT02982941
- Testing
- Enoblituzumab
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- MacroGenics
Tourette's Disorder
Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current study is to determine efficacy and safety of once-weekly aripiprazole in reducing Total Tic Severity (TTS) score in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01418339
- Testing
- Aripiprazole
- Where
- 39 sites in United States, Canada and 4 more
- Volunteers
- 135 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Cushing's Disease
Mifepristone in Children With Refractory Cushing's Disease
Study objectives are to obtain safety, pharmacokinetic, and pharmacodynamic data on the effect of mifepristone on glucose metabolism, body weight and the growth-hormone-IGF in children with refractory Cushing's disease.
- Trial name
- NCT01925092
- Testing
- mifepristone
- Where
- United States
- Sponsor
- Corcept Therapeutics
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Safety and Efficacy of Long-term Somatropin Treatment in Children
This study is conducted in Europe. The aim of this study is to evaluate safety during the long-term use of somatropin (Norditropin®) in children as well as efficacy on change in height.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Foetal Growth Problem, Small for Gestational Age
- Trial name
- GrowthWIN II (NCT01543867)
- Testing
- somatropin
- Where
- Germany
- Volunteers
- 5,442 people
- Sponsor
- Novo Nordisk A/S
Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma
MORAb-004 in Treating Young Patients With Recurrent or Refractory Solid Tumors or Lymphoma
This phase I trial studies the side effects and best dose of MORAb-004 in treating young patients with recurrent or refractory solid tumors or lymphoma.
- Conditions
- Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Childhood Burkitt Lymphoma
- Trial name
- NCT01748721
- Testing
- anti-endosialin/TEM1 monoclonal antibody MORAb-004, pharmacological study, laboratory biomarker analysis
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Morphotek
Leigh Syndrome
A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)
The goal of this clinical trial is to investigate oral TTI-0102 to evaluate its pharmacokinetics, safety, and preliminary efficacy in patients with Leigh syndrome spectrum, in three sequences.
- Trial name
- NCT06990984
- Testing
- TTI-0102: cysteamine-pantetheine disulfide, D-Mannitol
- Volunteers
- 18 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Meningococcal Disease
Safety and Immunogenicity of Novartis Meningococcal B Vaccine When Administered to Immunocompromised Children and Adolescents Compared to Healthy Subjects.
The study aims at evaluating the safety and immunogenicity of rMenB+OMV NZ when administered to subjects from 2 to 17 years of age with increased risk of meningococcal disease because either of primary or secondary complement…
- Trial name
- NCT02141516
- Testing
- rMenB+OMV
- Where
- 20 sites in Italy, Poland and 3 more
- Volunteers
- 239 people, healthy volunteers welcome
- Sponsor
- Novartis
SGA, Turner Syndrome, Noonan Syndrome, ISS
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow
The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature.
- Trial name
- REAL 8 (NCT05330325)
- Testing
- Somapacitan, Norditropin®
- Where
- 199 sites in United States, Austria and 31 more
- Volunteers
- 412 people
- Sponsor
- Novo Nordisk A/S
HIV Infections
The Safety and Effectiveness of Two Forms of Saquinavir Combined With Other Anti-HIV Drugs in HIV-Infected Infants and Children
To determine the pharmacokinetic properties, tolerance and safety of 2 formulations of saquinavir, given in combination with 2 nucleoside antiretroviral drugs (part 1) or in combination with nelfinavir and nucleoside…
- Trial name
- NCT00002380
- Testing
- Nelfinavir mesylate, Saquinavir
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Tourette Syndrome
Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment.
- Trial name
- NCT02679079
- Testing
- NBI-98854
- Where
- 44 sites in United States
- Volunteers
- 98 people
- Sponsor
- Neurocrine Biosciences
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 administered via intracerebroventricular (ICV) injection in pediatric…
- Trial name
- NCT06662188
- Testing
- JAG201
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Jaguar Gene Therapy, LLC
Asperger's Syndrome, Dyslexia
My Scrivener® - Measuring Effectiveness and Dose Response in Children
This study will assess whether a computer haptic peripheral device programmed to provide repetitive motion training is as effective as the same repetitive motion training provided by a human being.
- Conditions
- Asperger's Syndrome, Dyslexia, Cerebral Palsy, Attention Deficit Disorder
- Trial name
- NCT00919906
- Testing
- My Scrivener(R)
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- Obslap Research LLC