Clinical trials: CHILD Syndrome
Fragile X Syndrome
AFQ056 for Language Learning in Children With FXS
The purpose of this clinical trial is to investigate the impact of AFQ056 on language learning in 3-6 year old children with Fragile X Syndrome (FXS).
- Trial name
- FX-LEARN (NCT02920892)
- Testing
- AFQ056, Language Intervention
- Where
- 14 sites in United States
- Volunteers
- 110 people
- Sponsor
- Elizabeth Berry-Kravis
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
HIV Infections
Safety and Efficacy of Switching From Stavudine or Zidovudine to Tenofovir DF in HIV-1 Infected Children
The primary objective of this study is to assess the efficacy of switching to tenofovir disoproxil fumarate (TDF) compared to continuing stavudine or zidovudine in maintaining virologic suppression in HIV-1 infected children.
- Trial name
- NCT00528957
- Testing
- Tenofovir DF, Zidovudine, Stavudine
- Where
- 9 sites in United States, Panama and 1 more
- Volunteers
- 97 people
- Sponsor
- Gilead Sciences
Autism, Asperger's Disorder
An Open-Label Trial of Aripiprazole in Autism Spectrum Disorders
The aim of this study is to evaluate the efficacy, safety and tolerability of aripiprazole monotherapy in the treatment of children and adolescents suffering from Autism Spectrum Disorder (ASD) over a 12-week period.
- Conditions
- Autism, Asperger's Disorder, Pervasive Developmental Disorder
- Trial name
- NCT00308074
- Testing
- Aripiprazole
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Cambridge Health Alliance
Congenital Adrenal Hyperplasia (CAH)
Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia
This study was developed to determine if a combination of four drugs (flutamide, testolactone, reduced hydrocortisone dose, and fludrocortisone) can normalize growth in children with congenital adrenal hyperplasia.
- Trial name
- NCT00001521
- Testing
- Fludrocortisone, Hydrocortisone, Letrozole
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Obstructive Sleep Apnea
Effect of Montelukast Therapy in Obstructive Sleep Apnea(OSA) Children
The investigators leading hypotheses are: Oral therapy with montelukast may lead to improved sleep study findings in children with mild to moderate Obstructive Sleep Apnea Syndrome (OSAS) who require surgical removal of adenoids…
- Trial name
- 32543 (NCT01027806)
- Testing
- Montelukast
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- University of Chicago
Diarrhoea
Causes of Rotavirus Vaccine Failure in Zambian Children
Zambia recently introduced routine infant immunization against rotavirus - the most important cause of severe gastroenteritis and diarrhoea mortality in children.
- Trial name
- NCT01886833
- Where
- Zambia
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Centre for Infectious Disease Research in Zambia
Atypical Hemolytic Uremic Syndrome (aHUS)
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of the study is to assess the efficacy of ravulizumab to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor (complement inhibitor treatment-naïve), as…
- Trial name
- NCT03131219
- Testing
- Ravulizumab
- Where
- 18 sites in United States, Belgium and 5 more
- Volunteers
- 34 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome
The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of seizures in children and adults with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS).
- Trial name
- NCT05163314
- Testing
- Soticlestat
- Where
- 116 sites in United States, Australia and 18 more
- Volunteers
- 352 people
- Sponsor
- Takeda
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma
Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors
This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come back, do not respond to therapy, or are newly diagnosed.
- Conditions
- Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue
- Trial name
- NCT02867592
- Testing
- Cabozantinib, Cabozantinib S-malate, Pharmacological Study
- Where
- 142 sites in United States
- Volunteers
- 109 people
- Sponsor
- National Cancer Institute (NCI)
HIV Infections
Safety and Pharmacokinetics (PK) of Raltegravir in HIV (Human Immunodeficiency Virus)-Infected Children and Adolescents
Integrase is 1 of 3 HIV (Human Immunodeficiency Virus)-1 enzymes required for viral replication. Raltegravir is a drug that prevents integrase from working properly.
- Trial name
- NCT00485264
- Testing
- Raltegravir poloxamer film coated tablet, Raltegravir chewable tablet, Raltegravir oral granules for suspension (20 mg/mL)
- Where
- 42 sites in United States, Argentina and 4 more
- Volunteers
- 153 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV/AIDS Treatment, HIV Infections
Lopinavir/Ritonavir (Kaletra) PK in Children
The objective of this study is to compare the pharmacokinetics of lopinavir tablets administered to pediatric patients as either whole or crushed tablets.
- Trial name
- NCT00810108
- Testing
- lopinavir/ritonavir (Kaletra®) tablets
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Autism Spectrum Disorder, Autistic Disorder
A Folinic Acid Intervention for Autism Spectrum Disorders
Researchers at Arkansas Children's Hospital Research Institute are conducting a study looking at the effects of Folinic Acid on language in Autism Spectrum Disorder and language impairment. The study has 3 phases.
- Conditions
- Autism Spectrum Disorder, Autistic Disorder, Autism, Asperger's Syndrome
- Trial name
- NCT01602016
- Testing
- Folinic Acid
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- University of Arkansas
HIV Infections
Atazanavir Used in Combination With Other Anti-HIV Drugs in HIV-Infected Infants, Children, and Adolescents
The purpose of this study was to find a safe and tolerable dose of the protease inhibitor (PI) atazanavir (ATV), with or without a low-dose boost of the PI ritonavir (RTV), when taken with other anti-HIV drugs in HIV infected…
- Trial name
- NCT00006604
- Testing
- ATV, Ritonavir
- Where
- 36 sites in United States, Puerto Rico and 1 more
- Volunteers
- 195 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Idiopathic Growth Hormone Deficiency, Turner Syndrome
First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study
PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner…
- Trial name
- NCT01419249
- Testing
- Blood sampling
- Where
- 31 sites in Argentina, Canada and 7 more
- Volunteers
- 458 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
22q11 Deletion Syndrome
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
This is a Phase 2, randomized, placebo-controlled crossover trial to assess the safety and efficacy of NB-001 in children and adolescents with 22q11DS that manifest commonly associated neuropsychiatric symptoms.
- Trial name
- NCT05290493
- Testing
- NB-001
- Where
- 4 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Nobias Therapeutics, Inc.
Functional Abdominal Pain, Functional Dyspepsia
Efficacy of Open Label Placebo in Children With FGIDs
This study is aimed at investigating the efficacy of placebo for symptom relief in children with abdominal pain related functional gastrointestinal disorders.
- Conditions
- Functional Abdominal Pain, Functional Dyspepsia, Irritable Bowel Syndrome, Functional Gastrointestinal Disorders
- Trial name
- Placebo (NCT02389998)
- Testing
- Hyoscyamine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Boston Children's Hospital
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory
First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
This is an open-label, multicenter, Phase 1/Phase 2, dose escalation and dose expansion study to evaluate the safety, pharmacokinetics, pharmacodynamics and anti-leukemic activity of SAR443579 in various hematological…
- Conditions
- Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia
- Trial name
- NCT05086315
- Testing
- SAR443579
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 101 people
- Sponsor
- Sanofi