Clinical trials: Congenital Diseases
Ataxia Telangiectasia, Growth Failure
Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT)
This study will evaluate the status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and composition and neuroprotection in children with Ataxia telangiectasia (AT).
- Trial name
- GHAT (NCT01052623)
- Testing
- Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerate
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
Attention-Deficit/Hyperactivity Disorder, Tourette Syndrome
Computerized Working Memory Training in Children With ADHD and Comorbid Tourette Syndrome
The investigators will conduct a randomized placebo-controlled trial of a computerized intervention targeting working memory in 30 children with comorbid Attention-Deficit/Hyperactivity Disorder (ADHD) and Tourette Syndrome (TS).
- Trial name
- NCT02732496
- Testing
- Targeted Cognitive Training (TCT), Youth Appropriate Online Games
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Cystic Fibrosis
Investigating the Use of a Positive Parenting Programme to Improve Treatment Adherence in Cystic Fibrosis
Cystic fibrosis (CF) is an illness that makes the lungs clog up with sticky mucus. There is no cure and so treatments are used to help make the illness easier to live with.
- Trial name
- NCT02501369
- Testing
- Self-directed Teen Triple P
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Emma Wells
Familial Hypercholesterolemia
a Prospective Pilot Study of Screening Out Rate and Clinical Management of Familial Hypercholesterolemia
1. Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot…
- Trial name
- NCT03398954
- Where
- China
- Volunteers
- 1,128 people
- Sponsor
- China Cardiovascular Association
Myotonic Dystrophy 1
Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
Myotonic Dystrophy type 1 (DM1) is a multisystem disease that causes muscle weakness and myotonia. As a result upper limb function might become impaired.
- Trial name
- SOUL-DM1 (NCT05006924)
- Where
- 3 sites in Norway
- Volunteers
- 89 people
- Sponsor
- Oslo University Hospital
Umbilical Hernia
Caudal Versus Rectus Sheath Study
The literature has clearly demonstrated that the effective treatment of postoperative pain in infants and children is challenging.
- Trial name
- NCT01394523
- Testing
- Bupivacaine
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Nationwide Children's Hospital
Endourology, Stones, Kidney
Flexible URS in Management of Renal Stones in Anomalous Kidney
The urotheliasis is a common problem encountered daily by all urologists worldwide with rates up to 13% in North America, 9% in Europe and 5% in Asia .
- Conditions
- Endourology, Stones, Kidney, Laser, Congenital Disorders
- Trial name
- NCT05299996
- Testing
- flexible ureterorenoscopy
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Assiut University
Apnea of Prematurity, Retinopathy of Prematurity
Use of Sucrose to Relieve Pain During Eye Exams in Infants
The purpose of this study is to see if an oral sucrose solution can comfort premature infants during their necessary eye exams. The investigators believe that the use of this solution prior to the eye exams will lead to a…
- Conditions
- Apnea of Prematurity, Retinopathy of Prematurity, Pain
- Trial name
- NCT00161694
- Testing
- sucrose solution
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Weill Medical College of Cornell University
Spina Bifida
Physical Capacity of Spina Bifida and Factors Affecting
Objective: It was aimed to evaluate the physical characteristics of the cases with spina bifida and to review the factors that may affect the etiology of the disease.
- Trial name
- NCT06541366
- Testing
- case-control study
- Where
- Turkey (Türkiye)
- Volunteers
- 2 people
- Sponsor
- Saglik Bilimleri Universitesi Gazi Yasargil Training and Research Hospital
Hereditary Angioedema
Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema (HAE)
Primary Outcome Measures: The primary endpoint was the time to onset of symptom relief of the first attack in the double blind phase.
- Trial name
- FAST2 (NCT00500656)
- Testing
- Icatibant, Tranexamic Acid
- Where
- Italy
- Volunteers
- 85 people
- Sponsor
- Shire
Molar Incisor Hypomineralization, Asthma in Children
Prevalence of Molar Incisor Hypomineralization Among Children Treated With Asthmatic Drugs Early in Their Lives
The aim of this study is to assess the prevalence of molar incisor hypo mineralization among children who have been treated with asthmatic drugs during the first three years of life.
- Trial name
- NCT04103710
- Where
- Egypt
- Volunteers
- 167 people
- Sponsor
- Cairo University
Advanced Solid Tumors, Metastatic Solid Tumors
Phase 1b Trial of BGJ398/BYL719 in Solid Tumors
To study the safety and efficacy of the combination of BGJ398 with BYL719 in patients whose tumors express mutations to PIK3CA with or without alterations to FGFR 1-3.
- Trial name
- NCT01928459
- Testing
- BGJ398, BYL719
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 62 people
- Sponsor
- Novartis Pharmaceuticals
Bronchopulmonary Dysplasia
Trial of Late Surfactant for Prevention of Bronchopulmonary Dysplasia
The purpose of this study is to determine if late doses of Infasurf surfactant given when patients are receiving inhaled nitric oxide will interact to improve surfactant function and increase survival without BPD in treated…
- Trial name
- TOLSURF (NCT01022580)
- Testing
- Infasurf surfactant (ONY, Inc.)
- Where
- 21 sites in United States
- Volunteers
- 511 people
- Sponsor
- Roberta Ballard
Hearing Loss, Mental Retardation
Postnatal HCMV Infection in Very Preterm Infants. Implications, Morbidity, Growth and Neurodevelopmental Outcomes.
The aim of this study is to investigate short and long term consequences from early postnatal HCMV infection transmitted via human milk in very preterm infants (birth weight < 1500 g or gestational age < 32 weeks).
- Conditions
- Hearing Loss, Mental Retardation, Microcephaly, Chorioretinitis
- Trial name
- NCT01151462
- Where
- Norway
- Sponsor
- Oslo University Hospital
Ras/MAPK Pathway Vascular Anomalies, Vascular Anomalies
24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs
The purpose of this study is to assess the effectiveness and safety of Trametinib (the "Study Drug") in patients with Ras/MAPK pathway driven vascular anomalies (VA). Trametinib has been approved by the U.S.
- Conditions
- Ras/MAPK Pathway Vascular Anomalies, Vascular Anomalies, Vascular Anomaly
- Trial name
- VATCH (NCT07549646)
- Testing
- Trametinib
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Children's Hospital of Philadelphia
Leukemia, Myelodysplastic Syndromes
Cytarabine and Daunorubicin With or Without Gemtuzumab Ozogamicin in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes
RATIONALE: Drugs used in chemotherapy, such as cytarabine and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00121303
- Testing
- cytarabine, daunorubicin hydrochloride, gemtuzumab ozogamicin
- Where
- 6 sites in United Kingdom
- Volunteers
- 600 people
- Sponsor
- Stichting Hemato-Oncologie voor Volwassenen Nederland
Cancer
Immunotherapy in Treating Patients Who Are Undergoing Bone Marrow or Peripheral Stem Cell Transplantation
RATIONALE: White blood cells from donors who have been exposed to cytomegalovirus may be able to help prevent this infection from occurring in patients who are undergoing bone marrow or peripheral stem cell transplantation.
- Trial name
- NCT00002673
- Testing
- infection prophylaxis and management
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Fred Hutchinson Cancer Center
Erythropoietic Protoporphyria
Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria
The purpose of the study is to evaluate the effect of exercise and heat on the light sensibility of patients with erythropoietic protoporphyria
- Trial name
- NCT00206869
- Testing
- exercise, heat, red light illumination
- Where
- Denmark
- Sponsor
- Bispebjerg Hospital
Alzheimer Disease, Down Syndrome
Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)
The primary purpose of this study is to evaluate the safety and tolerability of ION269 in adults with Down syndrome with evidence of brain amyloid positivity.
- Trial name
- NCT06673069
- Testing
- ION269
- Where
- 6 sites in United States, Spain
- Volunteers
- 1 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transposition of Great Arteries
Cardiovascular Magnetic Resonance in Adults With SysTemic Right Ventricle (STAR)
The main aim of the study is to explore HFs in SRV, comparing their magnitude and direction with that of subpulmonary RV and systemic LV.
- Trial name
- STAR (NCT05690854)
- Where
- Italy
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- IRCCS Policlinico S. Donato
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