Clinical trials: Congenital Diseases
Down Syndrome, Speech and Language Disorder
Building Sentences With Preschoolers Who Use AAC
The focus of this investigation is to compare the effectiveness of the AAC Generative Language Intervention approach to an AAC Standard of Care condition on preschool sentence productions.
- Conditions
- Down Syndrome, Speech and Language Disorder, Speech Disorders in Children, Speech Intelligibility
- Trial name
- NCT03538925
- Testing
- AAC Generative Language Intervention, Standard of Care / Business as Usual
- Where
- 2 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University of Central Florida
Environmental Exposure, Pregnancy Outcome
The Ma'Anshan Birth Cohort (MABC)
The Ma'anshan Birth Cohort (MABC) is a population-based prospective pregnancy and birth cohort study that aims to explore the early environmental and genetic determinants of maternal and child health, with a focus on health…
- Conditions
- Environmental Exposure, Pregnancy Outcome, Birth Defects, Child Development
- Trial name
- NCT03215368
- Where
- China
- Volunteers
- 3,474 people, healthy volunteers welcome
- Sponsor
- Anhui Medical University
Tourette Syndrome, Tic Disorders
Trajectories of Change in Tourette Syndrome
This K23 Career Development Award is designed to provide the training needed for the PI to achieve her long-term career goal of conducting independent, programmatic intervention research in developmental populations.
- Trial name
- NCT06679790
- Testing
- Comprehensive Behavioral Intervention for Tics
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Jaundice, Neonatal
Screening for Neonatal Jaundice With a Mobile Health Device: a Validation Study in Oaxaca, Mexico.
The performance and accuracy of the smartphone application (app) Picterus Jaundice Pro (PicterusJP) will be validated by a cross-sectional study conducted in low resource settings in Mexico.
- Trial name
- NCT06276582
- Testing
- Screening for neonatal jaundice with a mobile health device
- Where
- Mexico
- Volunteers
- 177 people
- Sponsor
- Norwegian University of Science and Technology
Infant, Premature, Diseases, Birth, Preterm
The NEU-STIM Trial
The aim of this study is to determine the effect of repetitive tactile stimulation compared to selective stimulation on oxygenation of the infant at 5 minutes after birth.
- Trial name
- NEU-STIM (NCT05942924)
- Testing
- Repeated tactile stimulation, Selective tactile stimulation
- Where
- 43 sites in Austria, Belgium and 17 more
- Volunteers
- 3,280 people
- Sponsor
- Leiden University Medical Center
Fetal Growth Abnormality, Fetal Growth Restriction
Sildenafil Versus Low Molecular Weight Heparin in Fetal Growth Restriction Treatment
comparing the effect of using sildenafil citrate and LMWH in treatment of cases of IUGR due to placental insufficiency
- Trial name
- NCT03230162
- Testing
- Sildenafil, low molecular weight heparin
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Ain Shams University
Sickle Cell Disease
Assessing Function in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the FIM™ as a measure of daily function in children with sickle cell disease hospitalized with vasoocclusive pain.
- Trial name
- NCT00590148
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- Connecticut Children's Medical Center
Narrow Urethral Plate Hypospadias
Evaluation of Slit-like Adjusted Mathieu Versus Onlay Island Flap for Hypospadias Repair in Pediatrics
Purpose** To compare the Slit-like Adjusted Mathieu (SLAM) technique versus the inner transverse preputial onlay island flap for repair of anterior and mid-penile hypospadias with narrow urethral plate, evaluating complication…
- Trial name
- NCT07592026
- Testing
- Slit-like Adjusted Mathieu, onlay island flap
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Al-Azhar University
Hereditary Haemorrhagic Telangiectasia
Iron Deficiency and Hereditary Haemorrhagic Telangiectasia
Managing iron deficiency is important for more than 1 billion individuals worldwide, to avoid blood transfusions, or excessive strain on vital organs that depend on iron-containing haemoglobin to deliver oxygen to the tissues.
- Trial name
- NCT01908543
- Testing
- Ferrous sulphate 200mg oral tablet
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- Imperial College London
Tourette Syndrome
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Guanfacine for Tourette Syndrome and Attention Deficit Hyperactivity Disorder
OBJECTIVES: I. Evaluate the safety and efficacy of the alpha-2 adrenergic agonist guanfacine in children and adolescents with Tourette syndrome or other chronic tic disorder, and attention deficit hyperactivity disorder.
- Trial name
- NCT00004376
- Testing
- guanfacine
- Volunteers
- 35 people
- Sponsor
- National Center for Research Resources (NCRR)
Cleft Lip and Cleft Palate, Cleft Lip Bilateral
Improving the Outcomes of Nasoalveolar Molding in Complete Bilateral Cleft Lip and Palate Neonates Using a Modification in the Design of the Conventional Molding Appliance.
The goal of this clinical trial was to improve the outcome of using the nasoalveoalr molding appliances in patients with complete bilateral cleft lip and palate.
- Conditions
- Cleft Lip and Cleft Palate, Cleft Lip Bilateral, Complete Bilateral Cleft Lip and Palate
- Trial name
- NCT07814625
- Testing
- vomer molding appliance, conventional molding appliance
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Tanta University
Spinal Cord Injury, Spinal Cord Injury No Sensory/Motor in S4-S5
The Aim of This Research is to Better Understand How Complete Spinal Cord Injury May Change Erogenous Zones in Adult Males
A complete spinal cord injury (SCI) in men is characterized by the total loss of motor skills and sensations below the injury. The loss of penile sensation, the erogenous zone primarily involved in sexual response, is involved in…
- Trial name
- QualiEroSCI (NCT06908850)
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Fondation Garches
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease.
- Trial name
- PAPAYA (NCT01410890)
- Testing
- alglucosidase alfa
- Where
- 12 sites in United States, Bulgaria and 4 more
- Volunteers
- 21 people
- Sponsor
- Genzyme, a Sanofi Company
Acute Hepatic Porphyria, Hepatic Porphyrias
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic…
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP)
- Trial name
- NCT03547297
- Where
- 12 sites in United States
- Volunteers
- 212 people
- Sponsor
- Alnylam Pharmaceuticals
Gout
A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout
The purpose of this study is to: Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in…
- Trial name
- NCT05256810
- Testing
- ALN-XDH
- Where
- United Kingdom
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Patent Foramen Ovale, Ischemic Stroke
Discontinuation of Antithrombotic Treatment Following Patent Foramen Ovale Closure in Young Patients With Cryptogenic Stroke
To determine the safety of antithrombotic treatment discontinuation 12 months following successful transcatheter PFO closure.
- Conditions
- Patent Foramen Ovale, Ischemic Stroke, Bleeding Ulcer
- Trial name
- HALTI (NCT04475510)
- Testing
- Antiplatelet treatment discontinuation
- Where
- Canada
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Josep Rodes-Cabau
Hemophilia
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median…
- Trial name
- NCT04108260
- Testing
- Albutrepenonacog Alfa 1 UNT [IDELVION]
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- National Taiwan University Hospital
Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus…
- Conditions
- Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired
- Trial name
- EAP-001 (NCT02189720)
- Testing
- Amifampridine Phosphate
- Where
- United States
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fulcrum Therapeutics
Congenital Heart Disease (CHD)
Cord Clamping Among Neonates With Congenital Heart Disease
The goal of this clinical trial is to compare 2 different timepoints for clamping the umbilical cord at birth for term-born infants with a prenatal diagnosis of congenital heart disease (CHD).
- Trial name
- CORD-CHD (NCT06153459)
- Testing
- Umbilical Cord Clamping at ~30 seconds, Umbilical Cord Clamping at ~120 seconds, Umbilical Cord Milking
- Where
- 21 sites in United States, Canada
- Volunteers
- 500 people
- Sponsor
- Carl Backes, MD
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