Clinical trials: Birth Defect
Atopic Dermatitis
Emollients in the Management of Atopic Dermatitis
The purpose of this study is to confirm that emollients play a major role in the maintenance therapy after clearing of inflammatory lesions and can reduce occurrence of flares in children with atopic dermatitis.
- Trial name
- NCT01779258
- Testing
- glycerol, paraffin (liquid and white soft), Atopiclair®, Locatop@
- Where
- 15 sites in Estonia, France and 3 more
- Volunteers
- 347 people
- Sponsor
- Pierre Fabre Medicament
Gastroschisis, Bowel Obstruction
The Use of Gastrografin to Help Alleviate Bowel Obstruction in Gastroschisis Patients.
This study will investigate the use of a drug called Gastrografin to aid in bowel mobility for paediatric patients who have undergone gastroschisis surgery.
- Conditions
- Gastroschisis, Bowel Obstruction, Birth Defect
- Trial name
- NCT03334578
- Testing
- Gastrografin
- Where
- Canada
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia
Based on success in other diseases, the Fred Hutchinson Cancer Research Center (FHCRC) has developed a transplant procedure for Fanconi anemia (FA), which does not completely destroy the patient's remaining bone marrow.
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00093743
- Testing
- fludarabine phosphate, cyclosporine, total-body irradiation
- Where
- 5 sites in United States
- Volunteers
- 2 people
- Sponsor
- Fred Hutchinson Cancer Center
Adrenal Tumors, Adrenal Malignancies
Adrenal Scans With Radioiodine-Labeled Norcholesterol (NP-59)
The purpose of this study is to find out if your adrenal glands are normal or abnormal. This can be determined by whether or not your adrenal gland concentrates more of a labeled building block of adrenal hormone, norcholesterol.
- Conditions
- Adrenal Tumors, Adrenal Malignancies, Abnormal Hormonal Secretions, Electrolytes Abnormalities
- Trial name
- NCT00591643
- Testing
- Imaging, Adrenal acans, (NP-59)
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Thoracic Aorta Dilatation, Marfan Syndrome
Thoracic Aortic Dilatation Syndromes
Aortic dilatation syndromes are comprised by a group of different syndromes, of which Marfan syndrome is the best described. Many of the aorta dilatation associated syndromes are heritable connective tissue disorders but some…
- Trial name
- NCT02111668
- Where
- Denmark
- Volunteers
- 120 people
- Sponsor
- University of Aarhus
Batten Disease, Late-Infantile Neuronal Ceroid Lipfuscinosis
Genotype - Phenotype Correlations of LINCL
In a separate protocol the Department of Genetic Medicine is proposing to carry out a study using gene transfer to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a…
- Trial name
- NCT00151268
- Volunteers
- 18 people
- Sponsor
- Weill Medical College of Cornell University
Cystic Fibrosis,
Evaluation of Bronchial Inflammation in Allergic Bronchopulmonary Aspergillosis (ABPA)
Chronic bronchial inflammation is an important clinical feature in cystic fibrosis. Approximately 10% of patients with cystic fibrosis suffer from Allergic Bronchopulmonary Aspergillosis.
- Trial name
- ABPA (NCT00906568)
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
Bowel Cleansing Prior to Clinical Procedures, Colorectal Anastomosis
Oral Antibiotics vs. No Antibiotics in Pediatric Colorectal Surgery
This study aims to evaluate whether giving oral antibiotics before elective colorectal surgery can improve recovery and reduce complications in children.
- Trial name
- PEACO (NCT07713433)
- Testing
- oral antibiotics, No Oral Antibiotics
- Where
- Pakistan
- Volunteers
- 74 people
- Sponsor
- Muhammad Zubair Shoukat
Marfan Syndrome
The Oxford Marfan Trial
The primary objective of the trial is to estimate the effects of allocation to irbesartan, or doxycycline, or a combination of both irbesartan and doxycycline, compared with placebo, on measures of elastic function of the aorta…
- Trial name
- NCT01949233
- Testing
- Irbesartan 150-300mg capsules daily for 6 months, Doxycycline 100-200mg capsules daily for 6 months
- Where
- 2 sites in United Kingdom
- Volunteers
- 56 people
- Sponsor
- University of Oxford
Duchenne Muscular Dystrophy
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Researchers at Children's Hospital Boston Neurology Department invite children to participate in a new research study. Researchers are looking for boys ages 2 - 30 with Duchenne Muscular Dystrophy (DMD) and healthy boys ages 2 …
- Trial name
- QED (NCT01491555)
- Where
- United States
- Volunteers
- 73 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Risk-directed Therapy, Cytogenetic Abnormality
Risk-stratified Therapy Based on Molecular Cytogenetic Aberration and Treatment Response in AML
Risk-stratified therapy based on molecular and cytogenetic for acute myeloid leukemia (AML) is well accepted and benefits patients' survival.
- Conditions
- Risk-directed Therapy, Cytogenetic Abnormality, Molecular Abnormality, MRD
- Trial name
- NCT03620955
- Testing
- Justified risk stratification based on MRD after three course chemo therapy
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Moderate-to-Severe Atopic Dermatitis
Efficacy and Safety of Nemolizumab in Subjects With Moderate-to-Severe Atopic Dermatitis
The main purpose of the study was to assess the efficacy and safety of nemolizumab after a 16-week treatment period in adult and adolescent subjects with moderate-to-severe atopic dermatitis (AD) not adequately controlled with…
- Trial name
- NCT03985943
- Testing
- Nemolizumab
- Where
- 174 sites in United States, Australia and 12 more
- Volunteers
- 941 people
- Sponsor
- Galderma R&D
Dermatitis, Atopic
Wool Clothing for the Management of Childhood Atopic Dermatitis (DESSINE2)
The study is a sequentially recruited, cross-over-cohort, outpatient-based evaluation of the effectiveness of wool clothing, as compared to standard clothing, in reducing the severity of childhood atopic dermatitis (eczema) over…
- Trial name
- DESSINE2 (NCT04011215)
- Testing
- wool clothing, standard clothing
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Murdoch Childrens Research Institute
Pyruvate Dehydrogenase Complex Deficiency
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency.
- Trial name
- NCT06340685
- Testing
- Triheptanoin
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Jirair Krikor Bedoyan
Hemophilia A
Health Related Quality of Life of Youth and Young Adults With Haemophilia A
Research question: Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis? A 12-month prospective open-label, single-arm…
- Trial name
- RELOQ (NCT04728217)
- Testing
- Efmoroctocog Alfa Injection [Eloctate]
- Where
- Russia
- Volunteers
- 30 people
- Sponsor
- The League of Clinical Research, Russia
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fabry Disease
A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
No investigational drug will be administered in this study for the treatment of Fabry disease. This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not…
- Trial name
- NCT01839526
- Testing
- Iohexol
- Where
- 22 sites in United States, Argentina and 12 more
- Volunteers
- 39 people
- Sponsor
- Genzyme, a Sanofi Company
Clubfoot
Clubfoot DNA Repository
To build a DNA repository to enable participation in ongoing and future Clubfoot genetic linkage studies.
- Trial name
- NCT00607191
- Where
- United States
- Volunteers
- 379 people
- Sponsor
- Texas Scottish Rite Hospital for Children
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Hearing Loss, Genetic Disease
Auditory Restoration and Outcomes After Gene Therapy and Cochlear Implantation: a PRospective Cohort Analysis (AURORA)
This cohort study aims to explore the trends and differences in multidimensional perceptual levels of patients after cochlear implants or gene therapy, as well as to comprehensively assess the efficacy of gene therapy for…
- Conditions
- Hearing Loss, Genetic Disease, Speech Perception
- Trial name
- NCT06237790
- Where
- 6 sites in China
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Eye & ENT Hospital of Fudan University