Clinical trials: Newborn Diseases
Sickle Cell Anemia, Children, Only
Shared-Decision Making for Hydroxyurea
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent…
- Trial name
- ENGAGE-HU (NCT03442114)
- Testing
- Hydroxyurea SDM Toolkit, Clinician Pocket Guide
- Where
- 11 sites in United States
- Volunteers
- 176 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Glycogen Storage Disease VI, GLYCOGEN STORAGE DISEASE IXa1
GSD VI and GSD IX Natural History
Collection and review of clinical information related to Glycogen Storage Disease Type VI (GSD VI) OR Glycogen Storage Disease Type IX (GSD IX) generated during clinic visits.
- Conditions
- Glycogen Storage Disease VI, GLYCOGEN STORAGE DISEASE IXa1, GLYCOGEN STORAGE DISEASE IXa2, Glycogen Storage Disease IXB
- Trial name
- NCT04454216
- Testing
- No intervention
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- Duke University
Asthma
A Study to Evaluate the Safety, Tolerability and Absorption to the Blood After Administration of Single and Multiple Doses of AZD8154 in Healthy Participants.
This study is a Phase 1, first in human (FiH) study, consisting of 3 parts (Part 1, Part 2, and Part 3) in healthy male and female participants of non-childbearing potential, performed at a single study center.
- Trial name
- NCT03436316
- Testing
- AZD8154
- Where
- Germany
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
Osteotomy of Lower Extremity
PediLoc® Blade Plate in Distal Femoral Osteotomies
Background: The PediLoc® Locking Cannulated Blade Plate System, originally designed for proximal femoral osteotomies, has also been used for multidimensional deformity correction of the distal femur.
- Trial name
- NCT06950047
- Testing
- distal femoral osteotomies using the PediLoc® Locking Cannulated Blade Plate System, distal femoral osteotomies using the 90°LCP-PCP
- Where
- Switzerland
- Volunteers
- 111 people
- Sponsor
- University Children's Hospital, Zurich
Lactose Intolerance
Efficacy, Safety, and Tolerability Study of RP-G28 in Subjects With Lactose Intolerance
RP-G28 is being investigated for treatment of moderate to severe lactose intolerance and its potential to improve the tolerance of lactose (dairy products).
- Trial name
- NCT02673749
- Testing
- RP-G28
- Where
- United States
- Volunteers
- 377 people
- Sponsor
- Ritter Pharmaceuticals, Inc.
Giant Axonal Neuropathy, Gene Transfer
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly.
- Trial name
- NCT02362438
- Testing
- scAAv9/JeT-GAN
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cardiovascular Diseases, Defect, Congenital Heart
Family Study of Congenital Cardiovascular Malformations
To determine genetic mechanisms responsible for congenital cardiovascular malformations.
- Conditions
- Cardiovascular Diseases, Defect, Congenital Heart, Heart Diseases
- Trial name
- NCT00005258
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Total Anomalous Pulmonary Venous Connection
Sutureless Technique and Conventional Repairs for Total Anomalous Pulmonary Venous Connection
The purpose of this study is to evaluate the efficacy of conventional repair and sutureless surgical repair of total anomalous pulmonary venous connection.
- Trial name
- NCT04234711
- Testing
- Surgical strategy
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Guangdong Provincial People's Hospital
Corneal Dystrophies, Hereditary
Descemet Membrane Endothelial Keratoplasty (DMEK)
To evaluate isolated Descemet membrane transplantation as a refinement of previous advanced techniques for corneal transplantation, like Descemet Stripping Endothelial Keratoplasty (DSEK)
- Trial name
- DMEK (NCT00521898)
- Testing
- Descemet Membrane Endothelial Keratoplasty (DMEK)
- Where
- Netherlands
- Volunteers
- 1,000 people
- Sponsor
- Netherlands Institute for Innovative Ocular Surgery
Hereditary Angioedema (HAE)
Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers.
- Trial name
- NCT02663687
- Testing
- Recombinant human C1 esterase inhibitor, SHP623
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Shire
Noonan Syndrome
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested…
- Trial name
- NCT00351221
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Insmed Incorporated
Arterio-venous Fistula
Invasive Arterio-Venous Fistula Blood Pressure Monitoring & Fluid Dynamics Study
The surgical formation of an AVF offers a unique example of vascular remodelling and adaption. Yet, the specific factors which elicit remodelling events which determine successful maturation or failure have not been unambiguously…
- Trial name
- NCT04293614
- Where
- Ireland
- Volunteers
- 12 people
- Sponsor
- University Hospital of Limerick
Hereditary Ataxia
Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)
OBJECTIVES: I. Clinically evaluate members from families with a dominantly inherited ataxia and collect blood, skin and muscle samples for detailed molecular studies. II.
- Trial name
- NCT00004306
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Office of Rare Diseases (ORD)
Recurrent Colon Cancer, Extensive Stage Small Cell Lung Cancer
Vaccine Therapy in Treating Patients With Colon, Pancreatic, or Lung Cancer
RATIONALE: Vaccines made from mutated ras peptides may make the body build an immune response to and kill tumor cells. PURPOSE: Phase I trial to study the effectiveness of a vaccine containing mutated ras peptides and an immune…
- Conditions
- Recurrent Colon Cancer, Extensive Stage Small Cell Lung Cancer, Stage III Pancreatic Cancer, Stage III Rectal Cancer
- Trial name
- NCT00019006
- Testing
- Detox-B adjuvant, ras peptide cancer vaccine
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
Congenital Bleeding Disorder, Haemophilia A
Study for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A Patients
The study has descriptive purposes, with aim of assessing how turoctocog alfa is used in the everyday practice and to provide a baseline for the management of haemophilia A and does not involve any change in the clinical…
- Trial name
- NCT06574984
- Testing
- Turoctocog alfa
- Where
- 7 sites in Iraq
- Volunteers
- 329 people
- Sponsor
- Novo Nordisk A/S
Fabry Disease
A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
GR181413A/AT1001 (migalastat hydrochloride) is a low molecular weight iminosugar, an analog of the terminal galactose group that is cleaved from the substrate GL-3.
- Trial name
- NCT01853852
- Testing
- GR181413A/AT1001 solution, GR181413A/AT1001 capsule, Potable water
- Where
- Australia
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Turner Syndrome
A Study to Evaluate Subjects With Turner Syndrome Treated With Growth Hormone
This study is a multicenter, open-label, observational, postmarketing surveillance study of Genentech growth hormone (GH) products in the treatment of girls with Turner syndrome in the United States and Canada.
- Trial name
- NCT00097552
- Volunteers
- 1,696 people
- Sponsor
- Genentech, Inc.
Ornithine Transcarbamylase Deficiency
Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g.
- Trial name
- NCT04442347
- Testing
- ARCT-810
- Where
- 8 sites in United States
- Volunteers
- 16 people
- Sponsor
- Arcturus Therapeutics, Inc.
Atopic Dermatitis
Combination Therapy for Atopic Dermatitis
Atopic dermatitis is a chronic relapsing disease with acute flares. The standard therapy is to treat acute flares using topical medications.
- Trial name
- NCT00119158
- Testing
- Combination of pimecrolimus and fluticasone, pimecrolimus
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- Children's Hospital of Philadelphia
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