Clinical trials: Neonatal Diseases and Abnormalities
Krabbe Disease
Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease
This study is designed to learn about early brain development in children with Krabbe disease, and to use diffusion tensor imaging as an early diagnostic tool to identify newborns at risk for the disease.
- Trial name
- NCT00787865
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Ataxia-Telangiectasia (A-T)
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.
- Trial name
- NCT07380165
- Testing
- Levacetylleucine
- Sponsor
- IntraBio Inc
Erythropoietic Protoporphyria (EPP)
GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria
A Phase 2a study of PORT-77 in adults with erythropoietic protoporphyria (EPP)
- Trial name
- EPP (NCT06971900)
- Testing
- PORT-77
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Portal Therapeutics, Inc.
Idiopathic Clubfoot (Talipes Equinovarus)
Efficacy of Botox in Patients With Idiopathic Clubfoot
The purpose of this study was to determine the efficacy of adding Botox injection to serial manipulations and castings in patients with clubfoot.
- Trial name
- NCT00152334
- Testing
- Botox
- Where
- Canada
- Volunteers
- 500 people
- Sponsor
- University of British Columbia
Dermatitis, Atopic
Study to Evaluate the Use of RHT-3201 in SCORAD Reduction in Young Patients With Atopic Dermatitis
The study's objective is to confirm that RHT-3201 reduces the signs and symptoms of moderate atopic dermatitis determined by SCORAD, in patients aged 1 to 12 years, as compared to placebo.
- Trial name
- NCT03907228
- Testing
- RHT-3201
- Where
- South Korea
- Volunteers
- 102 people
- Sponsor
- IlDong Pharmaceutical Co Ltd
Hyperbilirubinemia, Erythroblastosis, Fetal
Efficacy of High-dose Intravenous Immunoglobulin Therapy for Hyperbilirubinemia Due Rh Hemolytic Disease
The use of intravenous immunoglobulin G (IVIG) therapy has been reported in hyperbilirubinemia of Rh hemolytic disease but we don't have enough evidences for it.
- Trial name
- NCT00288600
- Testing
- Intravenous Immunoglobulin, Normal saline solution
- Where
- Brazil
- Volunteers
- 92 people
- Sponsor
- Oswaldo Cruz Foundation
X-linked Adrenoleukodystrophy
SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)
X-linked Adrenoleukodystrophy (X-ALD) is one of the most frequent inborn error of metabolism caused by mutations in the ABCD1 gene, which codes for the transporter of saturated very long-chain fatty acids (VLCFA) for peroxisomal…
- Trial name
- NCT04687007
- Testing
- SMART-ALD, Waiting list
- Where
- 2 sites in Germany
- Volunteers
- 68 people
- Sponsor
- Leipzig University Medical Center
Atopic Dermatitis
A Pharmacokinetics and Safety Study of Nemolizumab in Adolescent Participants With Atopic Dermatitis (AD)
The purpose of this study was to evaluate the pharmacokinetics and safety of nemolizumab in adolescent participants with AD.
- Trial name
- NCT03921411
- Testing
- Nemolizumab
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- Galderma R&D
Thalassemia Major
Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase
Luspatercept represents the first and only erythroid maturation agent (EMA) approved by the European Commission (EC) and the Food and Drug Administration (FDA) capable of enhancing advanced erythrocyte maturation.
- Trial name
- LUSPA001 (NCT06913634)
- Testing
- Luspatercept (ACE-536)
- Where
- Italy
- Volunteers
- 140 people
- Sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Diabetes Mellitus Type 1, Diabetes Mellitus Type 2
Assessment of the Effects of a DPP-4 Inhibitor (Sitagliptin) Januvia on Immune Function in Healthy Individuals
Patients with diabetes have high blood sugar levels (hyperglycemia) because pancreatic beta-cells no longer produce sufficient insulin.
- Trial name
- NCT00813228
- Testing
- Sitagliptin (Januvia)
- Where
- United States
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Patent Ductus Arteriosus
PDA Post NICU Discharge
The purpose of this study is to track post-discharge outcomes on prematurely born infants who are discharged from the NICU with a patent ductus arteriosus (PDA).
- Trial name
- PDA (NCT02750228)
- Where
- 16 sites in United States
- Volunteers
- 208 people
- Sponsor
- Pediatrix
Atopic Dermatitis (AD), Atopic Dermatitis (Eczema)
Moisturization and Skin Hydration Study
Skin moisturization is important for patients with atopic dermatitis, commonly known as eczema. Moisturizing right after bathing is widely accepted as the best method to keep skin hydrated.
- Trial name
- NCT07270965
- Testing
- Moisturizing while in shower/bath, Moisturizing after towel drying
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- University of British Columbia
Cystic Fibrosis (CF)
Safety, Tolerability and Efficacy of MP-376 Given for 28 Days to Cystic Fibrosis (CF) Patients
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT00677365
- Testing
- MP-376
- Where
- 50 sites in United States, Germany and 1 more
- Volunteers
- 151 people
- Sponsor
- Amgen
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Study Evaluating rFIX; BeneFIX® in Hemophilia B
The primary objective of this clinical research study is to assess the safety and efficacy of rFIX for a minimum of 6 months in previously treated patients (PTPs) with hemophilia B (FIX:C ≤2%) during standard-of-care treatment…
- Trial name
- NCT00093171
- Testing
- rFIX
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Healthy Volunteers, Vascular Ehlers Danlos Syndrome
Non-Invasive Quantitative Imaging of Human Local Arterial Wall Elasticity Using Supersonic Shear Imaging
Arterial stiffness is associated with increased risk of cardiovascular events and death. Most of existing technique need dedicated device for arterial stiffness evaluation and indirect calculation of intra arterial pressure.
- Trial name
- ULTRAFASTECHO (NCT01096264)
- Testing
- SSI and SphygmoCor® evaluations
- Where
- France
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- French Cardiology Society
Porphyria
Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks
OBJECTIVES: Assess whether chronic administration of gonadotropin-releasing hormone analogues is safe and effective for the prevention of cyclic attacks of acute porphyria in women.
- Trial name
- NCT00004330
- Testing
- luteinizing hormone-releasing factor
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Intrauterine Growth Restriction
Evaluation of Infants With Intrauterine Growth Restriction
Purpose: Clinical assessment (anthropometric) and paraclinical (biochemical and immunological by dosing serum insuline growth factors IGF1 and IGF2 and their receptors) of neonates with intrauterine growth restriction (IUGR) and…
- Trial name
- ModeInfIUGR (NCT02515292)
- Where
- Romania
- Volunteers
- 60 people
- Sponsor
- Iuliu Hatieganu University of Medicine and Pharmacy