Clinical trials: Newborn
Fabry Disease
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
- Trial name
- NCT01124643
- Testing
- Replagal
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Shire
Burden, Caregiver
Social Support and Stress Reduction for Caregivers of Young Adults With IDD
The objective of this study is to evaluate the feasibility and stress reduction impacts of a yoga and support group intervention on caregivers of individuals with intellectual and developmental disabilities.
- Trial name
- CareWell (NCT05938998)
- Testing
- Yoga; Support Group
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Sickle Cell Anemia, Sickle ß0-Thalassemia
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT01179217
- Testing
- L-glutamine
- Where
- 31 sites in United States
- Volunteers
- 230 people
- Sponsor
- Emmaus Medical, Inc.
Hemophilia A
Study Evaluating The Safety Of Xyntha In Usual Care Settings
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year…
- Trial name
- NCT00765726
- Testing
- Moroctocog alfa(AF-CC), Blood draw for laboratory testing
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 12 people
- Sponsor
- Pfizer
Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants…
- Trial name
- NCT03959189
- Testing
- ERX-963
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Expansion Therapeutics, Inc.
Atopic Dermatitis
A Study of Lebrikizumab (LY3650150) With/Without Topical Corticosteroid Treatment in Participants With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to evaluate the efficacy and safety of lebrikizumab with/without Topical Corticosteroid Treatment in Participants with Moderate-to-Severe Atopic Dermatitis.
- Trial name
- NCT06280716
- Testing
- Lebrikizumab, Topical Corticosteroid
- Where
- 46 sites in China, South Korea
- Volunteers
- 301 people
- Sponsor
- Eli Lilly and Company
Premature Birth of Newborn
Supporting and Enhancing NICU Sensory Experiences (SENSE)
Seventy preterm infants born less than or equal to 32 weeks gestation were put into either the sensory-based intervention (experiment) group or traditional care (control) group. Consecutive admissions at St.
- Trial name
- SENSE (NCT03316547)
- Testing
- SENSE Program
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Cystic Fibrosis, Bronchiectasis Adult
Bacille Calmette-Guerin (BCG) Vaccine for Immune Protection Against Infections
This is pilot study of the immunologic effects of intradermal Bacille Calmette-Guerin (BCG) vaccination of adults with cystic fibrosis (CF), non-CF bronchiectasis (NCFB), and healthy volunteers.
- Conditions
- Cystic Fibrosis, Bronchiectasis Adult, Non-Tuberculous Mycobacteria
- Trial name
- NCT04884308
- Testing
- BCG TICE Vaccine
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Familial Hypercholesterolemia
Atorvastatin Three Year Pediatric Study
The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial…
- Trial name
- NCT00827606
- Testing
- atorvastatin
- Where
- 40 sites in United States, Belgium and 13 more
- Volunteers
- 272 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Duarte Galactosemia
Intervention and Outcomes in Duarte Galactosemia
The purpose of this study is to learn about Duarte galactosemia (DG). This study will examine the possible effects of Duarte galactosemia (DG) in children, and determine whether dietary exposure to milk in infancy or early…
- Trial name
- NCT02519504
- Where
- United States
- Volunteers
- 566 people, healthy volunteers welcome
- Sponsor
- Emory University
Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy
This study will test the safety and efficacy of an infusion of a baby's own (autologous) umbilical cord blood as compared with placebo in babies born with history and signs of hypoxic-ischemic brain injury.
- Trial name
- (HIE) (NCT02612155)
- Testing
- Infusion of autologous cord blood
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Michael Cotten
Hemophilia A
PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for…
- Trial name
- NCT02492984
- Testing
- Intravenous infusions of Xyntha
- Where
- 15 sites in China
- Volunteers
- 85 people
- Sponsor
- Pfizer
Tourette Syndrome
Thalamic Deep Brain Stimulation for the Treatment of Refractory Tourette Syndrome
This research is being performed to try to understand if the use of deep brain stimulation or DBS can treat the symptoms of Tourette syndrome that do not respond well to current medications.
- Trial name
- NCT01817517
- Testing
- Medtronic Activa Deep Brain Stimulation System
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Johns Hopkins University
Molybdenum Cofactor Deficiency, Type A
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A
- Trial name
- NCT02629393
- Testing
- ORGN001 (formerly ALXN1101)
- Where
- 11 sites in United States, Israel and 4 more
- Volunteers
- 5 people
- Sponsor
- Origin Biosciences
Epidermal Nevus Syndrome
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
KRN23 is a fully human immunoglobulin monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels.
- Trial name
- ENS (NCT04320316)
- Testing
- Crysvita (burosumab-twza) Treatment
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Short Stature Born Small for Gestational Age (SGA)
Long Term Study of Genotropin (Somatropin) for Short Children Born Small for Gestational Age (SGA)
To assess the long-term safety of Genotropin(somatropin) on Small for Gestational Age (SGA) without epiphyseal closing.
- Trial name
- NCT01859949
- Testing
- Genotropin (somatropin)
- Where
- 19 sites in Japan
- Volunteers
- 62 people
- Sponsor
- Pfizer
Heart Defects, Congenital
Safety and Performance of a Vascular Patch in Pediatric Patients Undergoing Bidirectional Cava-pulmonary Anastomosis
Xeltis developed biodegradable patch prosthesis, the Vascular Patch Model COR-VP-001, to be used as a vascular patch to augment the pulmonary trunk and pulmonary arteries.
- Trial name
- XEL-CR-06 (NCT02377700)
- Testing
- Xeltis Vascular Patch, Model COR-VP-001
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Xeltis
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase.
- Trial name
- NCT02279498
- Testing
- Liprotamase, porcine (pig) PERT
- Where
- 54 sites in United States, Canada and 5 more
- Volunteers
- 128 people
- Sponsor
- Anthera Pharmaceuticals
Nurse's Role, Stigma, Social
Testing an Arts-based Program to Reduce Nurse Stigma Towards Perinatal Substance Use
The goal of this pilot clinical trial is to evaluate the feasibility and limited effectiveness of a digital, arts-based educational intervention addressing nurse stigma towards perinatal substance use.
- Conditions
- Nurse's Role, Stigma, Social, Implicit Bias
- Trial name
- NCT06410287
- Testing
- ArtSpective
- Where
- 2 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Atopic Dermatitis
Study of Dupilumab in Adult Patients With Extrinsic Moderate-to-Severe Atopic Dermatitis
The primary objective was to assess the clinical efficacy of repeated subcutaneous (SC) doses of Dupilumab in adult participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01548404
- Testing
- Dupilumab
- Where
- 21 sites in Czechia, France and 3 more
- Volunteers
- 109 people
- Sponsor
- Regeneron Pharmaceuticals
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