Clinical trials: Newborns
Pantothenate Kinase-Associated Neurodegeneration
CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product.
- Trial name
- NCT04182763
- Testing
- CoA-Z
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- Oregon Health and Science University
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Tuberous Sclerosis Complex
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to…
- Trial name
- NCT02849457
- Testing
- Early Vigabatrin
- Where
- 13 sites in United States
- Volunteers
- 84 people
- Sponsor
- Martina Bebin
Skin Disorder
Characterization of Pre-Term Neonatal Skin
The skin of pre-term neonates is not fully developed and often leads to trans-epidermal water loss, trouble regulating temperature, and increased risk of skin wounds.
- Trial name
- NCT05127044
- Testing
- Diffuse Reflectance Spectroscopy, BiliChek
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Huntington's Disease
Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease
The pace of basic science research defining the mechanisms of selective neuronal degeneration in Huntington disease (HD) has far exceeded the pace of translation of this information into clinically effective treatments for the…
- Trial name
- HD (NCT00670709)
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Fragile X Syndrome, FXS
A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome
This is a single-center, randomized, double-blind, 2-period crossover study to explore the effects of BPN14770 on cognitive function and behavior in subjects with Fragile X Syndrome.
- Conditions
- Fragile X Syndrome, FXS, Fra(X) Syndrome
- Trial name
- NCT03569631
- Testing
- BPN14770
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Tetra Discovery Partners
Sickle Cell Disease
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the…
- Trial name
- NCT00911495
- Testing
- GMI-1070
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- GlycoMimetics Incorporated
Atopic Dermatitis
Comparison Trial of OPA-15406 Ointment in Pediatric Patients With Atopic Dermatitis
To demonstrate the superiority of the investigational medicinal product (IMP; 0.3% OPA-15406 ointment, 1% OPA-15406 ointment, or vehicle) to the vehicle when administered twice daily for 4 weeks using success rate in…
- Trial name
- NCT03911401
- Testing
- 0.3% OPA-15406, 1% OPA-15406
- Where
- Japan
- Volunteers
- 251 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Obstetric Pain
Effect of Preemptive Epidural Analgesia in Labor on Cytokine Production
During labor there is an increased production of inflammatory mediators called cytokines. Higher concentration of certain cytokines has been linked to adverse neonatal and maternal outcomes.
- Trial name
- NCT00361712
- Testing
- Preemptive epidural analgesia, Standard of care
- Where
- Israel
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Rabin Medical Center
Alpha1-antitrypsin Deficiency
Aralast alpha1-proteinase Inhibitor Surveillance Study
The primary objectives of this Phase 4, open label, prospective U.S. surveillance study are to evaluate the health outcomes of Alpha 1-Antitrypsin (AAT)-deficient subjects who are initiating treatment with ARALAST on…
- Trial name
- NCT00313144
- Testing
- ARALAST Alpha1-Proteinase Inhibitor
- Where
- United States
- Volunteers
- 127 people
- Sponsor
- Baxalta now part of Shire
Anemia, Sickle Cell
A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic, in Healthy Volunteers
This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO)…
- Trial name
- NCT03926819
- Testing
- HBI-002
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Hillhurst Biopharmaceuticals, Inc.
Cystic Fibrosis
Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients
This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval.
- Trial name
- NCT01784419
- Testing
- ivacaftor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, San Francisco
Prader-Willi Syndrome, Hyperphagia
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).
- Trial name
- ZEPHYR (NCT03790865)
- Testing
- Livoletide
- Where
- 37 sites in United States, Australia and 6 more
- Volunteers
- 158 people
- Sponsor
- Millendo Therapeutics SAS
Duchenne Muscular Dystrophy
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
- Trial name
- MISSION (NCT02606136)
- Testing
- Pamrevlumab
- Where
- 10 sites in United States
- Volunteers
- 21 people
- Sponsor
- Kyntra Bio
Labor Pain
Effects of Ambulation During First Stage of Labour on Maternal and Neonatal Outcomes
This study investigates the effect of ambulation (walking) during first stage of labour on maternal and neonatal outcomes. In the intervention group women will be encouraged to ambulate and women in the control group will receive…
- Trial name
- NCT03447015
- Testing
- ambulation
- Where
- Jordan
- Volunteers
- 290 people, healthy volunteers welcome
- Sponsor
- Mutah University
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
to Test the Hypothesis That NHFOV is More Effective Than nCPAP in the Treatment of Respiratory Distress Syndrome (RDS) in Verypreterm Neonates
NHFOV as Primary Support in Very Preterm Infants With RDS
This will be a prospective, multi-center, two-arms,parallel, randomized, controlled trial with a superiority design,conducted in China.
- Trial name
- NCT05141435
- Testing
- infants receive primary non-invasive respiratory support by mean of nCPAP, infants receive primary non-invasive respiratory support by mean of NHFOV
- Where
- 3 sites in China
- Volunteers
- 360 people
- Sponsor
- Jiulongpo No.1 People's Hospital
Sickle Cell Disease
Comparing Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease
The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment.
- Trial name
- COMPARE-VOE (NCT03933397)
- Testing
- Patient-Specific Protocol, Weight-based Protocol, Morphine
- Where
- 6 sites in United States
- Volunteers
- 328 people
- Sponsor
- Duke University
Atopic Dermatitis
A Study Investigating the Effect of EDP1815 in the Treatment of Mild, Moderate and Severe Atopic Dermatitis
The purpose of this research study is to determine whether the study drug, EDP1815, is safe and effective in the treatment of atopic dermatitis compared with placebo.
- Trial name
- NCT05121480
- Testing
- EDP1815
- Where
- 59 sites in United States, Australia and 4 more
- Volunteers
- 421 people
- Sponsor
- Evelo Biosciences, Inc.
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