Clinical trials: Newborn Diseases
Phenylketonuria (PKU)
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the…
- Trial name
- PALace (NCT05813678)
- Testing
- Pegvaliase
- Where
- 26 sites in United States, Germany and 1 more
- Volunteers
- 450 people
- Sponsor
- BioMarin Pharmaceutical
Methylmalonic Acidemia, Propionic Acidemia
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
The JUMP (Journey to Understand MMA and PA) Study is being conducted by HemoShear Therapeutics and AllStripes, a rare disease online research platform.
- Trial name
- JUMP (NCT05438485)
- Testing
- Registry
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 11 people
- Sponsor
- HemoShear Therapeutics
Seizures, Encephalopathy
The Impact of Electroencephalographic (EEG) Seizure Treatment in Neonatal Encephalopathy
This is a prospective, randomized, single-center pilot trial of term and near term (≥ 36 weeks gestation) infants with encephalopathy or seizures comparing a "EEG Seizure Treatment Group" or ESG with a "Clinical Seizure Treatment…
- Trial name
- NCT01027715
- Testing
- EEG monitoring and treatment of EEG seizures
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Washington University School of Medicine
Retinitis Pigmentosa
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa.
- Trial name
- NCT06291935
- Testing
- VG901
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- VeonGen Therapeutics GmbH
Newborn Bathing, Primiparous Pregnant Women
The Effects of Video-Based and Practical Newborn Bathing Education Provided to Primiparous Pregnant Women During the Third Trimester on Knowledge, Anxiety, and Bonding During the First Bath: A Randomized Controlled Trial
This study will be conducted in a randomized controlled experimental research design with pretest, posttest control group.
- Conditions
- Newborn Bathing, Primiparous Pregnant Women, Anxiety, Bonding
- Trial name
- NewbornBathing (NCT06661408)
- Testing
- Intervention group 1, Intervention group 2
- Where
- Turkey (Türkiye)
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Kahramanmaras Sutcu Imam University
Pruritus, Atopic Dermatitis
Study to Assess the Clinical Activity of a Sequential Dose of REGN846 on Refractory Pruritus in Patients With Atopic Dermatitis
The purpose of this study is to assess the clinical activity on refractory moderate-to-severe pruritus of a single intravenous (IV) dose of REGN846 in adult patients with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01605708
- Testing
- REGN846
- Where
- 14 sites in Finland, Germany and 1 more
- Volunteers
- 19 people
- Sponsor
- Regeneron Pharmaceuticals
Foramen Ovale, Patent
Risk of Ischaemic Stroke After Pulmonary Embolism in Patients With and Without Patent Foramen Ovale
stroke's risk is increased in patients with pulmonary embolism and PFO compared to patients without PFO. Does this increased risk persist years after pulmonary embolism ?
- Trial name
- EPIC-FOPCohort (NCT04987008)
- Where
- 3 sites in France
- Volunteers
- 53 people
- Sponsor
- University Hospital, Brest
Osteogenesis Imperfecta
Osteogenesis Imperfecta Blood Pressure Study
Osteogenesis Imperfecta (OI) is a genetic disorder caused by mutations to the alpha-1 or alpha-2 chain of type I collagen. Clinically, the disorder is characterized by bones that fracture easily, often from little or no apparent…
- Trial name
- NCT04169568
- Testing
- Manual cuff blood pressure
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Nemours Children's Clinic
Osteogenesis Imperfecta
Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study
Osteogenesis imperfecta (OI) is a genetic disease for which there is currently no known cure. OI causes the osteoblasts (bone-forming cells in the body) to grow poorly, which slows the growth of children with the disease and…
- Trial name
- NCT00187018
- Testing
- Bone marrow transplant
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- St. Jude Children's Research Hospital
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
This is a first-in-human (FIH), Phase 1/2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD.
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Trial name
- NCT06754852
- Testing
- HMB-002 (Part A), HMB-002 (Part B), HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)
- Where
- 25 sites in United States, Australia and 1 more
- Volunteers
- 108 people
- Sponsor
- Hemab ApS
Phenylketonurias
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria.
- Trial name
- NCT00104247
- Testing
- sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
- Where
- 13 sites in United States
- Volunteers
- 89 people
- Sponsor
- BioMarin Pharmaceutical
Urinary Incontinence, Menopause
The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence
This study aimed to determine the effects of Kegel exercise and Ba Duan Jin applications applied to premenopausal women with urinary incontinence on quality of life and psychological well-being.
- Conditions
- Urinary Incontinence, Menopause, Wolman Disease, Quality of Life
- Trial name
- NCT06287658
- Testing
- Training on Kegel exercise and Ba Duan Jin applications online, training on Kegel exercises and Ba Duan Jin applications after the research is completed
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Ondokuz Mayıs University
Nosocomial Infection
New Dosages of Inflammatory Markers for the Early Diagnosis of Nosocomial Bacterial Infections of the Newborn
This study aims to evaluate new dosage of inflammatory markers or new inflammatory markers for the diagnosis of nosocomial bacterial infection.
- Trial name
- NOSODIAG (NCT00701948)
- Where
- 2 sites in France
- Volunteers
- 240 people
- Sponsor
- University Hospital, Strasbourg, France
Parents of Preterm Infants Born Before 33 Weeks Gestation
PPrime: Parents Views on Diagnosis and Information-giving in Neonatal Care (Pilot Study)
The aim of the pilot study is to explore and identify the issues of concern to parents whose babies have been cared for in a neonatal unit and exposed to imaging, its influence on diagnosis and information-giving process
- Trial name
- PPrime (NCT01055548)
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Imperial College London
Alport Syndrome
Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1)
This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome.
- Trial name
- ALPESTRIA-1 (NCT06425055)
- Testing
- Vonafexor
- Where
- 13 sites in United States, France and 1 more
- Volunteers
- 26 people
- Sponsor
- Enyo Pharma
Nephropathic Cystinosis, Cystinosis
Pilot Study of TTI-0102 in Cystinosis
White blood cell (WBC) cystine levels serve as the primary therapeutic target for monitoring cysteamine treatment in cystinosis, with a goal of <1.9 nmol ½ cystine/mg protein as measured 5-6 hours post-dose when treated with…
- Trial name
- NCT07837726
- Testing
- TTI-0102 (cysteamine-pantetheine disulfide)
- Volunteers
- 6 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Retinitis Pigmentosa (RP)
Retrospective Natural History Study of Retinitis Pigmentosa
This is natural history study of rods and cones degenerations in patients with Retinitis Pigmentosa (RP) caused by pathogenic mutations in RHO, PDE6A or PDE6B gene mutations.
- Trial name
- PHENOROD1 (NCT03975543)
- Where
- France
- Volunteers
- 113 people
- Sponsor
- SparingVision
Depressive Symptoms, Perinatal Loss
e-Perinatal Butterfly: An Embedded Study for Parents Experiencing Perinatal Loss
This study is part of the e-Perinatal project and is embedded within the parent e-Perinatal cluster randomized controlled trial (NCT07613008). No additional randomization is performed for this embedded study.
- Conditions
- Depressive Symptoms, Perinatal Loss, Perinatal Grief, Anxiety Symptoms
- Trial name
- e-Butterfly (NCT07898176)
- Testing
- e-Perinatal Butterfly, Standard routine maternal care
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Seville
Alpha-Mannosidosis
Evaluation of Long-term Efficacy of Treatment With Lamazym
The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently receiving the treatment according to the AfterCare…
- Trial name
- rhLAMAN-10 (NCT02478840)
- Testing
- Lamazym
- Where
- Denmark
- Volunteers
- 18 people
- Sponsor
- Zymenex A/S
Duchenne Muscular Dystrophy
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen…
- Trial name
- EVOLVE (NCT06606340)
- Testing
- Eteplirsen, Golodirsen, Casimersen
- Where
- 23 sites in United States
- Volunteers
- 300 people
- Sponsor
- Sarepta Therapeutics, Inc.
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