Clinical trials: Neonate
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.
Urea Cycle Disorders
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
- Trial name
- NCT02489292
- Testing
- HepaStem
- Where
- 6 sites in Belgium, France and 2 more
- Volunteers
- 5 people
- Sponsor
- Cellaion SA
Sickle Cell Disease
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine).
- Trial name
- ASCENT1 (NCT05405114)
- Testing
- NDec - oral decitabine-tetrahydrouridine, HU - Hydroxyurea
- Where
- 118 sites in United States, Canada and 12 more
- Volunteers
- 96 people
- Sponsor
- Novo Nordisk A/S
Hereditary Angioedema
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05392114
- Testing
- Donidalorsen
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 154 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Mucopolysaccharidosis I
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
- Trial name
- NCT02597114
- Testing
- AGT-181
- Volunteers
- 3 people
- Sponsor
- ArmaGen, Inc
Cystic Fibrosis, Motor Neuron Disease
MN4000 for Treatment of CF and MND Patients in the Home Setting
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
- Conditions
- Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
- Trial name
- NCT03797677
- Testing
- MN 4000
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Hill-Rom
Endometrial Cancer
Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer
This is a Phase 2, multicenter, open-label, single-arm study to evaluate the efficacy and safety of sapanisertib and serabelisib (PIKTOR) with paclitaxel in participants with advanced or recurrent endometrial cancer.
- Trial name
- NCT06463028
- Testing
- Sapanisertib, Serabelisib, Paclitaxel
- Where
- 18 sites in United States
- Volunteers
- 40 people
- Sponsor
- Faeth Therapeutics
Hereditary Angioedema
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
- Trial name
- CHANGE 3 (NCT00462709)
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 48 sites in United States
- Volunteers
- 146 people
- Sponsor
- Shire
Dyslipidemias, High Cholesterol
Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
This study will be a placebo-controlled, double-blind, randomized, phase 3 study to Evaluate the Efficacy, Safety, and Tolerability of Obicetrapib in Participants with a History of Heterozygous Familial Hypercholesterolemia…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROOKLYN (NCT05425745)
- Testing
- Obicetrapib
- Where
- 96 sites in United States, Canada and 8 more
- Volunteers
- 354 people
- Sponsor
- NewAmsterdam Pharma
Dermatitis, Atopic
Safety and Efficacy of AN2728 Topical Ointment, 2% in Children, Adolescents, and Adults (Aged 2 Years and Older) With Atopic Dermatitis
The purpose of this study is to investigate the safety and efficacy of AN2728 Topical Ointment, 2% in children, adolescents, and adults (ages 2 years and older) with atopic dermatitis.
- Trial name
- NCT02118792
- Testing
- AN2728 Topical Ointment, 2%, Matching vehicle control
- Where
- United States
- Volunteers
- 764 people
- Sponsor
- Pfizer
Primary Immunodeficiency Disease
Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID
This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID)
- Trial name
- NCT02810444
- Testing
- IgG Next Generation (BT595)
- Where
- 19 sites in United States, Germany and 3 more
- Volunteers
- 81 people
- Sponsor
- Biotest
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Pompe Disease
Extension Study for Patients Who Have Participated in a BMN 701 Study
This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.
- Trial name
- NCT01435772
- Testing
- BMN 701
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Electroencephalographic Neonatal Seizures
A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures
The purpose of the study is to evaluate the pharmacokinetics (PK) of brivaracetam (BRV) in neonates who have seizures that are not adequately controlled with previous antiepileptic drug (AED) treatment, and to identify the…
- Trial name
- NCT03325439
- Testing
- Brivaracetam (BRV) intravenous (iv), Brivaracetam (BRV) oral
- Where
- 12 sites in Belgium, Czechia and 5 more
- Volunteers
- 9 people
- Sponsor
- UCB Biopharma SRL
Non Small Cell Lung Cancer (NSCLC), Squamous Cell Carcinoma of the Head and Neck (SCCHN)
Study of PX-866 and Docetaxel in Solid Tumors
Phase 1: To determine the maximally tolerated dose (MTD) or recommended dose (RD) and any potential efficacy of PX-866 in combination with docetaxel in patients with solid tumors.
- Trial name
- NCT01204099
- Testing
- Docetaxel, PX-866
- Where
- 33 sites in United States, Canada
- Volunteers
- 223 people
- Sponsor
- Cascadian Therapeutics Inc.
Congenital Heart Defect
Post-market Clinical Follow-up Study With Magnetic Resonance Imaging Conditional Guide Wire
This is post-market clinical follow-up study on 25 consecutive patients in two centers to evaluate the safety and performance of magnetic resonance imaging conditional guide wire and the acceptability of identified risks in the…
- Trial name
- MRWIREPMCF (NCT02493634)
- Testing
- pressure gradient measurement, MRWire
- Where
- 3 sites in Germany, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Nano4Imaging GmbH
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
Regional Saturation of Oxygen (rSO2)
Performance Comparison of Masimo O3 Regional Oximetry Sensors in Neonates
The aim of the study is to compare the performance of the O3 cerebral oximeter and sensor to a 510(k) cleared NIRS (Near-Infrared Spectroscopy) device on neonatal subjects based on data collected from the cutaneously placed…
- Trial name
- NCT03828487
- Testing
- Masimo O3 Neonatal Sensors, 510(k) cleared sensor
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Masimo Corporation
Fabry Disease
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
- Trial name
- NCT06270316
- Testing
- AMT-191
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- UniQure Biopharma B.V.