Clinical trials: Neonatal Disorder
Congenital Heart Disease
ABL90 FP MC Neonatal Arterial, Venous, Capillary Blood
The goal of this clinical trial is to validate performance claims for method comparison for ABL90 FLEX PLUS in heparinized neonatal arterial, venous, and capillary whole blood for ctBil and FHbF in a Point of Care (POC) setting.
- Trial name
- NCT06078943
- Testing
- method comparison
- Where
- United States
- Volunteers
- 612 people, healthy volunteers welcome
- Sponsor
- Radiometer Medical ApS
Retinopathy of Prematurity
Study to Assess the Efficacy, Safety, and Tolerability of Intravitreal Aflibercept Compared to Laser Photocoagulation in Patients With Retinopathy of Prematurity
The primary objective of the study is to assess the efficacy of aflibercept compared to laser in patients diagnosed with retinopathy of prematurity (ROP).
- Trial name
- BUTTERFLEYE (NCT04101721)
- Testing
- aflibercept, laser photocoagulation
- Where
- 52 sites in United States, Bulgaria and 11 more
- Volunteers
- 127 people
- Sponsor
- Regeneron Pharmaceuticals
Cystic Fibrosis
"Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (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
- NCT00840333
- Testing
- MP-376 (Levofloxacin solution for Inhalation)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Amgen
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
Retinitis Pigmentosa (RP)
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
The purpose of this study is: To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
- Trial name
- NCT01543906
- Testing
- QLT091001
- Where
- 2 sites in Canada, Ireland
- Volunteers
- 5 people
- Sponsor
- QLT Inc.
Hereditary Angioedema
A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children
The main aims of this study are to learn how lanadelumab moves through a child's body and if the children have any medical problems from lanadelumab.
- Trial name
- SPRING (NCT04070326)
- Testing
- Lanadelumab
- Where
- 17 sites in United States, Canada and 3 more
- Volunteers
- 21 people
- Sponsor
- Shire
Healthy Volunteers
A Study to Assess the Pharmacokinetics and Safety of Co-administered Oral Galicaftor, Navocaftor, and ABBV-576 in Healthy Adults for the Treatment of Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40.
- Trial name
- ABBV-576 DDI (NCT05530278)
- Testing
- Galicaftor, ABBV-576, Navocaftor
- Where
- 4 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- AbbVie
Leber Hereditary Optic Neuropathy (LHON)
Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON).
- Trial name
- NCT01892943
- Where
- 8 sites in Belgium, Denmark and 3 more
- Volunteers
- 306 people
- Sponsor
- Santhera Pharmaceuticals
Generalized Lipodystrophy
Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or…
- Trial name
- NCT06502990
- Testing
- Metreleptin
- Where
- 12 sites in Belgium, France and 2 more
- Volunteers
- 1 people
- Sponsor
- Amryt Pharma
Atopic Dermatitis
Immunomodulation by OM-85 (Broncho-Vaxom) in Early AD
Clinical data suggest that treatment with OM-85, by inducing an early contact with bacterial extracts, could modulate the immunity of children with Atopic Dermatitis, and thus play an active role in the treatment of Atopic…
- Trial name
- NCT05222516
- Testing
- Broncho-Vaxom
- Where
- 28 sites in France, Germany and 2 more
- Volunteers
- 63 people
- Sponsor
- OM Pharma SA
Fabry Disease
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration.
- Trial name
- NCT04519749
- Testing
- 4D-310
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- 4D Molecular Therapeutics
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of CMK389 in Patients With Moderate to Severe Atopic Dermatitis.
The main purpose of this phase 2 study was to assess the efficacy and safety of CMK389 in patients with atopic dermatitis.
- Trial name
- NCT04836858
- Testing
- CMK389
- Where
- 18 sites in Czechia, France and 4 more
- Volunteers
- 71 people
- Sponsor
- Novartis Pharmaceuticals
Huntington's Disease
A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease
The purpose of the study is to collect and assess long term data on the safety, tolerability, and efficacy of pridopidine in patients with Huntington's disease (HD).
- Trial name
- Open PRIDE-HD (NCT02494778)
- Testing
- Pridopidine
- Where
- 46 sites in United States, Australia and 9 more
- Volunteers
- 248 people
- Sponsor
- Prilenia
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Alpha-1 Antitrypsin Deficiency (AATD)
A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)
A Study of CTX460 in Adults with Emphysema Associated with Severe Alpha-1 Antitrypsin Deficiency (AATD).
- Trial name
- NCT07850076
- Testing
- CTX460
- Volunteers
- 68 people
- Sponsor
- CRISPR Therapeutics
Atopic Dermatitis
Dupilumab Skin Barrier Function Study in Atopic Dermatitis
Primary Objective: Evaluate changes in skin barrier function with transepidermal water loss (TEWL) assessed after skin tape stripping (STS) in pre-defined lesional skin in participants with moderate to severe atopic dermatitis…
- Trial name
- BALISTAD (NCT04447417)
- Testing
- Dupilumab SAR231893
- Where
- 2 sites in United States, Canada
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Sanofi
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.
- Trial name
- NCT03970278
- Testing
- No intervention
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Homozygous Familial Hypercholesterolemia (HoFH)
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease.
- Trial name
- NCT04080050
- Testing
- Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 8 people
- Sponsor
- REGENXBIO Inc.
Dermatitis, Atopic
Safety and Efficacy of FURESTEM-AD Inj. for Moderate to Severe Atopic Dermatitis (AD)
This clinical trial study is two-stage, multi-center, randomized, double-blind, placebo controlled, phase 3 clinical trial to evaluate the efficacy and safety of FURESTEM-AD Inj. for moderate to severe chronic atopic dermatitis.
- Trial name
- smart(FURIN) (NCT05004324)
- Testing
- FURESTEM-AD® inj. 5.0 X 10^7 cells/1.5 mL
- Where
- South Korea
- Volunteers
- 308 people
- Sponsor
- Kang Stem Biotech Co., Ltd.