Clinical trials: Newborn Diseases
Hemophilia A
Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery
The primary objective of this clinical research study is to evaluate the safety and efficacy of ReFacto in subjects with hemophilia A undergoing major surgery monitored using the chromogenic substrate assay at the local…
- Trial name
- NCT00092976
- Testing
- ReFacto
- Where
- 4 sites in United States
- Volunteers
- 15 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Respiratory Distress Syndrome In Premature Infants
Safety and Efficacy Study of the Draeger Babylog VN500 Device in HFOV Mode in VLBW Neonates
The purpose of this study is to determine the safety and effectiveness of the Babylog VN500 in high frequency oscillatory ventilation (HFOV) mode as a method for treating very low birth weight (VLBW) neonates requiring invasive…
- Trial name
- NCT02445040
- Testing
- Babylog VN500 in HFOV Mode
- Where
- 14 sites in United States
- Volunteers
- 225 people
- Sponsor
- Draeger Medical Systems, Inc.
Plagiocephaly, Brachycephaly
Cranial Remolding Orthosis Registry
A registry of infants fitted with a cranial remolding orthosis.
- Conditions
- Plagiocephaly, Brachycephaly, Asymmetric Head
- Trial name
- NCT06762691
- Testing
- MyCRO Band, Traditional CRO
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Otto Bock HealthCare LP
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.
Huntington Disease
A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.
This is a dose escalation and expansion clinical study to evaluate the safety, tolerability, PK profile and preliminary efficacy of ER2001 Injection vs. placebo in subjects with definitive diagnosis of early manifest HD.
- Trial name
- NCT07339514
- Testing
- ER2001 intravenous injection
- Where
- 3 sites in China
- Volunteers
- 27 people
- Sponsor
- ExoRNA Bioscience
Urea Cycle Disorders, Crigler Najjar Syndrome
Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
- Trial name
- NCT02051049
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Cellaion SA
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-3 (NCT05126758)
- Testing
- Deramiocel (CAP-1002)
- Where
- 20 sites in United States
- Volunteers
- 106 people
- Sponsor
- Capricor Inc.
Atopic Dermatitis
A Study of CM310 in Children Patients With Moderate-to-severe Atopic Dermatis
This is a multi-center, single arm, open-label study to evaluate safety and primary efficacy in children patients with moderate-to severe atopic dermatis.
- Trial name
- NCT05579925
- Testing
- CM310
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Keymed Biosciences Co.Ltd
Atopic Dermatitis
A Multi-Centre Clinical Trial to Evaluate a Left-Right Design in Adults With Mild to Moderate Atopic Dermatitis
The purpose of this study is to validate a left-right design with respect to detecting a difference in efficacy after 3 weeks of treatment between an active treatment and a vehicle in adults with mild to moderate atopic dermatitis
- Trial name
- NCT02103725
- Testing
- pimecrolimus 10 mg/g cream, Vehicle cream
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LEO Pharma
Dermatitis, Atopic
Effect of Moisturising Creams on Skin Moisture in Atopic Dermatitis
The aim of this study is evaluate the effect of three moisturising creams on Stratum corneum (SC) hydratation and barrier function.
- Trial name
- NCT00846235
- Testing
- Aqualan L, Aqualan Plus, Aqualan L + glycerol ad 20%
- Where
- Finland
- Volunteers
- 24 people
- Sponsor
- Orion Corporation, Orion Pharma
Retinopathy of Prematurity
Regulatory PMS Study for Lucentis® in Patients With Retinopathy of Prematurity
This study was an open-labeled, multicenter, single arm, observational post-marketing surveillance study under routine clinical practice with no mandated treatments, visits or assessments.
- Trial name
- NCT05304949
- Testing
- Lucentis
- Where
- 6 sites in South Korea
- Volunteers
- 69 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Hyperinsulinism
A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism
The purpose of this study is to evaluate the safety and clinical pharmacology of a single dose pharmacokinetics and pharmacodynamics of XOMA 358 in subjects with hypoglycemia associated with congenital hyperinsulinism.
- Trial name
- NCT02604485
- Testing
- Cohort 1, Cohort 2, Cohort 3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- XOMA (US) LLC
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Primary Ciliary Dyskinesia
Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia
To evaluate the safety and efficacy of treatment with VX-371 with and without ivacaftor, and the effect of VX-371 with and without ivacaftor on quality of life (QOL) in subjects with primary ciliary dyskinesia (PCD).
- Trial name
- CLEAN-PCD (NCT02871778)
- Testing
- VX-371, Hypertonic Saline, VX-371 + HS
- Where
- 34 sites in United States, Canada and 6 more
- Volunteers
- 123 people
- Sponsor
- Parion Sciences
Atopic Dermatitis
A Study of Barzolvolimab in Patients With Atopic Dermatitis
The purpose of this study is to assess the efficacy and safety of barzolvolimab in adults with Atopic Dermatitis
- Trial name
- NCT06727552
- Testing
- Barzolvolimab
- Where
- 33 sites in United States
- Volunteers
- 131 people
- Sponsor
- Celldex Therapeutics
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Hemophilia B
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics…
- Trial name
- NCT03307980
- Testing
- PF-06838435 (formerly SPK-9001)
- Where
- 25 sites in United States, Australia and 2 more
- Volunteers
- 21 people
- Sponsor
- Pfizer
Atopic Dermatitis
Safety, Tolerability, and Pharmacokinetic Study of QLM3003 in Healthy Adult Subjects
The purpose of the study is to evaluate the safety and tolerability of single ascending (SAD) doses of QLM3003 compared to placebo. Also, pharmacokinetics (PK) of qlm3003 will be evaluated.
- Trial name
- NCT05932888
- Testing
- QLM3003
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Urea Cycle Disorder
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
This is a randomized, controlled, open-label parallel arm study to assess the safety, tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to Sodium phenylbutyrate (NaPBA) in urea cycle disorder subjects…
- Trial name
- NCT03335488
- Testing
- RAVICTI, NaPBA
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 16 people
- Sponsor
- Amgen
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia
Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C)
This study is a Phase II, placebo-controlled, double-blind, randomized trial in 480 participants with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents (for example, diabetes and familial…
- Conditions
- Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes
- Trial name
- ORION-1 (NCT02597127)
- Testing
- ALN-PCSSC, Normal Saline
- Where
- 54 sites in United States, Canada and 3 more
- Volunteers
- 501 people
- Sponsor
- The Medicines Company
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