Clinical trials: Fetal Developmental Abnormality
Hypoplastic Left Heart Syndrome, Growth Failure
Comparison of Feeding Strategies for Hypoplastic Left Heart Syndrome Infants
The purpose of the study is to determine if a continuous feeding regimen as compared to an intermittent bolus feeding regimen leads to improved weight gain in infants with hypoplastic left heart syndrome (HLHS) after stage 1…
- Trial name
- NCT02657629
- Testing
- Continuous Feeding Regimen, Intermittent Bolus Feeding Regimen
- Volunteers
- 28 people
- Sponsor
- Columbia University
Skin Lesion, Skin Abnormalities
Comparative Study Between CellFX and Electrodessication in Sebaceous Hyperplasia (SH) Lesions
This study is designed as a multi-center, single-blinded, prospective, randomized, study to compare the safety and effectiveness of the CellFX System to the comparator group, Electrodessication for the treatment of SH lesions in…
- Conditions
- Skin Lesion, Skin Abnormalities, Sebaceous Hyperplasia
- Trial name
- NCT04539886
- Testing
- CellFX System, Intralesional Electrodesiccation
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- Pulse Biosciences, Inc.
Muscular Dystrophy (DMD)
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
- Trial name
- NCT02515669
- Testing
- RO7239361
- Where
- 12 sites in United States, Canada
- Volunteers
- 43 people
- Sponsor
- Hoffmann-La Roche
Cystic Fibrosis
Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF
The primary objective of this study is to evaluate the safety and efficacy of a CAT regimen with aztreonam for inhalation solution (AZLI) and tobramycin inhalation solution (TIS) in adult and pediatric subjects with cystic…
- Trial name
- AZLI CAT (NCT01641822)
- Testing
- AZLI, Tobramycin inhalation solution
- Where
- 71 sites in United States
- Volunteers
- 107 people
- Sponsor
- Gilead Sciences
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of Ruxolitinib Cream in Children With Atopic Dermatitis (TRuE-AD3)
The purpose of the study is to assess the efficacy and safety of ruxolitinib cream in children with Atopic Dermatitis. This is a randomized, double-blind, Vehicle Controlled study.
- Trial name
- NCT04921969
- Testing
- Ruxolitinib, Vehicle Cream
- Where
- 67 sites in United States, Canada
- Volunteers
- 330 people
- Sponsor
- Incyte Corporation
Osteogenesis Imperfecta
Growth Hormone Therapy in Osteogenesis Imperfecta
Growth deficiency is a key feature of severe Osteogenesis Imperfecta (OI) and a frequent feature of mild to moderate forms of the disease. The reason that children with OI are short is not fully understood.
- Trial name
- NCT00001305
- Testing
- Humatrope
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Phenylketonuria
Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)
SYNB1934-CP-003 was designed as a 3-part, adaptive study consisting of a dose-escalating, open-label period (DEP; Part 1) of up to 15 weeks, followed by a 4-week, double-blind, placebo-controlled, randomized withdrawal period…
- Trial name
- SYNPHENY-3 (NCT05764239)
- Testing
- SYNB1934v1
- Where
- 21 sites in United States, Canada and 2 more
- Volunteers
- 35 people
- Sponsor
- Synlogic
Fragile X Syndrome
Neurophysiological and Acute Pharmacological Studies in FXS Patients
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.
- Trial name
- NCT02998151
- Testing
- Acamprosate, Lovastatin, Minocycline
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cystic Fibrosis
Study to Assess the Safety and PK of Oral and IV Xenleta in Adults With Cystic Fibrosis
This study is intended to assess the pharmacokinetic (PK) and safety of a single dose of IV and oral formulations of lefamulin in adults with cystic fibrosis (CF).
- Trial name
- NCT05225805
- Testing
- Lefamulin
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- Nabriva Therapeutics AG
Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight
Transfusion of Prematures Trial
The objective of the TOP trial is to determine whether higher hemoglobin thresholds for transfusing ELBW infants resulting in higher hemoglobin levels lead to improvement in the primary outcome of survival and rates of…
- Conditions
- Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight, Infant, Small for Gestational Age, Bronchopulmonary Dysplasia (BPD)
- Trial name
- TOP (NCT01702805)
- Testing
- Liberal Cell Transfusion, Restricted red cell transfusion
- Where
- 20 sites in United States
- Volunteers
- 1,824 people
- Sponsor
- NICHD Neonatal Research Network
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00304512
- Testing
- migalastat HCl
- Where
- 6 sites in United States, Australia and 4 more
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Polycystic Ovary Syndrome, Hyperandrogenism
Effect of Metformin on Sensitivity of the GnRH Pulse Generator to Suppression by Estradiol and Progesterone
Many, but not all, girls with high levels of the male hormone testosterone go on to develop polycystic ovary syndrome (PCOS) as adults.
- Trial name
- NCT01427595
- Testing
- Metformin, Progesterone, estrace
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Virginia
Cleft Lip and Palate
Use of Mesenchymal Stem Cells for Alveolar Bone Tissue Engineering for Cleft Lip and Palate Patients
The aim of this study is to perform the bone tissue engineering to reconstruct the alveolar bone defect in cleft lip and palate patients using mesenchymal stem cells from deciduous dental pulp associated with a collagen and…
- Trial name
- NCT01932164
- Testing
- maxillary alveolar graft by tissue engineering, Bone tissue engineering using mesenchymal stem cells
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- Hospital Sirio-Libanes
Cardiac Disease
Beta Blockers and Angiotensin Receptor Blockers in Bicuspid Aortic Valve Disease Aortopathy (BAV Study)
The purpose of this study is to determine whether long-term treatment with a beta-blocker (BB) such as atenolol and/or an angiotensin receptor blocker (ARB) such as telmisartan, given to adult patients with bicuspid aortic valve…
- Trial name
- BAV (NCT01202721)
- Testing
- Atenolol, Telmisartan
- Where
- 13 sites in Canada
- Volunteers
- 85 people
- Sponsor
- Hamilton Health Sciences Corporation
Severe Hemophilia A
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A
- Trial name
- NCT03376516
- Testing
- Wilate
- Where
- 3 sites in Russia, Ukraine
- Volunteers
- 11 people
- Sponsor
- Octapharma
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05693 (P05713)
The objective of follow-up study P05713 is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of monofollicular growth in women who underwent ovulation induction (OI) in base study P05693…
- Trial name
- Care (NCT00702338)
- Testing
- corifollitropin alfa, recombinant Follicle Stimulating Hormone (recFSH), human Chorion Gonadotropin (hCG)
- Volunteers
- 1 people
- Sponsor
- Organon and Co
Von Willebrand Disease (VWD)
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants…
- Trial name
- NCT03879135
- Testing
- rVWF, rFVIII
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis
Hypertonic Saline and Mucociliary Clearance in Children
Previously, the investigators and others have shown that mucociliary clearance (MCC) is defective in patients with cystic fibrosis (CF) and it is now thought that alterations in airway mucus rheology figure prominently in the…
- Trial name
- NCT01293084
- Testing
- 0.12% saline, 7% saline
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins University
Congenital Ichthyosis
A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older…
- Trial name
- NCT04154293
- Testing
- Isotretinoin, Vehicle
- Where
- 11 sites in United States, Australia
- Volunteers
- 34 people
- Sponsor
- LEO Pharma
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation
This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744)…
- Trial name
- NCT02565914
- Testing
- TEZ/IVA, IVA
- Where
- 154 sites in United States, Australia and 14 more
- Volunteers
- 1,131 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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