Clinical trials: Malformation
Severe Hemophilia A
Pharmacokinetics of rFVIIIFc at Two Vial Strengths
The primary objective of the study is to characterize the pharmacokinetics (PK) of rFVIIIFc administered at vial strengths of 1000 and 3000 IU in subjects with severe hemophilia A.
- Trial name
- NCT02083965
- Testing
- rFVIIIFc
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Bioverativ Therapeutics Inc.
Infantile Colic
Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant Colic
This phase IIa study is designed as a multi-centre, multinational, randomised, double-blind, placebo controlled study in three parallel groups, with the aim to evaluate the efficacy and safety of Nepadutant given at two oral…
- Trial name
- no-cry (NCT01258153)
- Testing
- Nepadutant oral solution
- Where
- 9 sites in Germany, Poland and 2 more
- Volunteers
- 115 people
- Sponsor
- Menarini Group
Idiopathic Pulmonary Arterial Hypertension, Cardiomyopathy
Comparison of Inhaled Nitric Oxide and Oxygen in Participants Reactivity During Acute Pulmonary Vasodilator Testing
The primary purpose of this study is to compare the number of participants with reversible pulmonary hypertension (vasoreactivity) due to nitric oxide for inhalation and oxygen as compared to 100% oxygen.
- Trial name
- NCT00626028
- Testing
- Nitric Oxide for inhalation, Oxygen, Nitric Oxide plus Oxygen
- Where
- 18 sites in United States, France and 3 more
- Volunteers
- 136 people
- Sponsor
- Mallinckrodt
Sickle Cell Disease
A Rehabilitation Program in Children With Sickle Cell Disease and Cognitive Deficits: a Pilot Study
The overall goal of this project is to determine the feasibility of conducting a cognitive intervention for children with sickle cell disease.
- Trial name
- NCT00895154
- Testing
- General Tutoring, Memory Training
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Cystic Fibrosis
FDG-PET Imaging in Young Cystic Fibrosis Patients
The purpose of this research is to determine how a person's lungs will uptake [18F]fluorodeoxyglucose (FDG), as measured with positron emission tomography (PET) scanning in young cystic fibrosis (CF) patients.
- Trial name
- NCT00846053
- Testing
- FDG-PET
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
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
Atopic Dermatitis, Eczema
Daily Use of Lipikar Balm AP From Birth in Infants at High Risk of Developing Atopic Dermatitis
There is a lack of prospective scientific data on the regular use of moisturizers in patients at risk of developing atopic dermatitis.
- Conditions
- Atopic Dermatitis, Eczema, Asthma, Food Allergies
- Trial name
- NCT01577628
- Testing
- Lipikar Balm AP
- Where
- 4 sites in United States, Canada
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Cosmetique Active International
Long QT Syndrome, Cardiac Repolarization
Effect of Beta-blocker Therapy on QTc Response in Exercise and Recovery in Normal Subjects
Background. In congenital long QT syndrome type 1 (LQT1), episodes of ventricular tachycardia are usually triggered by exercise and can be prevented in most patients by beta-blocker therapy.
- Trial name
- NCT00588965
- Testing
- Propranolol LA
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- MetroHealth Medical Center
Thyroid Eye Disease
Twenty-four Hour Intraocular Pressure Patterns in Patients With Uncontrolled Thyroid Eye Disease
Patients with Thyroid Eye Disease (TED) often have enlarged extraocular muscles and higher orbital fat contents due to their disease process.
- Trial name
- NCT01798966
- Testing
- SENSIMED Triggerfish
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Sensimed AG
Arginase I Deficiency, Hyperargininemia
A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency
The purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who…
- Trial name
- NCT03378531
- Testing
- AEB1102
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 14 people
- Sponsor
- Immedica Pharma AB
Atopic Dermatitis
Long-term Extension Trial in Subjects With Atopic Dermatitis Who Participated in Previous Tralokinumab Trials - ECZTEND
The purpose of this extension trial is to evaluate the long-term safety of tralokinumab.
- Trial name
- NCT03587805
- Testing
- Tralokinumab
- Where
- 309 sites in United States, Belgium and 9 more
- Volunteers
- 1,672 people
- Sponsor
- LEO Pharma
Arterial Calcification, CD73 Deficiency
Etidronate for Arterial Calcifications Due to Deficiency in CD73 (ACDC)
Background: Arterial Calcifications due to Deficiency in CD73 (ACDC) is a rare genetic disease. People with ACDC develop calcification in the arteries of the lower extremities as well as calcium deposit in the joints of the…
- Trial name
- NCT01585402
- Testing
- Etidronate, MRI Scan
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Dasatinib, Cytarabine, and Idarubicin in Treating Patients With High-Risk Acute Myeloid Leukemia
This phase I/II trial studies the side effects and best dose of dasatinib when given together with cytarabine and idarubicin hydrochloride and to see how well they work in treating patients with acute myeloid leukemia that is…
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT01876953
- Testing
- cytarabine, idarubicin, dasatinib
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- City of Hope Medical Center
Sickle Cell Disease
SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD
Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease.
- Trial name
- SCD-Haplo (NCT02013375)
- Testing
- Haploidentical Transplant, Alemtuzumab, Total Body Irradiation
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Damiano Rondelli, MD
Hallux Valgus
Prospective Observational Study for the Evaluation of VEOFIX Varisation Staples
This clinical investigation was led as a post-market clinical follow-up study (post-market device development stage). Therefore, its objectives were as follows: To assess functional clinical score's evolution, To evaluate…
- Trial name
- NCT05523219
- Where
- France
- Volunteers
- 81 people
- Sponsor
- Societe dEtude, de Recherche et de Fabrication
Huntington Disease
An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies
This study will evaluate the long-term safety and tolerability of RO7234292 (RG6042) in participants who have completed other F.
- Trial name
- NCT03842969
- Testing
- RO7234292 (RG6042)
- Where
- 38 sites in United States, Austria and 6 more
- Volunteers
- 236 people
- Sponsor
- Hoffmann-La Roche
Kidney, Polycystic
HALT Progression of Polycystic Kidney Disease Study A
The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two…
- Trial name
- HALT PKD A (NCT00283686)
- Testing
- Lisinopril, Telmisartan, Standard Blood Pressure Control
- Where
- 7 sites in United States
- Volunteers
- 558 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Tourette Syndrome
Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression
The investigators will implant and monitor 8 research subjects with a subcortical closed-loop system for detection and suppression of tics in subjects with medically refractory debilitating Tourette Syndrome.
- Trial name
- NCT05371041
- Testing
- Medtronic Percept Neurostimulator
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Florida
Tourette Syndrome
A Study to Test if TEV-50717 is Effective in Relieving Tics Associated With Tourette Syndrome (TS)
Standard placebo-controlled, double-blind study design (TEV-50717 [low dose and high dose] vs. placebo in a 1:1:1 ratio) was chosen to determine whether study drug treatment results in a statistically significant effect on the…
- Trial name
- ARTISTS2 (NCT03571256)
- Testing
- TEV-50717
- Where
- 56 sites in United States, Argentina and 8 more
- Volunteers
- 158 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Gitelman Syndrome
A Translational Approach to Gitelman Syndrome
This study will test the response to a single dose of thiazide diuretic as a diagnostic test for Gitelman syndrome. Individuals with hypokalemic alkalosis will be enrolled.
- Trial name
- NCT00822107
- Testing
- Hydrochlorothiazide
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Oregon Health and Science University
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