Clinical trials: Malformation
Anemia, Sickle Cell
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the…
- Trial name
- NCT01054768
- Testing
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Cystic Fibrosis
Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
- Trial name
- APPLAUD (NCT03265288)
- Testing
- LAU-7b
- Where
- 40 sites in United States, Australia and 1 more
- Volunteers
- 166 people
- Sponsor
- Laurent Pharmaceuticals Inc.
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Lymphatic Malformations, Lymphatic Diseases
Sildenafil for the Treatment of Lymphatic Malformations
A Phase 2 study to evaluate safety and efficacy of sildenafil taken orally to improve or resolve lymphatic malformations in children.
- Trial name
- NCT02335242
- Testing
- Sildenafil 20 mg tablets
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Stanford University
Atopic Dermatitis
A Multicenter, Randomized, Placebo-Controlled, Double-Blind Study of SUN13834 in Adult Subjects With Atopic Dermatitis
The purpose of the study is to explore the efficacy and safety of SUN13834 vs placebo in adult participants with atopic dermatitis.
- Trial name
- NCT00717769
- Testing
- SUN13834
- Where
- 28 sites in United States
- Volunteers
- 270 people
- Sponsor
- Daiichi Sankyo
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
- Trial name
- NCT00283959
- Testing
- migalastat HCl
- Where
- 2 sites in Australia, Brazil
- Volunteers
- 4 people
- Sponsor
- Amicus Therapeutics
Infantile Neronal Ceroid Lipofuscinosis
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children.
- Trial name
- NCT00028262
- Testing
- Cystagon
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Patent Ductus Arteriosus
Preliminary Percutaneous Intervention Versus Observational Trial of Arterial Ductus in Low-weight Infants
This is a pilot study to collect preliminary data for a larger, multicenter clinical trial proposal. The study will examine two strategies commonly used to treat preterm infants diagnosed with a patent ductus arteriosus (PDA).
- Trial name
- PIVOTAL (NCT03982342)
- Testing
- Cather closure of patent ductus arteriosus (PDA), Conservative management of patent ductus arteriosus (PDA)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Nationwide Children's Hospital
Congenital Heart Defects
Use of Color Doppler in Routine Examination of Fetal Heart in Second Trimester
To evaluate the percentage of severe heart defect diagnosed with the use of color Doppler at a routine fetal scan in the second trimester of pregnancy
- Trial name
- NCT01201486
- Where
- Norway
- Volunteers
- 6,781 people
- Sponsor
- Helse Stavanger HF
Hernia, Hernia, Diaphragmatic
'TOTAL' (Tracheal Occlusion To Accelerate Lung Growth) Trial
This trial will test whether temporary fetoscopic endoluminal tracheal occlusion (FETO) rather than expectant management during pregnancy, followed by standardized postnatal management, increases survival at discharge and…
- Conditions
- Hernia, Hernia, Diaphragmatic, Hernia, DIaphragmatic, Congenital, Pathological Conditions, Anatomical
- Trial name
- TOTAL (NCT02875860)
- Testing
- GoldBal2 detachable balloon, Baltaccidbpe100 Delivery Catheter, Standardized postnatal care
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Michael A Belfort
Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
Bortezomib and Combination Chemotherapy in Treating Younger Patients With Recurrent, Refractory, or Secondary Acute Myeloid Leukemia
This phase II trial is studying the side effects and best dose of bortezomib and to see how well it works when given together with combination chemotherapy in treating younger patients with recurrent, refractory, or secondary…
- Conditions
- Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1)
- Trial name
- NCT00666588
- Testing
- idarubicin, cytarabine, bortezomib
- Where
- 82 sites in United States, Australia and 1 more
- Volunteers
- 52 people
- Sponsor
- National Cancer Institute (NCI)
Atopic Dermatitis
Efficacy and Safety of IDP-124 Lotion for the Treatment of Moderate to Severe Atopic Dermatitis in Pediatric and Adult Subjects
Efficacy and Safety of IDP-124 Lotion for the Treatment of Moderate to Severe Atopic Dermatitis in Pediatric and Adult Subjects
- Trial name
- NCT03058783
- Testing
- IDP-124 Lotion, IDP-124 Vehicle Lotion
- Where
- United States
- Volunteers
- 328 people, healthy volunteers welcome
- Sponsor
- Bausch Health Americas, Inc.
Muscular Dystrophy, Duchenne
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD.
- Trial name
- PolarisDMD (NCT03703882)
- Testing
- Edasalonexent
- Where
- 40 sites in United States, Australia and 6 more
- Volunteers
- 131 people
- Sponsor
- Catabasis Pharmaceuticals
Atopic Dermatitis
Phase 2a Study of Efficacy and Safety of OpSCF in Moderate to Severe AD (Atopic Dermatitis)
The purpose of this study is to determine the efficacy and safety of a monoclonal antibody, OpSCF, in the treatment of adults with moderate to severe Atopic Dermatitis (Eczema). OpSCF will be compared to a placebo.
- Trial name
- NCT06101823
- Testing
- OpSCF
- Where
- 22 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Insmed Incorporated
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.
- Trial name
- NCT01855360
- Testing
- Tauroursodeoxycholic Acid and Doxycycline
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
Craniosynostoses
Neurodevelopmental Outcomes in Craniosynostosis Repair
Context: Craniosynostosis is a common craniofacial abnormality which can be associated with various clinical syndromes. Though it has been established that children with craniosynostosis score lower on certain developmental…
- Trial name
- NCT04072783
- Testing
- MRI,DTI, functional MRI
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Plerixafor and Filgrastim For Mobilization of Donor Peripheral Blood Stem Cells Before A Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
RATIONALE: Giving chemotherapy and total-body irradiation (TBI) before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01076270
- Testing
- plerixafor, filgrastim, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Fred Hutchinson Cancer Center
Hyperkalemic Periodic Paralysis, Hypokalemic Periodic Paralysis
Hyper- and Hypokalemic Periodic Paralysis Study
The purpose of this study is to compare Dichlorphenamide with placebo (an inactive substance) for prevention of episodes and for improvement of strength in hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis.
- Trial name
- HYP-HOP (NCT00494507)
- Testing
- Dichlorphenamide (double-blind), Dichlorphenamide (open-label)
- Where
- 12 sites in United States, Italy and 1 more
- Volunteers
- 71 people
- Sponsor
- University of Rochester
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Vorinostat, Azacitidine, and Gemtuzumab Ozogamicin for Older Patients With Relapsed or Refractory AML
The purpose of this study is to test the safety of vorinostat (Zolinza) and azacitidine (Vidaza) when combined with gemtuzumab ozogamicin (GO) at different dose levels.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT00895934
- Testing
- vorinostat, gemtuzumab ozogamicin, azacitidine
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- National Cancer Institute (NCI)
Skin Laxity, Wrinkles
A Retrospective Study to Evaluate the Effectiveness of the Ulthera System
Up to 120 subjects within the study site's practice who have already had an Ultherapy™ treatment to the face and neck will be enrolled to monitor treatment effectiveness.
- Trial name
- NCT01519934
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.