Clinical trials: Birth Defects
Meningomyelocele, Spinal Dysraphism
Management of Myelomeningocele Study (MOMS)
Spina bifida (myelomeningocele) is a complex birth defect in which a portion of the spinal cord is not fully developed. The overlying bones and skin are incompletely formed and the underdeveloped area of the spinal cord is…
- Trial name
- MOMS (NCT00060606)
- Testing
- Prenatal Myelomeningocele Repair Surgery, Postnatal Myelomeningocele Repair Surgery
- Where
- 3 sites in United States
- Volunteers
- 183 people, healthy volunteers welcome
- Sponsor
- The George Washington University Biostatistics Center
Dermatitis, Atopic
Safety, Tolerability, and PK of AN2728 in Adolescents With Atopic Dermatitis
The purpose of this study is to investigate the safety, tolerability, and systemic exposure of AN2728 Topical Ointment, 2%, in subjects with atopic dermatitis.
- Trial name
- NCT01652885
- Testing
- AN2728 Topical Ointment, 2%
- Where
- 6 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Congenital Heart Disease
Angiotensin Converting Enzyme Inhibitors During Cardiopulmonary Bypass in Infants and Children
The purpose of this study is to determine whether ACE inhibitors alter the fibrinolytic, inflammatory, and hemodynamic response to cardiopulmonary bypass in infants and children with congenital heart disease.
- Trial name
- NCT00848250
- Testing
- Angiotensin Converting Enzyme Inhibitor, No ACE Inhibitor
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Vanderbilt University Medical Center
Leukemia, Lymphoma
Donor Peripheral Stem Cell Transplant, Fludarabine, and Busulfan in Treating Patients With Hematologic Cancers
Giving chemotherapy drugs, such as fludarabine and busulfan, before a donor peripheral stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00619645
- Testing
- busulfan, cyclosporine, fludarabine phosphate
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of California, Davis
Cystic Fibrosis
A Study of Tobramycin Inhalation Powder From a Modified Manufacturing Process Versus Placebo
This study is designed to show how well tobramycin inhalation powder works and how safe it is when produced by a modified manufacturing process
- Trial name
- EDIT (NCT00918957)
- Testing
- Tobramycin Inhalation Powder
- Where
- 24 sites in Bulgaria, Egypt and 7 more
- Volunteers
- 62 people
- Sponsor
- Novartis Pharmaceuticals
Polycystic Liver Disease
A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD…
- Trial name
- POSITANO (NCT05281328)
- Testing
- CAM2029
- Where
- 11 sites in United States, Belgium and 2 more
- Volunteers
- 71 people
- Sponsor
- Camurus AB
Cystic Fibrosis
A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with…
- Trial name
- NCT03224351
- Testing
- VX-659, TEZ/IVA, IVA
- Where
- 47 sites in United States, Ireland and 2 more
- Volunteers
- 124 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Angelman Syndrome
A Trial of Levodopa in Angelman Syndrome
This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS).
- Trial name
- NCT01281475
- Testing
- Levodopa
- Where
- 7 sites in United States
- Volunteers
- 67 people
- Sponsor
- Wen-Hann Tan
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.
Neurogenic Detrusor Overactivity
A Two-week Open-label Pharmacodynamic and Pharmacokinetic Study of Multiple Doses of a Darifenacin Liquid Oral Suspension in Children (2 - 15 Years) With Neurogenic Detrusor Overactivity
This study will evaluate the pharmacodynamics and pharmacokinetics of darifenacin liquid oral suspension in children, ages 2-15 years, with neurogenic detrusor overactivity.
- Trial name
- NCT00712322
- Testing
- Darifenacin
- Where
- 12 sites in United States, Colombia
- Volunteers
- 35 people
- Sponsor
- Warner Chilcott
Atopic Dermatitis
Pharmacokinetics and Safety of MM36 Topical Ointment in Pediatric Subjects With Atopic Dermatitis
The purpose of this study is to assess the pharmacokinetic parameters and safety of topical MM36 (OPA-15406) ointment in pediatric subjects with atopic dermatitis under maximal use conditions.
- Trial name
- NCT02945657
- Testing
- MM36 topical ointment, 1%
- Where
- 12 sites in United States, Honduras and 1 more
- Volunteers
- 32 people
- Sponsor
- Medimetriks Pharmaceuticals, Inc
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
- Conditions
- Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies, Genetic Disorders
- Trial name
- NCT01401257
- Testing
- PXT3003 Low dose, PXT3003 Intermediate Dose, PXT3003 High Dose
- Where
- 6 sites in France
- Volunteers
- 80 people
- Sponsor
- Pharnext S.C.A.
Hereditary Angioedema
A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II
This study is a randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adult subjects.
- Trial name
- NCT04208412
- Testing
- KVD900
- Where
- 25 sites in United States, Austria and 8 more
- Volunteers
- 84 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Cystic Fibrosis
Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation
Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic…
- Trial name
- NCT02564354
- Testing
- QR-010
- Where
- 5 sites in United States, Belgium and 1 more
- Volunteers
- 18 people
- Sponsor
- ProQR Therapeutics
Hemophilia B
A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
This study will examine the pharmacokinetics, safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children who have previously received factor replacement therapy for hemophilia B.
- Trial name
- NCT01662531
- Testing
- rIX-FP
- Where
- 17 sites in Australia, Austria and 8 more
- Volunteers
- 27 people
- Sponsor
- CSL Behring
Atopic Dermatitis
A Study of (LY3650150) Lebrikizumab to Assess the Safety and Efficacy of Adult and Adolescent Participants With Moderate-to-Severe Atopic Dermatitis and Skin of Color
The main purpose of this study is to determine the safety and efficacy lebrikizumab in adolescent and adult participants with moderate-to-severe atopic dermatitis (AD) and skin of color.
- Trial name
- ADmirable (NCT05372419)
- Testing
- Lebrikizumab
- Where
- 36 sites in United States
- Volunteers
- 90 people
- Sponsor
- Eli Lilly and Company
Atopic Dermatitis
Long-term Safety and Efficacy Study of Lebrikizumab (LY3650150) in Participants With Moderate-to-Severe Atopic Dermatitis (ADjoin)
This is designed to assess the long-term safety and efficacy of lebrikizumab for moderate-to-severe atopic dermatitis. It will last up to 33 months.
- Trial name
- ADjoin (NCT04392154)
- Testing
- Lebrikizumab
- Where
- 321 sites in United States, Australia and 14 more
- Volunteers
- 1,153 people
- Sponsor
- Eli Lilly and Company
Skin Laxity
Measuring Renuvion Soft Tissue Contraction Using Ultrasound
This is a prospective, single-center, non-randomized study of up to 5 subjects who are receiving treatment with the Renuvion APR system following power-assisted liposuction in the abdomen per investigator standard of care.
- Trial name
- NCT05968495
- Testing
- Renuvion APR Handpiece
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Apyx Medical
Neonatal Brachial Plexus Palsy
NMES Efficacy on Patients With NBPP
Neonatal Brachial Plexus Palsy (NBPP), characterized by weakness and sensory loss in the affected arm, results from damage in the prenatal period to the nerves extending from the spine to the arm.
- Trial name
- NCT01999465
- Testing
- Standard NMES device
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Michigan