Clinical trials: Congenital Deformity
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy
A Phase 1/ Phase 2 Study of TTHX1114(NM141)
Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study
- Conditions
- Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy, Endothelial Dysfunction
- Trial name
- INTREPID (NCT04520321)
- Testing
- TTHX1114(NM141)
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Trefoil Therapeutics, Inc.
Hemophilia A
Russian Kogenate Pediatric Study
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.
- Trial name
- NCT00632814
- Testing
- rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week, rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg), rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)
- Where
- 4 sites in Russia
- Volunteers
- 32 people
- Sponsor
- Bayer
Neuroblastoma, Medulloblastoma
Molecular-Guided Therapy for Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup.
- Conditions
- Neuroblastoma, Medulloblastoma, Glioma, Ependymoma
- Trial name
- NCT02162732
- Testing
- Guided Therapy
- Where
- 19 sites in United States, Lebanon
- Volunteers
- 186 people
- Sponsor
- Giselle Sholler
Cystic Fibrosis
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Atopic Dermatitis
Combination Therapy for Atopic Dermatitis
Atopic dermatitis is a chronic relapsing disease with acute flares. The standard therapy is to treat acute flares using topical medications.
- Trial name
- NCT00119158
- Testing
- Combination of pimecrolimus and fluticasone, pimecrolimus
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- Children's Hospital of Philadelphia
Asthma, Allergic Rhinitis
Immunologic Basis of Anti-IgE Therapy (Study II: On Patients With Asthma)
The purpose of this study is to look at measures that will help scientists understand the way Omalizumab, an FDA-approved anti-allergy medication, works.
- Conditions
- Asthma, Allergic Rhinitis, Atopic Dermatitis
- Trial name
- Xolair (NCT00367016)
- Testing
- Omalizumab
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, Davis
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Familial Adenomatous Polyposis
Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this randomized, double-blind, Phase III trial is to determine if the combination of eflornithine plus sulindac is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of…
- Trial name
- NCT01483144
- Testing
- Eflornithine, Sulindac 150 MG
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 171 people
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Muscular Dystrophy, Duchenne
Spironolactone Versus Prednisolone in DMD
This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy.
- Trial name
- NCT03777319
- Testing
- Spironolactone, Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Skin Laxity
Radio Frequency Microneedling for Suprapatellar Skin
The human skin aging process is characterized by thinning dermis, atrophy of the extracellular matrix, and reduced collagen synthesis.
- Trial name
- NCT03507036
- Testing
- Profound system
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer
RATIONALE: Methadone, morphine, or oxycodone may help relieve pain caused by cancer. It is not yet known whether methadone is more effective than morphine or oxycodone in treating pain in patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00726830
- Testing
- methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- M.D. Anderson Cancer Center
Fragile x Syndrome
Ganaxolone Treatment in Children With Fragile X Syndrome
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS).
- Trial name
- NCT01725152
- Testing
- Ganaxolone
- Where
- 2 sites in United States, Belgium
- Volunteers
- 59 people
- Sponsor
- Marinus Pharmaceuticals
Pediatric Glaucoma, Elevated IOP in Pediatric Patients
Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients
The purpose of this study is to evaluate the intraocular pressure (IOP)-lowering efficacy of Travoprost 0.004% POLYQUAD (PQ) ophthalmic solution compared to Timolol ophthalmic solution (0.5% or 0.25%) in pediatric glaucoma…
- Conditions
- Pediatric Glaucoma, Elevated IOP in Pediatric Patients, Ocular Hypertension in Pediatric Patients
- Trial name
- NCT01652664
- Testing
- Travoprost 0.004% PQ ophthalmic solution, Timolol, 0.5% or 0.25% ophthalmic solution, Travoprost Vehicle
- Volunteers
- 184 people
- Sponsor
- Alcon Research
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Fludarabine, Cyclophosphamide, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing a Donor Umbilical Cord Blood Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00255684
- Testing
- graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Rochester
Congenital Heart Disease, Cardiovascular Disease
Pilot Study of B-Type Natrieutic Peptide (BNP) Levels in Patients With Congenital Heart Disease(BNP)
The object of this study is to measure the levels of B-type Natriuretic Peptide (BNP) in patients with congenital heart disease, normal individuals, and patients with acquired heart failure, and compare the results from each…
- Trial name
- NCT00308230
- Testing
- Six minute walk test, Minnesota Living with Heart Failure Questionnaire
- Volunteers
- 34 people
- Sponsor
- Emory University
Huntington's Disease
Open Label Extension Study To Investigate Long Term Safety, Tolerability And Efficacy Of Pf-02545920 In Subjects With Huntington's Disease Who Completed Study A8241021
This study is a 12 month open label extension study of PF-02545920 20 mg dosed BID following study A8241021 in subjects with HD. Primary endpoints will be to assess long-term safety and tolerability of 20 mg BID of PF-02545920.
- Trial name
- NCT02342548
- Testing
- 20 mg BID of PF-02545920
- Where
- 50 sites in United States, Canada and 3 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Patient Empowerment
Mobile Video Education to Improve Patient Clinician Communication During Prenatal Clinic Visits
The goal of this clinical trial is to test an animated video delivered by text message in patients whose pregnancies are complicated with fetal anomalies.
- Trial name
- NCT06323343
- Testing
- Video Intervention, Webpage links
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical College of Wisconsin
Sleep, Pregnancy
Halifax PrenaBelt Trial
Back and right-sided sleeping position in pregnant women has recently emerged as a potential risk factor for low birthweight (LBW) and stillbirth (SB) in the medical literature.
- Conditions
- Sleep, Pregnancy, Stillbirth, Infant, Low Birth Weight
- Trial name
- NCT02377817
- Testing
- PrenaBelt
- Where
- Canada
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Allan Kember
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