Clinical trials: Fetal Developmental Abnormality
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen.
- Trial name
- NCT01263379
- Testing
- LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
Short Stature
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
With this study we want to investigate the pharmacokinetic (PK) effect of a single injection of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy controls.
- Trial name
- NCT02636270
- Testing
- Increlex
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cystic Fibrosis
Absorptive Clearance After Inhaled Osmotics in Cystic Fibrosis
Blockage of the breathing tubes of the lungs by thick, sticky mucus is a major cause of lung problems for people with cystic fibrosis (CF).
- Trial name
- NCT01223183
- Testing
- hypertonic saline (7%), isotonic saline
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Pittsburgh
Epidermolysis Bullosa, Pruritus
Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients
Our goal is to determine whether daily oral administration of VPD-737 (5 mg) is effective and safe in treating moderate to severe pruritus in patients with Epidermolysis Bullosa (EB).
- Trial name
- NCT02654483
- Testing
- VPD-737
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Jean Yuh Tang
Stroke, Transient Ischemic Attack
GORE® Septal Occluder Device for Patent Foramen Ovale (PFO) Closure in Stroke Patients
The primary objective is to determine if patent foramen ovale (PFO) closure with the GORE® HELEX® Septal Occluder or GORE® CARDIOFORM Septal Occluder plus antiplatelet medical management is safe and effective and reduces the risk…
- Trial name
- REDUCE (NCT00738894)
- Testing
- Septal Occluder Device, Antiplatelet Medical Therapy
- Where
- 2 sites in United States, Denmark
- Volunteers
- 664 people
- Sponsor
- W.L.Gore & Associates
Ototoxicity
SPI-1005 for Prevention and Treatment of Tobramycin Induced Ototoxicity
The primary objective of this study is to determine the safety and efficacy of SPI-1005 treatment in CF patients with active pulmonary exacerbation that are receiving an IV course of tobramycin, determined by comparing hearing…
- Trial name
- NCT02819856
- Testing
- SPI-1005 Ebselen 200mg Capsule x1, SPI-1005 Ebselen 200mg Capsule x2, SPI-1005 Ebselen 200mg Capsule x3
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sound Pharmaceuticals, Incorporated
Port Wine Stains
Combined Photodynamic and Pulsed Dye Laser Treatment of Port Wine Stains
This research study was designed to determine the effect on port wine stains (PWS) of liposomal benzoporphyrin derivative monoacid (BPD-MA) termed verteporfin by intravenous (IV) infusion for photodynamic therapy (PDT) or…
- Trial name
- PDT/PDL (NCT00556946)
- Testing
- Combined Photodynamic & Pulsed Dye Laser Treatment
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of California, Irvine
Autosomal Dominant Polycystic Kidney, ADPKD
Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD
The goal of the study was to compare and evaluate safety and efficacy of tesevatinib 50 milligrams (mg) versus placebo in participants with autosomal dominant polycystic kidney disease (ADPKD).
- Trial name
- NCT03203642
- Testing
- Tesevatinib
- Where
- 19 sites in United States
- Volunteers
- 80 people
- Sponsor
- Kadmon, a Sanofi Company
Metabolism, Inborn Errors, Hearing Loss
North Carolina Newborn Exome Sequencing for Universal Screening
The NC NEXUS research study is exploring the utility of next generation sequencing in newborn screening and parental decision making.
- Conditions
- Metabolism, Inborn Errors, Hearing Loss, Hereditary Disease
- Trial name
- NC_NEXUS (NCT02826694)
- Testing
- Well infant, whole exome sequencing, Diagnosed, whole exome sequencing
- Where
- United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe steatoblepharon (excess eyelid fat).
- Trial name
- NCT02230761
- Testing
- XOPH5 Ointment
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Topokine Therapeutics, Inc.
Congenital Hyperinsulinism (CHI), Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)
Phase II Safety and Efficacy Study of 18FDOPA PET-CT in Children With Hyperinsulinemic Hypoglycemia
Children with congenital hyperinsulinism (CHI) have low blood sugar, and some of these children may require surgery to remove part or all of their pancreas.
- Trial name
- 18FDOPA (NCT01468454)
- Testing
- 18 F-DOPA
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
- Trial name
- NCT02875366
- Testing
- LUM/IVA
- Where
- 12 sites in Australia, United Kingdom
- Volunteers
- 70 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young…
- Trial name
- DMD (NCT03340675)
- Testing
- Ifetroban
- Where
- 10 sites in United States
- Volunteers
- 46 people
- Sponsor
- Cumberland Pharmaceuticals
Neuroendocrine Tumors, Carcinoid Tumors
A Phase II Study of Pertuzumab and Erlotinib for Metastatic or Unresectable Neuroendocrine Tumors
To determine objective response rates (RR) by RECIST guideline version 1.1 for all patients treated with this strategy consisting of initial therapy with pertuzumab as a single agent and then addition of erlotinib for those who…
- Conditions
- Neuroendocrine Tumors, Carcinoid Tumors, Adrenal Gland Tumors, Neuroblastoma
- Trial name
- NCT00947167
- Testing
- pertuzumab, erlotinib
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Pamela L. Kunz
Respiratory Syncytial Virus Infection, Premature Birth
Palivizumab for Prevention of Severe Respiratory Syncytial Virus Infection in Russian Children
100 Russian children of 2 years of age and less in high-risk populations (preterm, and/or with heart and lung problems) will receive palivizumab (Synagis) 15 mg/kg intramuscularly as prophylaxis to severe respiratory syncytial…
- Conditions
- Respiratory Syncytial Virus Infection, Premature Birth, Bronchopulmonary Dysplasia, Congenital Heart Disease
- Trial name
- NCT01006629
- Testing
- palivizumab
- Where
- 19 sites in Russia
- Volunteers
- 100 people
- Sponsor
- Abbott
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Clinical Trial
The primary objective of this investigation is to assess the effectiveness of transcutaneous electrical nerve stimulation applied using VECTTOR to reduce the symptoms of Duchenne Muscular Dystrophy and reduce the impact of DMD…
- Trial name
- DMD (NCT01874275)
- Testing
- VECTTOR
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alan Neuromedical Technologies, LLC
Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis
Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome
This study will examine whether five drugs (pravastatin, Losartan, Zileuton, N-acetylcysteine and erythromycin) used together can slow the course of pulmonary fibrosis (scarring of the lung tissue) in patients with…
- Conditions
- Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency
- Trial name
- NCT00467831
- Testing
- Losartan, Zileuton, N-Acetylcysteine
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Osteoarthritis, Rheumatoid Arthritis
Persona TM Tibia Clinical Outcomes Study
The primary objective of this study is to evaluate clinical performance for the commercially available Persona TM Tibia used in primary cementless tibia total knee arthroplasty.
- Conditions
- Osteoarthritis, Rheumatoid Arthritis, Traumatic Arthritis, Polyarthritis
- Trial name
- NCT03589300
- Testing
- Persona TM Tibia
- Where
- 7 sites in United States
- Volunteers
- 148 people
- Sponsor
- Zimmer Biomet
Primary Immunodeficiency
CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL
Summary for SCGAM-03: Clinical phase III study to monitor the safety, tolerability and efficacy of subcutaneous human immunoglobulin (Octanorm) in patients with primary immunodeficiency diseases who have completed the SCGAM-01…
- Trial name
- NCT03907241
- Testing
- Octanorm 16.5%
- Where
- 7 sites in United States, Canada
- Volunteers
- 27 people
- Sponsor
- Octapharma
Mucopolysaccharidosis I
A Study of JR-171 in Patients With Mucopolysaccharidosis I
Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).
- Trial name
- NCT04227600
- Testing
- JR-171 (lepunafusp alfa)
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 18 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
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