Clinical trials: Newborn
Hemophilia B
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
- Trial name
- NCT05164471
- Testing
- verbrinacogene setparvovec
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Spur Therapeutics
Papillon-Lefevre Disease
Cytokines in Papillon-Lefèvre Syndrome
Papillon-Lefèvre syndrome (PLS) is characterised by aggressively progressive periodontitis combined with palmo-plantar hyperkeratosis. It is caused by "loss of function" mutations in the cathepsin C gene.
- Trial name
- NCT01116934
- Where
- Germany
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Goethe University
Cystic Fibrosis
Self-dispersing Liquids as Aerosol Drug Carriers
Inhaled medications are often used to treat lung diseases such as cystic fibrosis. We are performing this study to determine whether inhaled medications dissolved in surfactant-based solutions will distribute more evenly…
- Trial name
- NCT00628134
- Testing
- calfactant, isotonic saline
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Pittsburgh
Rett Syndrome, RTT
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.
- Trial name
- ARCH (NCT03848832)
- Testing
- GWP42003-P
- Where
- 26 sites in United States, Italy and 2 more
- Volunteers
- 29 people
- Sponsor
- Jazz Pharmaceuticals
Degenerative Joint Disease, Congenital Deformity
Augment® Injectable Bone Graft Compared to Autologous Bone Graft as a Bone Regeneration Device in Hindfoot Fusions
STUDY OBJECTIVES: To demonstrate equivalent clinical and radiologic outcomes as "gold standard" (Autologous Bone Graft) in a representative clinical model (hindfoot fusions) STUDY HYPOTHESIS: Augment® Injectable is an equivalent…
- Conditions
- Degenerative Joint Disease, Congenital Deformity, Arthritis, Osteoarthritis
- Trial name
- NCT01305356
- Testing
- Augment® Injectable Bone Graft, Autologous bone graft
- Where
- 25 sites in United States, Canada
- Volunteers
- 299 people
- Sponsor
- BioMimetic Therapeutics
Atopic Dermatitis
A Study to Determine the Safety and Tolerability of Dupilumab (REGN668/SAR231893) in Patients Aged ≥6 to <18 Years With Atopic Dermatitis (Eczema)
The primary objective of the study is to characterize the safety and pharmacokinetics (PK) of dupilumab in pediatric patients with moderate-to-severe atopic dermatitis (AD) (for adolescents ≥12 to <18 years of age) or severe AD…
- Trial name
- NCT02407756
- Testing
- Dupilumab
- Where
- 26 sites in Canada, Czechia and 4 more
- Volunteers
- 78 people
- Sponsor
- Regeneron Pharmaceuticals
Hemorrhagic Hereditary Telangiectasia (HHT)
Efficacy and Safety of a 0.1% Tacrolimus Nasal Ointment as a Treatment for Epistaxis in Hemorrhagic Hereditary Telangiectasia (HHT)
The recognized manifestations of HHT are all due to abnormalities in vascular structure. Epistaxis due to telangiectases formation is spontaneous, very variable, recurrent in 90% of patients, and associated with severe anemia in…
- Trial name
- TACRO (NCT03152019)
- Testing
- Protopic® (Tacrolimus) 0.1% ointment
- Where
- 3 sites in France
- Volunteers
- 50 people
- Sponsor
- Hospices Civils de Lyon
Nasopharyngeal Carcinoma
Safety and Efficacy Study of PDR001 in Patients With Recurrent or Metastatic Nasopharyngeal Carcinoma
The purpose of this randomized controlled Phase II study is to assess the efficacy of PDR001 versus investigator's choice of chemotherapy in patients with advanced nasopharyngeal carcinoma (NPC).
- Trial name
- NCT02605967
- Testing
- Spartalizumab, Investigator choice of chemotherapy
- Where
- 18 sites in United States, China and 5 more
- Volunteers
- 122 people
- Sponsor
- Novartis Pharmaceuticals
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Neonatal Encephalopathy
CoolCap Followup Study-Coordination of Participating Centers
This observational study will assess long-term functional outcome of survivors from neonatal encephalopathy who were previously treated with either head cooling or with conventional care in a randomized clinical trial.
- Trial name
- NCT00581581
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- University of Rochester
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
Family Relationship, Stress
Impact of Increased Parent Presence in the Neonatal Intensive Care Unit on Parent & Infant Outcomes
The purpose of this pilot study is to compare parent and infant outcomes and unit outcomes pre and post a planned unit-wide intervention aimed at increasing parent presence in the Neonatal Intensive Care Unit (NICU).
- Conditions
- Family Relationship, Stress, Breastfeeding, Bottle Feeding
- Trial name
- NCT02901665
- Testing
- FCC intervention
- Where
- 2 sites in United States
- Volunteers
- 191 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Mucopolysaccharidosis IVA, Morquio A Syndrome
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug…
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01515956
- Testing
- BMN 110
- Where
- 5 sites in United States, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Optical Coherence Tomography (OCT) of the Retina
Low Cost OCT for Point of Care
The purpose of this study is to compare the performance of a prototype optical coherence tomography (OCT) machine with currently available high resolution OCT machines.
- Trial name
- NCT03323307
- Testing
- OCT imaging
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Duke University
Familial Hypoalphalipoproteinemia
CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases
The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when…
- Trial name
- TANGO (NCT02697136)
- Testing
- CER-001
- Where
- 19 sites in United States, Belgium and 5 more
- Volunteers
- 30 people
- Sponsor
- Cerenis Therapeutics, SA
Duodenal Adenomas, Familial Adenomatous Polyposis
CryoBalloon Ablation for Treatment of Duodenal Adenomas
This multicenter prospective non-randomized interventional study (clinical trial) that will assess the safety and efficacy of cryoballoon ablation treatment using the C2 Cryoballoon device (Pentax Medical Corporation) as an…
- Trial name
- C2D2 (NCT03847636)
- Testing
- CryoBalloon ablation
- Where
- 4 sites in United States
- Volunteers
- 33 people
- Sponsor
- Johns Hopkins University
Pectus Excavatum
Post Operative Pain Control Using Ropivacaine and the ON-Q System in the Adult Pectus Excavatum Patient
Pectus excavatum (PE) is a common chest wall deformity where the sternum is displaced posteriorly. In severe cases, surgery is performed to correct the defect.
- Trial name
- NCT01816477
- Testing
- ON-Q soaker catheter system, Epidural (ropivicaine 0.25%)
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mayo Clinic
Severe Hemophilia A
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
The primary objectives of this study are: to evaluate the safety and tolerability of rFVIIIFc administered as a prophylaxis (Arm 1), weekly (Arm 2), on-demand (Arm 3), and surgical treatment regimen; to evaluate the efficacy of…
- Trial name
- NCT01181128
- Testing
- Factor VIII (rFVIIIFc), Advate®
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 165 people
- Sponsor
- Bioverativ Therapeutics Inc.
Retinopathy of Prematurity (ROP)
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
- Trial name
- PEDAL (NCT02386839)
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 16 sites in United States, Italy and 4 more
- Volunteers
- 76 people
- Sponsor
- Shire
Sickle Cell Disease
Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain…
- Trial name
- SUSTAIN (NCT01895361)
- Testing
- SelG1
- Where
- 56 sites in United States, Brazil and 1 more
- Volunteers
- 198 people
- Sponsor
- Reprixys Pharmaceutical Corporation
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