Clinical trials: Newborn
Cystic Fibrosis
Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
The purpose of this study was to evaluate the safety and efficacy of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
- Trial name
- AIR-CF2 (NCT00104520)
- Testing
- AZLI 75 mg two times a day (BID)/three times a day (TID)
- Where
- 56 sites in United States
- Volunteers
- 211 people
- Sponsor
- Gilead Sciences
Molar Incisor Hypomineralization, Dental Caries
Silver Diamine Fluoride and Papain-Based Gel for Management of MIH-affected Molar in Paediatric Patients
Molar incisor hypomineralization (MIH) is a developmental defect of enamel affecting the first permanent molar and frequently the incisors with a prevalence of 16-16.9% in Malaysia.
- Conditions
- Molar Incisor Hypomineralization, Dental Caries, Hypomineralization of Enamel
- Trial name
- NCT04710927
- Testing
- Silver Diamine Fluoride, Papain-Based Gel, High Viscosity Glass Ionomer Cement (HVGIC)
- Where
- Malaysia
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- University of Malaya
Hunter Syndrome
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in…
- Trial name
- NCT00937794
- Testing
- Neurobehavioral testing, Visual and auditory assessments
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Shire
Atopic Dermatitis
To Assess the Safety and Activity of GBR 830, Compared to Placebo, in Adults With Moderate-to-severe Atopic Dermatitis
The purpose of this study is to determine the effect of GBR 830 on biomarkers in atopic dermatitis to enable further studies in this indication.
- Trial name
- NCT02683928
- Testing
- GBR 830
- Where
- 17 sites in United States, Canada
- Volunteers
- 64 people
- Sponsor
- Ichnos Sciences SA
Pompe Disease, Glycogen Storage Disease Type II
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset…
- Trial name
- NCT00701129
- Testing
- Alglucosidase Alfa, Methotrexate, Rituximab
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Hemophilia A
A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480)
- Trial name
- NCT03061201
- Testing
- SB-525 (PF-07055480)
- Where
- 22 sites in United States
- Volunteers
- 13 people
- Sponsor
- Pfizer
Respiratory Distress Syndrome, Newborn
Aerosolized Surfactant in Neonatal RDS
Respiratory distress syndrome (RDS), caused by surfactant deficiency, is the leading cause of mortality and morbidity in preterm infants.
- Trial name
- AS-02 (NCT02294630)
- Testing
- Surfactant
- Where
- United States
- Volunteers
- 159 people
- Sponsor
- Sood, Beena G., MD, MS
Cystic Fibrosis, Advanced Lung Disease
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
- Trial name
- NCT02390219
- Testing
- Lumacaftor, Ivacaftor
- Where
- 6 sites in United States
- Volunteers
- 46 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Neonatal Abstinence Syndrome
Comparison Between Methadone and Morphine for Neonatal Opiate Withdrawal
Hypothesis is that the effectiveness of opiate treatment with morphine will result in shorter duration of opiate medication treatment and fewer infants treated with a second drug.
- Trial name
- NAS (NCT01804075)
- Testing
- Methadone, Morphine
- Volunteers
- 31 people
- Sponsor
- Eastern Maine Medical Center
Hemophilia A
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older.
- Trial name
- HAVEN 3 (NCT02847637)
- Testing
- Emicizumab, Factor VIII (FVIII)
- Where
- 39 sites in United States, Australia and 12 more
- Volunteers
- 152 people
- Sponsor
- Hoffmann-La Roche
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Alvocidib, Cytarabine, and Mitoxantrone Hydrochloride or Cytarabine and Daunorubicin Hydrochloride in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
This randomized phase II trial is studying how alvocidib, cytarabine, and mitoxantrone hydrochloride work compared to cytarabine and daunorubicin hydrochloride in treating patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01349972
- Testing
- alvocidib, daunorubicin hydrochloride, mitoxantrone hydrochloride
- Where
- 11 sites in United States
- Volunteers
- 172 people
- Sponsor
- National Cancer Institute (NCI)
Cystic Fibrosis
Rollover Study of VX-770 in Cystic Fibrosis Subjects
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF).
- Trial name
- NCT01117012
- Testing
- Ivacaftor
- Where
- 58 sites in United States, Australia and 6 more
- Volunteers
- 192 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Equinus Contracture
The Effect of Botulinum Toxin A Injections on Ankle Dorsiflexion Following Internal Fixation of Tibial Pilon Fractures
The purpose of this study is to determine if botulinum toxin type A (Botox) injections, at the time of surgery for pilon fractures, will improve ankle range-of-motion and functionality.
- Trial name
- Botox (NCT02051933)
- Testing
- Botulinum toxin type A, sodium chloride
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Wake Forest University Health Sciences
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea
Gastroschisis
Evaluation of Glycerin Suppositories to Improve Bowel Function in Gastroschisis
Gastroschisis is a congenital defect of the abdominal wall that leads to evisceration of various amounts of the abdominal organs.
- Trial name
- NCT03355326
- Testing
- Glycerin Suppository
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Indiana University
Hereditary Angioedema, Pediatric
Berotralstat Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic…
- Trial name
- APeX-P (NCT05453968)
- Testing
- Berotralstat
- Where
- 15 sites in Austria, Canada and 8 more
- Volunteers
- 29 people
- Sponsor
- BioCryst Pharmaceuticals
Hemophilia B
Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.
- Trial name
- NCT00714415
- Testing
- BeneFIX
- Where
- 21 sites in Austria, Germany
- Volunteers
- 80 people
- Sponsor
- Pfizer
Impetigo, Folliculitis
Twice Daily Altabax Application for the Treatment of Uncomplicated Soft Tissue Infection
The purpose of this study is to document the clinical and bacteriological efficacy of retapamulin in the treatment of subjects with bacterial infections, including impetigo, folliculitis, and minor soft tissue infections…
- Conditions
- Impetigo, Folliculitis, Secondarily Infected Eczema, Minor Soft Tissue Infections
- Trial name
- NCT01126268
- Testing
- Retapamulin (Altabax)
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- The University of Texas Health Science Center, Houston
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