Clinical trials: Newborn
Sickle Cell Disease
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs).
- Trial name
- NCT05098028
- Testing
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bausch Health Americas, Inc.
Neonatal Abstinence Syndrome, Neonatal Opiate Withdrawal Syndrome
Buprenorphine Pharmacometric Open Label Research Study of Drug Exposure
Neonatal withdrawal syndrome is a series of signs and symptoms in infants exposed to opioids in utero. Buprenorphine has demonstrated a 40% reduction in length of pharmacologic treatment compared to oral morphine.
- Trial name
- B-PHORE (NCT03608696)
- Testing
- Buprenorphine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Thomas Jefferson University
Childhood Glaucoma
Efficacy and Safety of Topically Applied Medical Therapy for the Treatment of Pediatric Glaucoma
Objectives: The study will assess the ocular hypotensive effect of latanoprost and dorzolamide in a selected sample of patients affected by primary Pediatric Glaucoma (PG), refractory to surgical procedures.
- Trial name
- NCT01527682
- Testing
- Latanoprost, Dorzolamide
- Where
- 2 sites in Italy
- Volunteers
- 37 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Hereditary Angioedema (HAE)
A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants
The objective of this study is to evaluate the efficacy, pharmacokinetics (PK), and safety of icatibant for the treatment of acute attacks in Japanese participants with type I or type II hereditary angioedema (HAE).
- Trial name
- NCT03888755
- Testing
- Icatibant
- Where
- 8 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Neurofibromatosis, NF1
Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas
This is a pilot study to determine if adults and children with neurofibromatosis type 1 who have plexiform tumors given Sutent® respond to this drug therapy.
- Conditions
- Neurofibromatosis, NF1, Plexiform Neurofibromas
- Trial name
- NCT01402817
- Testing
- Sutent®/Sunitinib
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Indiana University
Severe Hemophilia A
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
- Trial name
- NCT01712438
- Testing
- Human cl rhFVIII
- Where
- 37 sites in United States, Belarus and 15 more
- Volunteers
- 110 people
- Sponsor
- Octapharma
Hereditary Angioedema (HAE)
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.
- Trial name
- NCT05460325
- Testing
- Lanadelumab
- Where
- 4 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00759876
- Testing
- Ataluren
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), a fatal muscle degenerative disorder, arises from mutations in the dystrophin gene. Antisense therapy with the use of antisense oligonucleotides (AON) has the potential to restore effectively…
- Trial name
- NCT00159250
- Testing
- AVI-4658 (PMO)
- Where
- United Kingdom
- Volunteers
- 7 people
- Sponsor
- Imperial College London
Primary Immunodeficiency Diseases (PID)
Phase 2/3 Study of IGSC, 20% in PIDD
The purpose of this study is to develop a 20% subcutaneous (SC) immunoglobulin preparation for the treatment of patients with primary immunodeficiency diseases (PIDD).
- Trial name
- NCT01218438
- Testing
- Immune Globulin Intravenous (Human), 10% Solution, Immune Globulin Subcutaneous (Human), 20% Solution
- Where
- 18 sites in United States, Canada
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia…
- Trial name
- guardian™4 (NCT01493778)
- Testing
- turoctocog alfa
- Where
- 69 sites in United States, Algeria and 16 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Biliary Atresia
Safety Study of Intravenous Immunoglobulin (IVIG) Post-Portoenterostomy in Infants With Biliary Atresia
The Children Liver Disease Research and Education Network (ChiLDREN) is conducting a clinical trial to determine the feasibility, acceptability, tolerability and safety profile of IVIG treatment administered to infants after…
- Trial name
- PRIME (NCT01854827)
- Testing
- Intravenous immunoglobulin (IVIG)
- Where
- 7 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Pancreatic Cancer
Analyzing Childhood Recall Antigens in Patients With Pancreatic Cancer
The investigator is developing an immune therapy against pancreatic cancer. Immune cells, known as "T cells with tumor killing capacity", are involved in this immune therapy.
- Trial name
- NCT03848182
- Testing
- Gemcitabine, TT vaccine booster
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Atopic Dermatitis
A Study to Evaluate Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Therapies spread over the skin may not be enough to control the AD in trial participants who require systemic…
- Trial name
- Level Up (NCT05601882)
- Testing
- Dupilumab, Upadacitinib
- Where
- 257 sites in United States, Australia and 27 more
- Volunteers
- 920 people
- Sponsor
- AbbVie
Dystrophic Epidermolysis Bullosa
Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
This is a feasibility study to see if Granulocyte Colony Stimulating Factor (GCSF) is effective as a treatment of Dystrophic Epidermolysis Bullosa (EB). Patients will receive one course of treatment with the study drug.
- Trial name
- NCT01538862
- Testing
- Granulocyte Colony Stimulating Factor (GCSF)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
Gaucher Disease
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from…
- Trial name
- NCT03625882
- Where
- 35 sites in Japan
- Volunteers
- 63 people
- Sponsor
- Takeda
Familial Adenomatous Polyposis
Lyophilized Black Raspberries in Adults With Familial Adenomatous Polyposis (FAP)
This is a 36 week dietary intervention pilot study to evaluate the effects of lyophilized black raspberries on rectal polyp burden and biomarkers in subjects with FAP.
- Trial name
- NCT00770991
- Testing
- Black raspberry (BRB) Slurry, Black Raspberry (BRB) Suppositories
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- The Cleveland Clinic
Healthy
Study of PF 04965842 Effect on MATE1/2K Activity in Healthy Participants
This is a Phase 1, randomized, 2 way crossover, open label study of the effect of PF-04965842 on metformin (a probe for MATE1/2K activity) PK in healthy adult participants.
- Trial name
- NCT03796182
- Testing
- Metformin, PF-04965842
- Where
- Belgium
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Dental Fluorosis, Dental Caries
Effect of pH and Fluoride Concentration of Dentifrices on Caries Control
This study aims to assess the overall effect of pH and fluoride concentration of liquid dentifrices in the control of children dental caries of a fluoridated area, through visual inspection and the quantitative light-induced…
- Trial name
- EPHFCDCC (NCT01049503)
- Testing
- Low fluoride and conventional dentifrices with different pH
- Where
- Brazil
- Volunteers
- 315 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.