Clinical trials: Fetal Developmental Abnormality
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Inherited Ophthalmic Diseases, Inherited Retinal Degeneration
Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)
Background: People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. This can cause vision loss.
- Trial name
- NCT02338973
- Testing
- Interferon gamma-1b
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Eye Institute (NEI)
Hereditary Angioedema
A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).
- Trial name
- NCT04654351
- Testing
- TAK-667
- Where
- 3 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Takeda
Dermatitis, Atopic
Crisaborole Ointment 2% Skin Biomarker Biopsy Study in Atopic Dermatitis
This study is being conducted to characterize the mechanism of action of crisaborole ointment 2%, by evaluation of efficacy and changes in key skin biomarkers in atopic dermatitis (AD) lesions treated with crisaborole ointment 2%…
- Trial name
- NCT03233529
- Testing
- Crisaborole ointment 2% BID
- Where
- Canada
- Volunteers
- 40 people
- Sponsor
- Pfizer
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Atopic Dermatitis
Phase 2 Study of OPA-15406 Ointment in Pediatric Patients With Atopic Dermatitis
To evaluate the safety, efficacy (dose response) and pharmacokinetics of 0.3% and 1% OPA-15406 when applied twice daily for 4 weeks in pediatric patients with atopic dermatitis.
- Trial name
- NCT03018691
- Testing
- OPA-15406
- Where
- 4 sites in Japan
- Volunteers
- 73 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Hemophilia, Factor XIII Deficiency
Clinical Trial of Factor XIII (FXIII) Concentrate
Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion.
- Trial name
- NCT00640289
- Testing
- Fibrogammin P
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Children's Hospital of Orange County
Maternal Death, Maternal Infections Affecting Fetus or Newborn
Azithromycin-Prevention in Labor Use Study (A-PLUS)
Maternal and neonatal infections are among the most frequent causes of maternal and neonatal deaths, and current antibiotic strategies have not been effective in preventing many of these deaths.
- Conditions
- Maternal Death, Maternal Infections Affecting Fetus or Newborn, Neonatal SEPSIS, Maternal Sepsis During Labor
- Trial name
- NCT03871491
- Testing
- Azithromycin
- Where
- 8 sites in Bangladesh, Democratic Republic of the Congo and 5 more
- Volunteers
- 58,747 people, healthy volunteers welcome
- Sponsor
- NICHD Global Network for Women's and Children's Health
Retinopathy of Prematurity (ROP)
Inositol to Reduce Retinopathy of Prematurity
This is a Phase 3, randomized, double-masked, placebo-controlled study designed to determine the effectiveness of myo-Inositol 5% Injection to increase the incidence of survival without severe Retinopathy of Prematurity (ROP)…
- Trial name
- INS-3 (NCT01954082)
- Testing
- myo-Inositol 5% Injection
- Where
- 19 sites in United States
- Volunteers
- 638 people
- Sponsor
- NICHD Neonatal Research Network
Cystic Fibrosis
Study of Ataluren (PTC124) in Cystic Fibrosis
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study…
- Trial name
- NCT02107859
- Testing
- Ataluren
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 61 people
- Sponsor
- PTC Therapeutics
Dermatitis, Atopic Dermatitis
Efficacy and Safety Study of Apremilast in Subjects With Moderate to Severe Atopic Dermatitis
A study to evaluate the efficacy and safety of apremilast (CC-10004) in subjects with moderate to severe atopic dermatitis
- Trial name
- NCT02087943
- Testing
- Apremilast
- Where
- 31 sites in United States, Canada and 1 more
- Volunteers
- 191 people
- Sponsor
- Amgen
Patent Ductus Arteriosus, Preterm Infant
Randomized Controlled Trial to Evaluate the Safety and Efficacy of Acetaminophen in Preterm Infants Used in Combination With Ibuprofen for Closure of the Ductus Arteriosus
The purpose of the present study is to determine whether treatment of hemodynamically significant patent ductus arteriosus with a combined therapy of intravenous Ibuprofen and oral acetaminophen has higher success rate in closing…
- Conditions
- Patent Ductus Arteriosus, Preterm Infant, Bronchopulmonary Dysplasia
- Trial name
- NCT03701074
- Testing
- Ibuprofen and acetaminophen
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of South Alabama
Hemophilia B
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02618915
- Testing
- DTX101
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Cystinosis
Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body…
- Trial name
- NCT00001213
- Testing
- Cysteamine
- Where
- United States
- Volunteers
- 328 people
- Sponsor
- National Eye Institute (NEI)
Postoperative Ileus
Subcutaneous Methylnaltrexone Versus Placebo for Postoperative Ileus Prevention
This randomized controlled trial will prospectively evaluate the clinical benefit for subcutaneous methylnaltrexone (MNTX) in counteracting the obstipatory (causing constipation) effects of spinal surgery without increasing…
- Trial name
- NCT03852524
- Testing
- Methylnaltrexone
- Where
- United States
- Volunteers
- 82 people
- Sponsor
- Ohio State University
Myotonic Dystrophy 1
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
- Trial name
- NCT02858908
- Testing
- Tideglusib
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- AMO Pharma Limited
Atopic Dermatitis
Efficacy and Safety of Orally Administered DS107 in Adult Patients With Moderate to Severe Atopic Dermatitis
The objective of this study is to compare the efficacy and safety of orally administered DS107 (2g) versus placebo in the treatment of moderate to severe Atopic Dermatitis (AD).
- Trial name
- NCT03817190
- Testing
- DS107
- Where
- 50 sites in United States, Austria and 3 more
- Volunteers
- 219 people
- Sponsor
- DS Biopharma
Hypospadias
Effect of Steroids on Post-Operative Complications Following Proximal Hypospadias Repair
The primary objective of this study is to determine the efficacy of administering a course of postoperative oral steroids in pediatric patients undergoing proximal hypospadias repair as prevention against complications.
- Trial name
- NCT02162810
- Testing
- Prednisolone
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Francis Schneck
Alpha 1-Antitrypsin Deficiency
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)
The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor…
- Trial name
- NCT00242385
- Testing
- Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 25 people
- Sponsor
- Baxalta now part of Shire
Seizure Disorder, Hypogonadism
Treatment of Sexual Dysfunction in Hypogonadal Men With Epilepsy With Testosterone and Either Anastrozole or Placebo
The purpose of this study is to determine if treatment using a medication (anastrozole/Arimidex), which lowers estrogen levels in the blood is better than placebo, a tablet that does not contain any active medication, when…
- Conditions
- Seizure Disorder, Hypogonadism, Erectile Dysfunction
- Trial name
- NCT00179517
- Testing
- Anastrozole 1mg
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Beth Israel Deaconess Medical Center
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