Clinical trials: Fetal Developmental Abnormality
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
Phenylketonuria
The Effectiveness of Kuvan in Amish PKU Patients
The purpose of this study is to determine if Amish patients with PKU show responsiveness after a high dose, prolonged Saproterin trial.
- Trial name
- NCT02677870
- Testing
- saproterin dihydrochloride, Diet treatment
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University Hospitals Cleveland Medical Center
Fragile X Syndrome
Trial of Sertraline to Treat Children With Fragile X Syndrome
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and…
- Trial name
- NCT01474746
- Testing
- Sertraline
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Randi J. Hagerman, MD
Fetal Alcohol Syndrome, Attention Deficit Hyperactivity Disorder (ADHD)
Open-Label Study of the Long Term Tolerability and Safety of Atomoxetine in Children With FASD and ADD/ADHD
Determine if atomoxetine is safe and well tolerated by children with fetal alcohol syndrome.
- Trial name
- B4Z-US-050 (NCT00418262)
- Testing
- Atomoxetine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Oklahoma
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
Neonatal Abstinence Syndrome
Donor Human Milk in Neonatal Abstinence Syndrome
This study is designed to develop pilot data on the acceptability and benefit of donor human milk for infants undergoing pharmacologic treatment for NAS.
- Trial name
- DHM&NAS (NCT02182973)
- Testing
- donor human milk
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Neolac Inc dba Medolac Laboratories
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals
Dilated Cardiomyopathy, Heart Failure
PUFAs and Left Ventricular Function in Heart Failure
The purpose of this study is to test the hypothesis that n-3 PUFAs improve left ventricular systolic function in patients with stable chronic HF secondary to nonischemic dilated cardiomyopathy (NICM).
- Trial name
- CS-PUFA-02 (NCT01223703)
- Testing
- n-3 PUFAs
- Where
- Italy
- Volunteers
- 133 people
- Sponsor
- Università degli Studi di Brescia
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