Clinical trials: Congenital Deformity
Atopic Dermatitis, Healthy Volunteers
Safety Study of Tezepelumab (AMG 157) in Healthy Adults and Adults With Atopic Dermatitis
This study is a single dose escalation study of tezepelumab (AMG 157) in healthy adults (Part A) and adults with moderate to severe atopic dermatitis (Part B).
- Trial name
- NCT00757042
- Testing
- Tezepelumab
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Amgen
Thyroid Eye Disease
Twenty-four Hour Intraocular Pressure Patterns in Patients With Uncontrolled Thyroid Eye Disease
Patients with Thyroid Eye Disease (TED) often have enlarged extraocular muscles and higher orbital fat contents due to their disease process.
- Trial name
- NCT01798966
- Testing
- SENSIMED Triggerfish
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Sensimed AG
Cystic Fibrosis
To Assess the Safety, Tolerability and Pharmacokinetics of AZD5634 Following Inhaled and Intravenous (IV)Dose Administration
This is a Phase 1, first-in-human (FIH) single ascending dose study being conducted to better understand the safety, tolerability and pharmacokinetics of AZD5634 in healthy subjects
- Trial name
- NCT02679729
- Testing
- AZD5634 for inhalation, AZD5634 for infusion
- Where
- 2 sites in United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Homozygous Familial Hypercholesterolemia
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- ORION-5 (NCT03851705)
- Testing
- Inclisiran Sodium for injection
- Where
- 13 sites in Hong Kong, Israel and 6 more
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Hereditary Tyrosinemia, Type I
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product 1) and Nitisinone 10 mg Tablets 'Baked' for 6 months @ 40°C/75% RH (Test Product 2) are bioequivalent to the reference product Orfadin 10 mg…
- Trial name
- NCT02750345
- Testing
- Nitisinone, Nitisinone Baked Tablet, Orfadin
- Where
- South Africa
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Neurofibromatosis 1
Medical Treatment of "High-Risk" Neurofibromas
Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction…
- Trial name
- NCT00846430
- Testing
- Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Corewell Health West
Huntington Disease
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
- Trial name
- NCT04556656
- Testing
- Pridopidine
- Where
- 58 sites in United States, Austria and 9 more
- Volunteers
- 499 people
- Sponsor
- Prilenia
Cystic Fibrosis
Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease
The purpose of this study is to determine whether sildenafil can decrease inflammation in CF lung disease.
- Trial name
- NCT00659529
- Testing
- sildenafil
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- National Jewish Health
Growth Hormone Deficiency, Panhypopituitarism
Growth Hormone and Endothelial Function in Children
Objective: This study is designed to determine whether growth hormone treatment in children 8 to 18 years of age alters function of the lining of the arteries.
- Conditions
- Growth Hormone Deficiency, Panhypopituitarism, Short Stature
- Trial name
- NCT00373386
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ohio State University
Cleft Palate
Timing of Primary Surgery for Cleft Palate
This trial is a randomised controlled trial with a parallel design taking place in centres across the UK, Scandinavia and Brazil.
- Trial name
- TOPS (NCT00993551)
- Testing
- primary surgery for cleft palate
- Where
- 23 sites in Brazil, Denmark and 3 more
- Volunteers
- 558 people
- Sponsor
- Kevin Munro
Chronic Pancreatitis
Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis
Primary objective: To describe and compare the safety and efficacy of treatment with AAT in chronic pancreatitis patients who undergo total pancreatectomy and islet autotransplantation (TP-IAT).
- Trial name
- NCT02947087
- Testing
- Prolastin-C
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical University of South Carolina
Arrhythmogenic Right Ventricular Dysplasia, Brugada Syndrome
Distribution of Cell-cell Junction Proteins in Arrhythmic Disorders
Every week in the UK, 12 apparently healthy and fit individuals under the age of 35 die suddenly, a tragic event known as sudden cardiac death (SCD).
- Conditions
- Arrhythmogenic Right Ventricular Dysplasia, Brugada Syndrome, Cardiac Channelopathy
- Trial name
- NCT04257994
- Testing
- obtaining a buccal smear sample
- Where
- United Kingdom
- Volunteers
- 26 people
- Sponsor
- St. George's Hospital, London
Hypothalamic Obesity
A Trial of Setmelanotide in Acquired Hypothalamic Obesity
The goal of this trial is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with acquired Hypothalamic Obesity (HO).
- Trial name
- NCT05774756
- Testing
- Setmelanotide
- Where
- 28 sites in United States, Canada and 4 more
- Volunteers
- 143 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Fragile X Syndrome
Neurophysiological and Acute Pharmacological Studies in FXS Patients
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.
- Trial name
- NCT02998151
- Testing
- Acamprosate, Lovastatin, Minocycline
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
CAH - Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, dose-escalation study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 in up to 30 adult female and male subjects (18 to 50 years of…
- Trial name
- NCT03525886
- Testing
- NBI-74788
- Where
- 6 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neurocrine Biosciences
Hypophosphatasia
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01176266
- Testing
- asfotase alfa
- Where
- 23 sites in United States, Australia and 10 more
- Volunteers
- 69 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Graft Versus Host Disease, Leukemia
Busulfan, Cyclophosphamide, & Antithymocyte Globulin Followed by Stem Cell Transplant in Treating Hematologic Cancer
RATIONALE: Giving chemotherapy before a donor bone marrow transplant or peripheral stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00611351
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Nebraska
Growth Disorders, Intrauterine Growth Retardation
Genotropin Treatment in Short Prepubertal Children With Intra-Uterine Growth Retardation
To evaluate the effect of continuous and intermittent administration of Genotonorm on stature in short prepubertal children with intra-uterine growth retardation
- Trial name
- NCT01073605
- Testing
- Genotonorm
- Where
- 31 sites in France
- Volunteers
- 208 people
- Sponsor
- Pfizer
G6PD Deficiency
Validation of Diagnostics to Identify Glucose-6-phosphate Dehydrogenase Activity in the US
The primary objective of this study is to assess the accuracy of the SD Biosensor STANDARD G6PD Analyzer in measuring G6PD activity when used by trained health care workers.
- Trial name
- NCT04010695
- Testing
- SD Biosensor G6PD Analyzer, Pointe Scientific Test Kit, HemoCue System
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- PATH
Gout, Chronic Kidney Diseases
CSP594 Comparative Effectiveness in Gout: Allopurinol vs. Febuxostat
This trial will compare two effective therapies, allopurinol and febuxostat, to lower serum uric acid and therefore prevent further gout attacks. These therapies have never been compared at appropriate doses.
- Trial name
- NCT02579096
- Testing
- allopurinol capsule, 100-800 mg by mouth once daily, febuxostat tablet 40-120 mg by mouth once daily
- Where
- 23 sites in United States
- Volunteers
- 950 people
- Sponsor
- VA Office of Research and Development
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