Clinical trials: Deficiency Disease
Hereditary Angioedema (HAE)
A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland
The main aim of this study is to learn about how many persons with HAE type I or type II are attack-free when treated with lanadelumab in real life, how many attacks occur and how many of these attacks need rescue treatment and…
- Trial name
- CHOPIN (NCT05147181)
- Where
- 14 sites in Poland
- Volunteers
- 48 people
- Sponsor
- Takeda
Nutritional Support
Interpretation and Optimization of Nutrition in the Intensive Care Units
Despite recommendations, inadequate nutritional intake in intensive care unit (ICU) patients remains frequent and can lead to complications such as infections, increased length of stay, prolonged weaning from ventilation…
- Trial name
- IONIC (NCT06342895)
- Testing
- Nutritional support prescription and delivery monitoring feedback, Usual care
- Where
- France
- Volunteers
- 144 people
- Sponsor
- Dim3
Alpha1-Antitrypsin Deficiency
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency
This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of Alpha-1 MP (alpha1-proteinase inhibitor (human), modified process) for 8 weeks.
- Trial name
- NCT02870309
- Testing
- Alpha-1 MP
- Where
- 2 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC
HIV Infections, Smallpox
Safety Study of MVA Smallpox Vaccine in HIV-positive Subjects Who Are Vaccinia Naive
The purpose of this study is to assess the safety and immunogenicity of two MVA smallpox vaccine injections in healthy adults that are 18-35 years of age with HIV infection
- Trial name
- NCT00282581
- Testing
- MVA Smallpox Vaccine
- Where
- 6 sites in United States
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
HIV Infection
Single DermaVir Immunization in HIV-1 Infected Patients on HAART
DermaVir is a plasmid DNA-containing synthetic nanomedicine. It is administered topically with DermaPrep to target Langerhans cells.
- Trial name
- GIHU004 (NCT00712530)
- Testing
- DermaVir, HAART
- Where
- Hungary
- Volunteers
- 9 people
- Sponsor
- Genetic Immunity
Alpha1-antitrypsin Deficiency
Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
The study was a Phase 1B/2A, uncontrolled, open-label, single-center study in individuals with congenital AAT (alpha 1-antitrypsin) deficiency.
- Trial name
- NCT00157092
- Testing
- Aerosolized, Recombinant Alpha 1-Antitrypsin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Baxalta now part of Shire
Dravet Syndrome, Lennox Gastaut Syndrome
Intermediate-Size Expanded Access Protocol (EAP) for LP352
This is an intermediate-size expanded access program (EAP) study. The purpose of this EAP is to provide continued access to LP352, an investigational drug product being investigated in participants with DEEs.
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Developmental and Epileptic Encephalopathies
- Trial name
- NCT06149663
- Testing
- LP352
- Where
- 22 sites in United States, Australia
- Sponsor
- Longboard Pharmaceuticals
Familial Lipoprotein Lipase Deficiency
Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects
This trial is designed to expand the currently available data on the safety and efficacy of alipogene tiparvovec treatment in lipoprotein lipase deficiency (LPLD) and to further the understanding of possible mechanisms of action…
- Trial name
- NCT00891306
- Testing
- Alipogene Tiparvovec (AMT-011), Human LPL [S447X], mycophenolate mofetil, cyclosporine
- Where
- 2 sites in Canada
- Volunteers
- 5 people
- Sponsor
- Amsterdam Molecular Therapeutics
Diagnosis of Adult Growth Hormone Deficiency (AGDH)
Investigation of a New, Oral Growth Hormone Secretagogue, AEZS-130 as a Growth Hormone Stimulation Test.
The diagnosis of growth hormone deficiency (GHD) in adults is established by laboratory testing in patients with an appropriate clinical history of hypothalamic pituitary disease.
- Trial name
- NCT00448747
- Testing
- AEZS-130 (formerly ARD-07), L-ARG+GHRH
- Where
- 13 sites in United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- AEterna Zentaris
Cytomegalovirus Retinitis, HIV Infections
A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS
To evaluate the safety and tolerance of long-term ganciclovir (DHPG) therapy for newly diagnosed macular threatening Cytomegalovirus (CMV) retinitis in AIDS patients.
- Trial name
- NCT00002034
- Testing
- Zidovudine, Ganciclovir
- Where
- 9 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Hoffmann-La Roche
HIV, AIDS
HIV Cohort Study At Johns Hopkins University, University of North Carolina at Chapel Hill and Vanderbilt University
Human Immunodeficiency Virus (HIV) infected patients in the HIV registries of Johns Hopkins University, University of North Carolina and Vanderbilt University will be followed in the routine clinical care to estimate the rates of…
- Trial name
- NCT01339416
- Volunteers
- 8,202 people
- Sponsor
- ViiV Healthcare
Hereditary Angioedema, HAE
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in participants with hereditary angioedema (HAE).
- Trial name
- OPuS-2 (NCT02303626)
- Testing
- BCX4161
- Where
- 40 sites in United States, Belgium and 6 more
- Volunteers
- 110 people
- Sponsor
- BioCryst Pharmaceuticals
Infection, Human Immunodeficiency Virus, HIV Infections
A Study of GSK1349572 Versus Raltegravir (RAL) With Investigator Selected Background Regimen in Antiretroviral-Experienced, Integrase Inhibitor-Naive Adults
ING111762 is a 48 week, randomized, double-blind, active-controlled, multicenter, parallel group, non-inferiority study. The study will be conducted in at least 688 HIV-1 infected antiretroviral experienced, integrase-naïve…
- Trial name
- SAILING (NCT01231516)
- Testing
- GSK1349572, Raltegravir
- Where
- 180 sites in United States, Argentina and 18 more
- Volunteers
- 724 people
- Sponsor
- ViiV Healthcare
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset)
Alglucosidase Alfa Temporary Access Program
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- ATAP (NCT00520143)
- Testing
- alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
- Where
- 74 sites in United States
- Sponsor
- Genzyme, a Sanofi Company
Meningitis, Cryptococcal, HIV Infections
Comparison of Fluconazole and Amphotericin B in the Treatment of Brain Infections in Patients With AIDS
To compare the safety and effectiveness of a new drug, fluconazole, with that of the usual therapy, amphotericin B, in the prevention of a relapse of cryptococcal meningitis (CM) in patients with AIDS who have been successfully…
- Trial name
- NCT00001017
- Testing
- Fluconazole, Amphotericin B
- Where
- 28 sites in United States
- Volunteers
- 330 people
- Sponsor
- Pfizer
Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency
Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
This study will demonstrate Short-, mid-, and long-term safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this…
- Conditions
- Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency, Neurocognitive Deficit, Malnutrition
- Trial name
- NCT04807478
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Sponsor
- Fresenius Kabi
Acute Myeloid Leukemia, Myelodysplastic Syndrome
A Phase I, Open-Label, Dose Escalation and Cohort Expansion Study of BS HH 002.SA in Patients With AML and MDS
Indication:Relapsed or refractory AML in patients for whom no established treatment options are available (this indication will heretofore be referred to as the protocol AML indication), or adult patients with MDS who are…
- Trial name
- NCT04743115
- Testing
- BS HH 002.SA
- Volunteers
- 66 people
- Sponsor
- Shanghai Bensen Pharmaceutical Co., Ltd.
Iron Deficiency Anemia (IDA)
Evaluate the Utility of Serum Hepcidin Levels to Predict Response to Oral or IV Iron
The main objective of this study is to compare the safety, effect on quality of life, and resource utilization of Injectafer vs.
- Trial name
- NCT01950247
- Testing
- Injectafer, SOC
- Volunteers
- 1,025 people
- Sponsor
- American Regent, Inc.
HIV-Infections
TMC125HIV1083 - Swallowability of Uncoated 200 mg Etravirine Tablets in HIV-1 Infected Patients
This trial is designed to assess the swallowability of uncoated and film-coated 200 mg etravirine tablets in HIV-1 infected patients.
- Trial name
- NCT01090648
- Testing
- etravirine
- Volunteers
- 49 people
- Sponsor
- Tibotec Pharmaceuticals, Ireland
HIV Infections
A Study of the Safety and Effectiveness of Combination Anti-HIV Therapy in HIV-Infected Adults
The purpose of this study is to see if a certain combination of anti-HIV drugs is safe and effective in HIV-infected patients. The drug combination includes a tablet containing lamivudine and zidovudine (called Combivir) plus…
- Trial name
- NCT00004585
- Testing
- Abacavir sulfate, Lamivudine and Zidovudine, Lamivudine/Zidovudine, Abacavir sulfate
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Glaxo Wellcome
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