Clinical trials: Protein Deficiency
HIV Infections, Cytomegalovirus
CMV Disease and IRIS in HIV-1 Infected Persons
Various diagnostic methods are available for CMV infection. But none of them could be a standard and highly valuable. Our first goal is to setup a series of molecular diagnostic tools for HIV-1 infected person.
- Trial name
- NCT00456664
- Testing
- IE gene
- Where
- Taiwan
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
HIV Infections
An Escalating Dose Tolerance Trial of BG8962 (rCD4) in Patients Who Are HIV Antibody Positive
To determine the maximal safe daily dose of BG8962 (rCD4) which can be administered by continuous subcutaneous infusion (CSCI) over 24 hours; to determine the pharmacokinetics of BG8962 when it is administered by intramuscular…
- Trial name
- NCT00000721
- Testing
- CD4 Antigens
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168012
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 42 people
- Sponsor
- CSL Behring
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or…
- Trial name
- NCT04812106
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Inborn Errors of Metabolism
Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
Usual dietary therapies of mitochondrial fatty acid oxidation disorders (FAO) are based on 3 strategies: limitation of lipid intake in the diet; supplementation of the diet with medium-chain triglycerides (MCT) for patients…
- Trial name
- NCT00328159
- Testing
- Oil special 107 and MYGLIOL 810
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Lung Diseases, Respiratory Distress Syndrome, Newborn
Epidemiology of Surfactant Protein-B Deficiency
The purpose of this study is to test the hypothesis that excess, rare, functionally disruptive single nucleotide polymorphisms (SNPs) characterize genes (e.g., the surfactant protein-B gene)(SFTPB) and gene networks (e.g., the…
- Conditions
- Lung Diseases, Respiratory Distress Syndrome, Newborn, Pulmonary Surfactant, Lung Diseases, Interstitial
- Trial name
- NCT00014859
- Where
- United States
- Volunteers
- 5,176 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Kwashiorkor, Marasmus
Pilot Study of PUFA-optimized RUTF for Severe Acute Malnutrition
To test whether a ready-to-use therapeutic food (RUTF) enriched with polyunsaturated fatty acids (RUTF-P) is as effective for the treatment of severe acute malnutrition (SAM) as standard RUTF.
- Trial name
- NCT02053857
- Testing
- RUTF, RUTF-P, Amoxicillin
- Where
- Malawi
- Volunteers
- 141 people
- Sponsor
- Washington University School of Medicine
Mevalonate Kinase Deficiency
Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency
Mevalonate kinase deficiency (HyperIgD syndrome, HIDS) is an inborn error of immunity caused by a block in the mevalonate pathway.
- Trial name
- NCT06497829
- Testing
- geranylgeraniol
- Where
- Czechia
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University Hospital, Motol
Lymphoma, Non-Hodgkin Lymphoma
AZD3470 as Monotherapy or in Combination With Anticancer Agent(s) in Participants With Haematologic Malignancies.
This study is designed to evaluate the safety, tolerability, PK, pharmacodynamics, and preliminary efficacy following oral administration of AZD3470 as a monotherapy, and in combination with other anticancer agents in…
- Conditions
- Lymphoma, Non-Hodgkin Lymphoma, Hodgkin Lymphoma, Peripheral T-cell Lymphoma (PTCL)
- Trial name
- PRIMAVERA (NCT06137144)
- Testing
- AZD3470, Pembrolizumab
- Where
- 38 sites in United States, Australia and 9 more
- Volunteers
- 161 people
- Sponsor
- AstraZeneca
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
- Trial name
- NCT05933200
- Testing
- Triheptanoin, MCT Oil
- Where
- 15 sites in Czechia, Germany and 5 more
- Volunteers
- 69 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
CD55-Deficient Protein-Losing Enteropathy
Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
The program is to provide access to an experimental drug called pozelimab and to document the long-term safety of pozelimab in patients with Protein-Losing Enteropathy (PLE).
- Trial name
- CHAPLE Disease (NCT06003881)
- Testing
- Pozelimab
- Sponsor
- Regeneron Pharmaceuticals
HIV Infections, Pregnancy
A Phase I Study of the Safety and Pharmacokinetics of Recombinant CD4 Immunoglobulin G (rCD4-IgG) in HIV-1 Seropositive Women During the Last Trimester of Pregnancy and Their Newborns
Part 1: To determine both the safety, tolerance, and pharmacokinetic profile (blood levels) of recombinant CD4 immunoglobulin G (rCD4-IgG) by intravenous bolus administration (given through the vein) in women with HIV infection…
- Trial name
- NCT00000642
- Testing
- CD4-IgG
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Factor XIII Deficiency
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00945906
- Testing
- FXIII Concentrate (Human) (FXIII)
- Where
- 25 sites in United States
- Volunteers
- 61 people
- Sponsor
- CSL Behring
Primary Immunodeficiency Disease
Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)
The purpose of this study is to assess efficacy, safety and pharmacokinetics of Kedrion Immunoglobulin 10% (KIg10) in pediatric patients with Primary Immunodeficiency Disease (PID).
- Trial name
- KIDCARES10 (NCT04944979)
- Testing
- Kedrion IVIG 10%
- Where
- 21 sites in United States, Hungary and 4 more
- Volunteers
- 30 people
- Sponsor
- Kedrion S.p.A.
Congenital Fibrinogen Deficiency, Afibrinogenemia
Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap
The purpose of this study is to investigate pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/RiaSTAPTM in patients with congenital fibrinogen deficiency
- Trial name
- NCT01575756
- Testing
- Octafibrin, Haemocomplettan® P or RiaSTAPTM
- Where
- 10 sites in United States, Bulgaria and 4 more
- Volunteers
- 22 people
- Sponsor
- Octapharma
Anogenital Warts, HIV Infection
Effectivity of Protein Purified Derivative Treatment for Anogenital Warts in HIV Patient
Warts are a common viral infection of the skin and are prevalent throughout the world, with an overall prevalence in the United States estimated at 2-20%.
- Conditions
- Anogenital Warts, HIV Infection, Tuberculin Purified Protein Derivative
- Trial name
- NCT06587542
- Testing
- Injection of protein purified derivative
- Volunteers
- 30 people
- Sponsor
- Universitas Padjadjaran
HIV Infections
A Phase I/II Double-Blind Controlled Trial to Determine the Safety and Immunogenicity of HIV-1 MN rgp160 Immuno AG Vaccine Therapy in HIV-Infected Individuals With Greater Than or Equal to 500/mm3 CD4+ T Cells and 200-400/mm3 CD4+ T Cells
To evaluate the safety and immunogenicity of HIV-1 MN rgp160 (Immuno-AG) in HIV-infected patients. To evaluate the immunogenicity of HIV-1 MN rgp160 immunogen by lymphocyte proliferation, specific antibody responses, and DTH…
- Trial name
- NCT00000822
- Testing
- Ritonavir, gp160 Vaccine (Immuno-AG), Stavudine
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
GLUT1DS1, Epilepsy
Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
Forty-five subjects receiving no dietary therapy with a proven G1D diagnosis will be enrolled. To evaluate the effect of C7 supplementation of a regular diet on a EEG activity in addition to IQ, language, working memory…
- Conditions
- GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders
- Trial name
- NCT03181399
- Testing
- Triheptanoin
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- University of Texas Southwestern Medical Center
Primary Immune Deficiency Disorder
Gamma Globulin Observations and Outcomes Database for Patients With Primary Immunodeficiency Disease (GOOD-SHEPARD-PI)
Primary: Demonstrate the utility of an electronic data capture (EDC) system (CareExchange™) using infusion nurse and patient measured physical, quality of life (QOL), respiratory, laboratory, and disability assessments in…
- Trial name
- NCT01883921
- Testing
- Immunoglobulin Therapy
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- BriovaRx Infusion Services
Child Nutrition Disorders
A Nutrition/Hygiene Education Program for the Prevention of Child Malnutrition in Rural Kenya
This study will determine if an education program about hygiene and child feeding practices, taught by local village community health workers, will improve child growth and decrease the prevalence of childhood malnutrition in a…
- Trial name
- NCT01679535
- Testing
- nutrition and hygiene education
- Where
- United States
- Volunteers
- 246 people, healthy volunteers welcome
- Sponsor
- University of New Mexico