Clinical trials: Protein Deficiency
Factor VIII Deficiency, Congenital
Hemlibra in Mild Hemophilia A
This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild…
- Trial name
- NCT04567511
- Testing
- Emicizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Indiana Hemophilia &Thrombosis Center, Inc.
Acute Bacterial Infections, Acute Viral Infections
300 Antibody Diagnostic Test Kit
For the development of a Point of Care IVD test kit for acute phase disease detection against a variety of bacterial and viral infections.
- Trial name
- Antibody 300 (NCT01646411)
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- The Plasma Source
CD55-deficient Protein-losing Enteropathy, CHAPLE
Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
The primary objective of the study is to determine the effect of pozelimab on active CD55-deficient protein-losing enteropathy (PLE; CHAPLE).
- Trial name
- NCT04209634
- Testing
- Pozelimab
- Where
- 3 sites in United States, Thailand and 1 more
- Volunteers
- 10 people
- Sponsor
- Regeneron Pharmaceuticals
Acquired Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with acquired hemophilia A.
- Trial name
- NCT01178294
- Testing
- OBI-1
- Where
- 12 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Baxalta now part of Shire
HIV Infections, Systemic Lupus Erythematosus
Evaluating HIV-1 Neutralization Antibody Breadth in Response to HIV gp120 Protein Vaccine in HIV-uninfected Adults With Quiescent Systemic Lupus Erythematosus
The purpose of this study is to evaluate the breadth and potency of HIV-1 neutralizing antibody (nAb) responses and examine the safety and tolerability of an HIV gp120 protein vaccine (AIDSVAX® B/E) in HIV-uninfected adults…
- Trial name
- NCT03618056
- Testing
- AIDSVAX® B/E
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Liver Steatosis, Insulin Resistance
Effects of Amino Acid Supplementation on Liver Lipid Content and Protein Metabolism in Obese Children
The purpose of this study is to assess the effect of an eight-week dietary supplementation with essential amino acids plus arginine on liver and plasma lipid content, whole-body fat oxidation, whole-body insulin sensitivity…
- Conditions
- Liver Steatosis, Insulin Resistance, Whole Body Protein Metabolism, Body Composition, Beneficial
- Trial name
- NCT02337894
- Testing
- Essential amino acids plus arginine
- Volunteers
- healthy volunteers welcome
- Sponsor
- Arkansas Children's Hospital Research Institute
Factor VII Deficiency
Treatment of Inherited Factor VII Deficiency
FVII deficiency is a rare coagulation disorder. A limited number of patients are found in most treatment centres and countries. Treatment demands vary considerably amongst FVII deficient patients.
- Trial name
- STER (NCT01269138)
- Testing
- plasma derived Factor VII, recombinant FVIIa, Fresh Frozen Plasma
- Where
- Italy
- Volunteers
- 223 people
- Sponsor
- University of L'Aquila
Factor IX Deficiency
Natural History Study of Factor IX Treatment and Complications
This study will examine two groups of subjects with factor IX (FIX) deficiency: 1) those with a current or history of inhibitors to FIX, and; 2) groups of two or more affected brothers, with or without inhibitors.
- Trial name
- B-Natural (NCT02502409)
- Testing
- Standard care with blood and urine sample collection
- Where
- United States
- Volunteers
- 550 people
- Sponsor
- Skane University Hospital
Antithrombin III Deficiency
Atenativ Effect on Uterine Blood Flow and Preeclampsia
The study will be an open controlled pilot study of 6 patients with early-onset severe preeclampsia. Patients will receive Atenativ in addition to conventional therapy The patients will be followed up within the study until three…
- Trial name
- NCT02278575
- Testing
- Atenativ
- Where
- Sweden
- Sponsor
- Vastra Gotaland Region
HIV Infection
A Phase I Safety and Immunogenicity Preventive Vaccine Trial Based on the HIV-1 Tat and V2-deleted Env Proteins (ISS P-002)
This Phase I study was directed at evaluating the safety profile and the immunogenicity of the vaccination with recombinant HIV-1 Tat and V2-deleted Env (delta-V2 Env) proteins administered in association in healthy…
- Trial name
- NCT01441193
- Testing
- HIV-1 Tat/delta-V2 Env combined vaccine, HIV-1 delta-V2 Env vaccine, HIV-1 Tat vaccine 7.5 microg
- Where
- 3 sites in Italy
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Barbara Ensoli, MD
Hemophilia B, Factor IX Deficiency
Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency
OBJECTIVES: Assess the safety and long-term efficacy of monoclonal factor IX concentrate in patients with factor IX deficiency.
- Trial name
- NCT00004801
- Testing
- monoclonal factor IX replacement therapy
- Volunteers
- 3 people
- Sponsor
- National Center for Research Resources (NCRR)
HIV Infections
Safety of and Immune Response to an HIV Vaccine (SF-2 gp120) With or Without MTP-PE/MF59 Adjuvant
Part A: To compare the safety and immunogenicity of two dose levels of gp120 (CHO) in MF59 emulsion alone or with MTP-PE/MF59 adjuvant, administered at 0, 1, and 6 months.
- Trial name
- NCT00001019
- Testing
- rgp120/HIV-1 SF-2, MTP-PE/MF59
- Where
- 4 sites in United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Low CETP Activity, CETP Deficiency
Relationship Between CETP Deficiency and Atherosclerosis in Patients With Hyperalphalipoproteinemia
The aim of this study is to determine whether high high density lipoprotein-cholesterol(HDL-C) level and low Cholesteryl Ester Transfer Protein(CETP) activity is atherogenic or not in subjects who received health checkups.
- Conditions
- Low CETP Activity, CETP Deficiency, Hyperalphalipoproteinemia, Hyper-LDL-cholesterolemia
- Trial name
- INFINITY (NCT01916512)
- Where
- Japan
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Osaka University
Hemophilia B
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector).
- Trial name
- NCT00076557
- Testing
- Adeno-Associated Viral with Human Factor IX
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Avigen
Secondary Immune Deficiency
Single-arm Study of IgPro20 in Adults With Secondary Immune Deficiencies Due to Hematologic Malignancies Treated With B-cell Targeting Chimeric Antigen Receptor T-cell and T-cell Redirecting Therapies
This is a prospective, multicenter, open-label, single-arm study to assess the efficacy, safety, and pharmacokinetics (PK) of IgPro20 in adults with hematologic malignancies treated with B-cell targeting Chimeric antigen receptor…
- Trial name
- NCT07648264
- Testing
- IgPro20
- Where
- 60 sites in United States, Belgium and 9 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring
HIV Infections
Evaluating the Safety and Immunogenicity of the IHV01 Protein Vaccine Primed and Co-Administered With HIV DNA CON-S Env Vaccine in Healthy, HIV-1-Uninfected Adults
The purpose of this study is to evaluate the safety and immunogenicity of the IHV01 protein vaccine primed and co-administered with HIV DNA CON-S env vaccine in healthy, HIV-1-uninfected adults.
- Trial name
- NCT03505060
- Testing
- DNA CON-S env, IHV01
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Long Chain Fatty Acid Oxidation Disorders
Expanded Access to Triheptanoin
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
- Trial name
- NCT03773770
- Testing
- Triheptanoin
- Sponsor
- Ultragenyx Pharmaceutical Inc
Primary Immune Deficiency Disorder, Common Variable Immunodeficiency
The Impact of Exercise on Stress, Fatigue, and Quality of Life in Individuals With Primary Immunodeficiency Disease
This aim of this research project is to determine if low to moderate level exercise can have an impact on stress, fatigue, and quality of life for individuals diagnosed with a primary immunodeficiency disease.
- Conditions
- Primary Immune Deficiency Disorder, Common Variable Immunodeficiency, Specific Antibody Deficiency, Hypergammaglobulinemia
- Trial name
- NCT03211689
- Testing
- Exercise Program
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Stockton University
Squamous Cell Carcinoma of the Head and Neck, Melanoma
SL-279252 (PD1-Fc-OX40L) in Subjects With Advanced Solid Tumors or Lymphomas
This is a Phase 1 first in human, open label, multi-center, dose escalation and dose expansion study to evaluate the safety, tolerability, PK, anti-tumor activity and pharmacodynamic effects of SL-279252 in subjects with advanced…
- Conditions
- Squamous Cell Carcinoma of the Head and Neck, Melanoma, Non Small Cell Lung Cancer, Urothelial Carcinoma
- Trial name
- NCT03894618
- Testing
- SL-279252
- Where
- 5 sites in United States, Belgium and 2 more
- Volunteers
- 49 people
- Sponsor
- Shattuck Labs, Inc.
Hypophosphatasia
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042).
- Trial name
- NCT01205152
- Testing
- asfotase alfa
- Where
- 8 sites in United States, United Arab Emirates and 1 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.