Clinical trials: Protein Deficiency
Common Variable Immunodeficiency, Agammaglobulinemia
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period.
- Trial name
- NCT00520494
- Testing
- Vivaglobin
- Where
- 6 sites in Canada, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
Hemophilia, AIDS
Host Genetic Factors Influencing HIV1 and HCV Viral Loads and AIDS Clinical Progression in a Hemophilia Cohort (HGDS-3)
Background: Over 80% of the hemophiliac population who became infected with HIV prior to 1985 are also co-infected with HCV. Thus, hemophiliacs represent an important population for studies of the natural history of these chronic…
- Trial name
- NCT00340548
- Where
- 15 sites in United States
- Volunteers
- 333 people
- Sponsor
- National Cancer Institute (NCI)
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.
- Trial name
- NCT04632953
- Testing
- No Intervention
- Where
- 16 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
HIV Infections, Pregnancy
Active Immunization of HIV-1 Infected, Pregnant Women With CD4 Lymphocyte Counts >= 400/mm3: A Phase I Study of Safety and Immunogenicity of VaxSyn Recombinant gp160 (NOTE: Some Patients Receive Placebo)
To evaluate the safety of gp160 vaccine (VaxSyn) in HIV-1 infected pregnant women with CD4 counts >= 400 cells/mm3. To evaluate the immunogenicity of this vaccine in pregnant women and the passive acquisition of vaccine-specific…
- Conditions
- HIV Infections, Pregnancy, HIV Seronegativity
- Trial name
- NCT00000777
- Testing
- gp160 Vaccine (MicroGeneSys)
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
Congenital Afibrinogenemia, Hypofibrinogenemia
FIB Grifols Congenital Deficiency for On-demand Treatment and Surgical Prophylaxis
This is a phase 3, multi-center, prospective, open-label, single-arm, clinical trial to be carried out in subjects with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia.
- Trial name
- NCT04636268
- Testing
- FIB Grifols
- Sponsor
- Instituto Grifols, S.A.
Protein C Deficiency
Efficacy and Safety Study of Protein C Concentrate in Subjects With Severe Congenital Protein C Deficiency
The purpose of this study is to show that Protein C Concentrate is a safe and effective treatment for subjects with congenital protein C deficiency.
- Trial name
- NCT00157118
- Testing
- Protein C Concentrate (Human) Vapor Heated
- Where
- 12 sites in United States
- Volunteers
- 18 people
- Sponsor
- Baxalta now part of Shire
Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital
Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe
This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor).
- Conditions
- Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital, Autosomal Hemophilia A, Classic Hemophilia
- Trial name
- NCT03034044
- Volunteers
- 106 people
- Sponsor
- Pfizer
Primary Immunodeficiency Disease
Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients
The purpose of this study was to assess efficacy and safety of Kedrion Immunoglobulin 10% (KIg10) in participants with Primary Immunodeficiency (PID).
- Trial name
- CARES10 (NCT03961009)
- Testing
- Kedrion IVIG 10%
- Where
- 11 sites in United States
- Volunteers
- 47 people
- Sponsor
- Kedrion S.p.A.
XLA, CVID
Immune Regulation in Patients With Common Variable Immunodeficiency and Related Inborn Errors of Immunity (IEI)
This study aims to understand the causes and progression of Common Variable Immunodeficiency (CVID) and related inborn errors of immunity (IEI).
- Conditions
- XLA, CVID, Yao Syndrome, Blau Syndrome
- Trial name
- NCT00001244
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
IgG Deficiency, Infections
Treatment of Deficient Subclass or Anti-polysaccharide Antibody Response
There is no consensus on the treatment of patients with recurrent infections and isolated immunoglobulin G (IgG)-subclass deficiency and/or selective antipolysaccharide antibody deficiency.
- Trial name
- Subklasse (NCT00522821)
- Testing
- intravenous immunoglobulins, antibiotics
- Where
- 9 sites in Netherlands
- Volunteers
- 55 people
- Sponsor
- Prothya Biosolutions
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)
Congenital Afibrinogenemia, Congenital Hypofibrinogenemia
Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
This was a prospective, open-label, multicenter, phase I/III study investigating the 14-day single-dose pharmacokinetic and pharmacodynamic properties, efficacy and safety of BT524 following intravenous administration in the…
- Trial name
- NCT02065882
- Testing
- BT524 (Part I), BT524 (Part II)
- Where
- 6 sites in Bulgaria, Egypt and 3 more
- Volunteers
- 67 people
- Sponsor
- Biotest
Factor VII Deficiency
Study Comparing the Pharmacokinetic of Biosimilar Eptacog Alfa With Novoseven®, in Patients With Congenital Factor VII Deficiency
The purpose of this multicentre, randomized, double blinded, single dose, two-way cross-over study, is to compare the pharmacokinetics (PK) of biosimilar eptacog alfa (activated) with Novoseven in 24 patients, adult and children…
- Trial name
- NCT03079063
- Testing
- Eptacog alfa, biosimilar, Novoseven
- Where
- 2 sites in Iran
- Volunteers
- 24 people
- Sponsor
- AryoGen Pharmed Co.
Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN)
TIRCON International NBIA Registry
TIRCON-reg aims to continue the provision of a global registry and natural history study for NBIA disorders harmonize and cover existing national and single site registries enable participation of countries and single sites that…
- Conditions
- Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN), Beta-Propeller Protein-Associated Neurodegeneration (BPAN), Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN)
- Trial name
- TIRCON (NCT05522374)
- Where
- 9 sites in Canada, Czechia and 6 more
- Volunteers
- 2,000 people
- Sponsor
- LMU Klinikum
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IgPro10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168025
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 89 people
- Sponsor
- CSL Behring
Primary Immunodeficiency, Agammaglobulinemia
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
- Conditions
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
- Trial name
- NCT01581593
- Testing
- Kedrion IVIG 10%
- Where
- 15 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Kedrion S.p.A.
Factor X Deficiency
A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months. The secondary objectives of the study are: 1.
- Trial name
- NCT01721681
- Testing
- FACTOR X
- Where
- 3 sites in United Kingdom
- Volunteers
- 9 people
- Sponsor
- Bio Products Laboratory
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)