Clinical trials: Protein Deficiency
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) 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
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
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
Insulin Sensitivity
Effect of Vitamin D Replacement on Insulin Sensitivity
Healthy subjects with low Vitamin D levels will be randomly assigned to either Vitamin D replacement or placebo for a period of 8 weeks.
- Trial name
- NCT01268111
- Testing
- Ergocalciferols
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Texas Tech University Health Sciences Center
HIV Infections
A Study to Assess the Safety and Immune Response to Env-C DNA and Protein Vaccines in Kenya
This is a study of HIV vaccines. A vaccine is a medical product given to prevent certain diseases. The vaccine may educate the body to form a defensive response to try to prevent the disease from the beginning, or preventing it…
- Trial name
- NCT04826094
- Testing
- Env-C Plasmid DNA, HIV Env gp145 C.6980 protein, Rehydragel®
- Where
- Kenya
- Volunteers
- 143 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Congenital Protein C Deficiency
A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
Pharmacokinetic Part: This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time.
- Trial name
- NCT04984889
- Testing
- TAK-662
- Where
- 4 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda
Antithrombin III Deficiency, Covid19
Antithrombin III in Infectious Disease Caused by COVID-19
The purpose of this research study is to see if participants who have SARS-CoV-2 and low levels of AT3 in the blood will benefit by being given AT3.
- Trial name
- NCT04899232
- Testing
- Antithrombin III
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Enrique Ginzburg
HIV Infections
An International Study to Evaluate Recombinant Interleukin-2 in HIV Positive Patients Taking Anti-retroviral Therapy
The purpose of this study is to see if it is effective to give HIV positive patients recombinant interleukin-2 (rIL-2) in addition to anti-HIV therapy.
- Trial name
- ESPRIT (NCT00004978)
- Testing
- Recombinant interleukin-2 (rIL-2)
- Where
- 248 sites in United States, Argentina and 23 more
- Volunteers
- 4,150 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal…
- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1
- Trial name
- RICHI (NCT01998633)
- Testing
- Hematopoietic Stem Cell Transplant
- Where
- 22 sites in United States, Canada
- Volunteers
- 47 people
- Sponsor
- Medical College of Wisconsin
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing Nivolumab as a Potential Targeted Treatment in Cancers With Mismatch Repair Deficiency (MATCH-Subprotocol Z1D)
This phase II MATCH treatment trial identifies the effects of nivolumab in patients whose cancer has a genetic change called mismatch repair deficiency.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439214
- Testing
- Nivolumab
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- National Cancer Institute (NCI)
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
Hemophilia B
A Gene Therapy Study for Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
- Trial name
- NCT02484092
- Testing
- SPK-9001
- Where
- 10 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Pfizer
Factor X Deficiency
Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery
To primary efficacy variable is to assess the presence or absence of excessive blood loss during and after surgery. The secondary efficacy endpoints are as follows: 1.
- Trial name
- Ten03 (NCT01086852)
- Testing
- FACTOR X
- Where
- 7 sites in United States, Spain and 2 more
- Volunteers
- 4 people
- Sponsor
- Bio Products Laboratory
HIV Infections
Evaluating the Safety and Immunogenicity of an HIV Vaccine (gp145 C.6980) in Healthy, HIV-Uninfected Adults in the United States
The purpose of this study is to evaluate the safety, tolerability, and immunogenicity of an HIV vaccine (gp145 C.6980) with aluminum hydroxide adjuvant in healthy, HIV-1-uninfected adults in the United States.
- Trial name
- NCT03382418
- Testing
- gp145 C.6980 Vaccine, Aluminum Hydroxide Adjuvant
- Where
- 3 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency
Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy.
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- Triheptanoin (NCT01379625)
- Testing
- Triheptanoin
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- Oregon Health and Science University
Hemophilia A
Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A
The purpose of this study is to evaluate the rate of response when administering rituximab to suppress or eliminate the anti-body in a patient's blood that inhibits the effectiveness of their factor replacement product compared…
- Trial name
- NCT00306670
- Testing
- Rituxan, prednisone
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency
High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT01494051
- Testing
- Diet counseling
- Volunteers
- 13 people
- Sponsor
- Oregon Health and Science University
Factor VIII Deficiency
A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
Primary Objective: To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-1 (NCT04161495)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 47 sites in United States, Argentina and 17 more
- Volunteers
- 159 people
- Sponsor
- Bioverativ, a Sanofi company
Agammaglobulinemia, IgG Deficiency
Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)
The objectives of this trial are the assessment of safety and efficacy of IgPro10 in patients with PID, and the assessment of tolerability of high infusion rates.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00322556
- Testing
- Immunoglobulins Intravenous (Human)
- Where
- 10 sites in United States
- Volunteers
- 55 people
- Sponsor
- CSL Behring
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period.
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome
- Trial name
- NCT01289847
- Testing
- Gammaplex
- Where
- 10 sites in United States, Chile and 1 more
- Volunteers
- 25 people
- Sponsor
- Bio Products Laboratory