Clinical trials: Protein Deficiency
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
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Factor X Deficiency
A Study Investigating Treatment Factor X in People With Factor X Deficiency
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg. The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding…
- Trial name
- NCT00930176
- Testing
- Human Coagulation FACTOR X
- Where
- 14 sites in United States, Germany and 3 more
- Volunteers
- 16 people
- Sponsor
- Bio Products Laboratory
Anal Intraepithelial Neoplasia, High Grade Squamous Intraepithelial Neoplasia
VGX-3100 and Electroporation in Treating Patients With HIV-Positive High-Grade Anal Lesions
This phase II trial studies the use of human papillomavirus (HPV) deoxyribonucleic acid (DNA) plasmids therapeutic vaccine VGX-3100 (VGX-3100) and electroporation in treating patients with human immunodeficiency virus…
- Conditions
- Anal Intraepithelial Neoplasia, High Grade Squamous Intraepithelial Neoplasia, HIV Positivity, Human Papillomavirus-16 Positive
- Trial name
- NCT03603808
- Testing
- Electroporation, HPV DNA Plasmids Therapeutic Vaccine VGX-3100, Laboratory Biomarker Analysis
- Where
- 8 sites in United States, Puerto Rico
- Volunteers
- 44 people
- Sponsor
- AIDS Malignancy Consortium
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases
The main objective of the study is to determine the pharmacokinetics profile of Subgam-VF. The secondary objectives are to assess the safety of Subgam-VF and refine the dose adjustment coefficient for Subgam-VF needed for…
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyperimmunoglobulin M Syndrome
- Trial name
- SCIG03 (NCT01884311)
- Testing
- Subgam
- Where
- 16 sites in United States
- Volunteers
- 38 people
- Sponsor
- Bio Products Laboratory
HIV Infections
Evaluating the Safety and Immunogenicity of ALVAC-HIV and MF59®- or AS01B-adjuvanted Bivalent Subtype C gp120 in Healthy, HIV-uninfected Adult Participants
A phase 1/2a clinical trial to evaluate the safety and immunogenicity of ALVAC-HIV (vCP2438) and of MF59®- or AS01B-adjuvanted clade C Env protein, in healthy, HIV-uninfected adult participants
- Trial name
- HVTN 120 (NCT03122223)
- Testing
- ALVAC-HIV (vCP2438), Bivalent subtype C gp120/MF59, Bivalent subtype C gp120/AS01(B)
- Where
- 12 sites in United States, Tanzania and 2 more
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hypogammaglobulinemia, Lung Transplantation
Intravenous Immunoglobulin (IVIG) in Lung Transplantation
The purpose of this study is to determine if intravenous immunoglobulin (IVIG) can prevent bacterial infections in lung transplant patients with low serum levels of immunoglobulin.
- Trial name
- NCT00115778
- Testing
- IVIG
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Columbia University
Chronic Lymphocytic Leukemia, Fatigue
Lenalidomide and Ofatumumab in Treating Participants With Previously Treated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
This phase II trial studies how well lenalidomide and ofatumumab work in treating participants with previously treated chronic lymphocytic leukemia or small lymphocytic lymphoma.
- Conditions
- Chronic Lymphocytic Leukemia, Fatigue, Fever, Hypogammaglobulinemia
- Trial name
- NCT01002755
- Testing
- Lenalidomide, Ofatumumab
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- M.D. Anderson Cancer Center
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
Hemophilia, Factor XIII Deficiency
Clinical Trial of Factor XIII (FXIII) Concentrate
Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion.
- Trial name
- NCT00640289
- Testing
- Fibrogammin P
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Children's Hospital of Orange County
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases
The primary objective is to demonstrate the bioequivalence of Gammaplex® 10 intravenous immunoglobulin (IGIV) and Gammaplex® 5% IGIV with respect to area under the curve within a 28-day dosing interval (AUC0-28) in a cohort of…
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyper-IgM Syndrome
- Trial name
- GMX07 (NCT01963143)
- Testing
- Gammaplex (5%), Gammaplex 10
- Where
- 17 sites in United States, Hungary and 1 more
- Volunteers
- 48 people
- Sponsor
- Bio Products Laboratory
Hypophosphatasia
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01163149
- Testing
- asfotase alfa
- Where
- 3 sites in United States, Canada
- Volunteers
- 19 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Antithrombin III Deficiency
Recombinant Human Antithrombin (rhAT) in Patients With Hereditary Antithrombin Deficiency Undergoing Surgery or Delivery
Patients with hereditary antithrombin deficiency are at increased risk of venous thrombosis and pulmonary embolism, particularly during certain high risk procedures.
- Trial name
- NCT00110513
- Testing
- Recombinant human antithrombin (rhAT)
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 18 people
- Sponsor
- rEVO Biologics
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
HIV-associated Enteropathy
Impact of SBI, a Medical Food, on Nutritional Status in Patients With HIV-associated Enteropathy
The purpose of the study is to evaluate the effectiveness of the oral nutritional therapy serum-derived bovine immunoglobulin protein isolate (SBI) 2.5 g twice a day (BID) and SBI 5.0 g versus placebo on supporting nutrient…
- Trial name
- NCT01828593
- Testing
- Serum-derived bovine immunoglobulin protein isolate (SBI)
- Where
- 10 sites in United States
- Volunteers
- 103 people
- Sponsor
- Entera Health, Inc
Hypophosphatasia
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01176266
- Testing
- asfotase alfa
- Where
- 23 sites in United States, Australia and 10 more
- Volunteers
- 69 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further…
- Trial name
- NCT02531867
- Testing
- Asfotase Alfa
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Diabetic Nephropathy, Proteinuria
N-Acetylcysteine and Milk Thistle for Treatment of Diabetic Nephropathy
The study is done to find out whether the combined use of the nutritional supplements N-acetylcysteine and Siliphos (milk thistle extract) corrects the shedding of urine protein and oxidative damage (damage to cells and organs…
- Conditions
- Diabetic Nephropathy, Proteinuria, Oxidative Stress
- Trial name
- CGDN (NCT01265563)
- Testing
- N-acetylcysteine active and silibin active, N-acetylcysteine active + high-dose silibin active
- Where
- United States
- Volunteers
- 108 people
- Sponsor
- VA Office of Research and Development
Systemic Juvenile Idiopathic Arthritis, Hereditary Periodic Fevers
A Study of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 Studies
The objective of this extension protocol is to collect safety data (serious and non-serious adverse events) and to provide continuous canakinumab to patients in France who completed study CACZ885G2301E1(NCT00891046), CACZ885G2306…
- Trial name
- NCT02334748
- Testing
- canakinumab
- Where
- 3 sites in France
- Volunteers
- 31 people
- Sponsor
- Novartis Pharmaceuticals
Antithrombin III Deficiency
Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate
To assess the safety, pharmacokinetics and efficacy of a plasma-derived AT-III concentrate in the treatment of subjects with congenital AT-III deficiency.
- Trial name
- NCT00319228
- Testing
- Plasma-derived AT-III concentrate
- Volunteers
- 3 people
- Sponsor
- Instituto Grifols, S.A.