Clinical trials: Protein Deficiency
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
Hypofibrinogenemia, Congenital, Afibrinogenemia, Congenital
Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
- Trial name
- NCT02094430
- Testing
- biological: human fibrinogen concentrate
- Where
- 4 sites in France, Lebanon and 2 more
- Volunteers
- 16 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
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
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease
- Trial name
- NCT00290628
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Protein-Energy Malnutrition
Usefulness of Protein-enriched Infant Formula in Pediatric Intensive Care.
A multicenter prospective randomized controlled trial (RCT) will be performed in three hospitals. Patients meeting inclusion criteria will be randomly allocated to one of three enteral feeding formula with different protein…
- Trial name
- NCT03901742
- Testing
- Protein-enriched nutrition, High Protein-enriched Nutrition, Standard Enteral Nutrition
- Where
- 3 sites in Spain
- Volunteers
- 90 people
- Sponsor
- Hospital General Universitario Gregorio Marañon
Leukemia, Lymphoma
Blood Samples to Identify Biomarkers of Busulfan
Specific Aim 1: To determine whether endogenous metabolomics-based biomarkers obtained before IV BU administration can predict IV BU clearance. Specific Aim 2: To characterize IV BU metabolism by metabolomics.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Immunodeficiency
- Trial name
- NCT02291965
- Where
- United States
- Volunteers
- 139 people
- Sponsor
- Fred Hutchinson Cancer Center
Antithrombin III Deficiency
Belgian Antithrombin Deficiency Registry
Inherited antithrombin deficiency is a rare autosomal dominant disorder that predisposes to the development of venous thromboembolism, even at young age.
- Trial name
- NCT05891899
- Testing
- observation
- Where
- Belgium
- Volunteers
- 1,000 people
- Sponsor
- Universitair Ziekenhuis Brussel
CD55-deficient Protein-losing Enteropathy
Compassionate Use of Pozelimab
Provide compassionate use of pozelimab
- Trial name
- NCT05232110
- Testing
- Pozelimab
- Sponsor
- Regeneron Pharmaceuticals
Severe Acute Malnutrition, Kwashiorkor
Dietary Rehabilitation in Severely Acutely Malnourished Children
Severe acute malnutrition (SAM) is a life threatening condition and is defined by 1) a weight-for-height Z-score more than three standard deviations (SD) below the median based on the 2006 World Health Organization (WHO) growth…
- Conditions
- Severe Acute Malnutrition, Kwashiorkor, Nutritional Edema, Marasmus
- Trial name
- NCT05009823
- Testing
- Standard F75, Alternative F75 With CMV, Alternative F75 Without CMV
- Where
- Burkina Faso
- Volunteers
- 1,959 people
- Sponsor
- University Ghent
Acute Leukemia, Immune Deficiency Disorder
Umbilical Cord Blood Transplantation From Unrelated Donors
This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often engraftment of umbilical cord stem cells transplanted from an unrelated…
- Conditions
- Acute Leukemia, Immune Deficiency Disorder, Congenital Hematological Disorder, Metabolism Disorder
- Trial name
- NCT03016806
- Testing
- Total Body Irradiation 1200 cGy, Total Body Irradiation 200 cGy, Cyclophosphamide
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Rochester
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease
Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Inborn errors of metabolism (IEM) are not have specific clinical signs, they masquerade as other diseases, and are difficult to diagnose using only clinical manifestations or routine laboratory tests.
- Conditions
- Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease, Citrullinemia, Argininosuccinic Aciduria
- Trial name
- NCT05910151
- Testing
- Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years
- Where
- Kazakhstan
- Volunteers
- 2,250 people, healthy volunteers welcome
- Sponsor
- West Kazakhstan Medical University
Ataxia Telangiectasia, ATM Gene Mutation
Effects of Vitamin B3 in Patients With Ataxia Telangiectasia
This clinical trial investigates the effects of nicotinamide riboside (vitamin B3) on the disease course of patients with ataxia telangiectasia.
- Trial name
- NCT03962114
- Testing
- Vitamin B3
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Radboud University Medical Center
Phenylketonurias, Hyperphenylalaninaemia
Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of phenylalanine-free protein substitute tablets for young children with PKU aged of 7 years or older.
- Conditions
- Phenylketonurias, Hyperphenylalaninaemia, Tetrahydrobiopterin Deficiency
- Trial name
- NCT04375592
- Testing
- Phenylalanine-free protein substitute in tablet form (XPhe minis)
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- metaX Institut fuer Diatetik GmbH
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency, Glanzmann's Thrombasthenia
- Trial name
- NCT03372993
- Where
- 2 sites in Iran
- Volunteers
- 200 people
- Sponsor
- AryoGen Pharmed Co.
Fibrinogen Deficiency
Human Fibrinogen - Pharmacokinetics
This study evaluated the single-dose pharmacokinetics of human fibrinogen concentrate and clot strength (maximum clot firmness [MCF]) in subjects with congenital fibrinogen deficiency.
- Trial name
- NCT00496262
- Testing
- Human Fibrinogen Concentrate
- Where
- 15 sites in United States, Italy
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Glycogen Storage Disease Type II
High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease
The research protocol will be submitted for approval to the institutional review board of Columbia University Medical Center. An attempt will be made to recruit at least 6 juvenile patients between the ages of 8 and 17…
- Trial name
- NCT01656590
- Testing
- High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
- Where
- United States
- Sponsor
- Columbia University
Von Willebrand Disease
Willebrand International Non-interventional Global Surveillance
Collect information about WILLFACT or WILFACTIN in their real life clinical use and identify the therapeutic practices in an international environment.
- Trial name
- NCT01949220
- Where
- 18 sites in Belgium, Czechia and 5 more
- Volunteers
- 80 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
HIV Infections, HIV Seronegativity
A Phase I Study to Evaluate the Safety and Immunogenicity of Recombinant HIV-1 Envelope Antigen in Children Born to HIV-Infected Mothers
PRIMARY: To determine the safety of envelope recombinant proteins rgp120/HIV-1MN (Genentech) and rgp120/HIV-1SF2 (Chiron/Biocine) in infants who are of indeterminate HIV status born to HIV-infected women.
- Trial name
- NCT00000774
- Testing
- rgp120/HIV-1MN, rgp120/HIV-1 SF-2
- Where
- 37 sites in United States, Puerto Rico
- Volunteers
- 156 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage
HEMOLEVEN® Expanded Access Program Prevention of Surgical/Postpartum Hemorrhage Severe Inherited Factor XI Deficiency
The objective of the Expanded Access Program is to provide HEMOLEVEN, a replacement coagulation factor XI, to patients with severe inherited factor XI deficiency where, in the opinion of the treating physician, the benefits of…
- Conditions
- Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage, Surgery
- Trial name
- EAP (NCT01701947)
- Testing
- Hemoleven
- Where
- United States
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies