Clinical trials: Protein Deficiency
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
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Vitamin D Deficiency
Vitamin D in Armenia: Vitamin D Repletion Strategies With Dibase, and Vitamin D and Vitamin D Binding Protein and COVID
This is a three-arm unblinded prospective interventional study of vitamin D supplementation in participants with insufficient vitamin D at three different induction and maintenance dosing regimens (high dose, low frequency…
- Trial name
- NCT06406543
- Testing
- Vitamin D3 (Cholecalciferol)
- Where
- Armenia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Columbia University
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
Non-Small Cell Lung Cancer
Ph1/2 Trial Of Navlimetostat With Pumitamig In MTAP-Deficient Advanced Non-Small Cell Lung Cancer
The goal of this clinical research study is to find a safe and tolerable dose of navlimetostat in combination with pumitamig that can be given to patients with MTAP-deficient advanced non-small cell lung cancer (NSCLC).
- Trial name
- NCT07579221
- Testing
- Navlimetostat, Pumitamig
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- M.D. Anderson Cancer Center
Body Weight, Appetitive Behavior
High Fat vs High Protein and Appetite Hormones
This study will investigate the effects of high-protein, high-fat snacks, specifically Greek yogurt, and peanuts, on satiety, gut hormones, and insulin secretion in overweight and obese women.
- Conditions
- Body Weight, Appetitive Behavior, Dietary Exposure, Hormone Deficiency
- Trial name
- NCT04518930
- Testing
- Peanuts or plain Greek yogurt
- Where
- Jordan
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Al-Balqa Applied University
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168012
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 42 people
- Sponsor
- CSL Behring
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
Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma
A Clinical Trial to Evaluate Efficacy, Safety, and Pharmacokinetics of Gamunex in Participants With Secondary Antibody Deficiency Associated With Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma
The main goal of this study is to show that people with certain immune problems (from Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma) get fewer serious infections when they receive Gamunex C through an IV…
- Trial name
- SIGMA (NCT07582432)
- Testing
- Gamunex-C, 10% Injectable Solution
- Where
- 5 sites in United States
- Volunteers
- 49 people
- Sponsor
- Grifols Biologicals, LLC
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Adenocarcinoma - GEJ, Cancer of Esophagus
Temozolomide + Nivolumab in MGMT Methylated Oesophagogastric Cancer
An open label single arm phase II trial in patients with advanced unresectable previously treated oesophagogastric adenocarcinoma which is MGMT deficient.
- Trial name
- ELEVATE (NCT04984733)
- Testing
- Temozolomide, Temozolomide 50mg/m2/day, Temozolomide 3 month
- Where
- United Kingdom
- Volunteers
- 13 people
- Sponsor
- University of Southampton
HIV Infections
Effectiveness of Giving an HIV Vaccine (Remune) to HIV-Positive Patients Receiving an Anti-HIV Drug Combination
The purpose of this study is to look at the effects of the HIV vaccine Remune on viral load (level of HIV in the blood) and on the way the immune system responds to HIV.
- Trial name
- NCT00005758
- Testing
- ALVAC(2)120(B,MN)GNP (vCP1452), HIV-1 Immunogen
- Where
- 17 sites in United States
- Volunteers
- 472 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Haemophilia A
Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens
This trial is designed to assess if there is evidence of subclinical joint bleeding on MRI/X-Ray in adults with severe Haemophilia A while on standard and/or pharmacokinetically tailored prophylaxis regimens.
- Trial name
- PERSONAL (NCT02314325)
- Testing
- ADVATE [Antihemophilic Factor (Recombinant)]
- Where
- Ireland
- Volunteers
- 20 people
- Sponsor
- St. James's Hospital, Ireland
HIV Infections
Safety of and Immune Response to Polyvalent HIV-1 Vaccine in HIV Uninfected Adults
This study will evaluate the safety of and immune response to a new HIV vaccine. The vaccine in this trial uses pieces of HIV DNA and HIV proteins. The vaccine itself cannot cause HIV infection or AIDS.
- Trial name
- NCT00061243
- Testing
- HIV-1 DNA vaccine with protein vaccine boost
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Factor XIII Deficiency
A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding.
- Trial name
- NCT00883090
- Testing
- FXIII Concentrate (Human)
- Where
- 6 sites in United States, Spain
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Nutrition Poor, Protein-Energy Malnutrition
Three Channel Food Concept: the Effect Meal Service on Food Intake During Hospitalization
The goal of this observational study is to assess food intake in hospitalized patients. The present study will evaluate the impact of a novel in-hospital meal concept (three channel food concept) on total energy and protein…
- Trial name
- NCT06399250
- Testing
- meal service
- Where
- Netherlands
- Volunteers
- 102 people
- Sponsor
- Maastricht University Medical Center
Lung Diseases, Respiratory Distress Syndrome, Newborn
Epidemiology of Surfactant Protein-B Deficiency
The purpose of this study is to test the hypothesis that excess, rare, functionally disruptive single nucleotide polymorphisms (SNPs) characterize genes (e.g., the surfactant protein-B gene)(SFTPB) and gene networks (e.g., the…
- Conditions
- Lung Diseases, Respiratory Distress Syndrome, Newborn, Pulmonary Surfactant, Lung Diseases, Interstitial
- Trial name
- NCT00014859
- Where
- United States
- Volunteers
- 5,176 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Hemophilia A, Hemophilia
Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
- Conditions
- Hemophilia A, Hemophilia, Factor VIII Deficiency
- Trial name
- NCT03006965
- Testing
- octocog alfa, rurioctocog alfa pegol
- Where
- 14 sites in Spain
- Volunteers
- 50 people
- Sponsor
- Spanish Society of Thrombosis and Haemostasis