Clinical trials: Protein Deficiency
Hypogammaglobulinemia, Congenital Heart Disease
Post-bypass Prophylactic IVIG in Infants and Neonates
The purpose of this study protocol is to determine if administering Intravenous Immunoglobulin (IVIG) for treatment of cardiopulmonary bypass (CPB) induced hypogammaglobulinemia in the early post-operative period can impact…
- Trial name
- NCT02043379
- Testing
- IVIG
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Alabama at Birmingham
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
This is a monocentric, no-profit, retrospective and prospective observational cohort study conducted at IRCCS Ospedale San Raffaele. The study does not entail any additional procedures or interventions for participants.
- Trial name
- HEAL-MD (NCT07745218)
- Where
- Italy
- Volunteers
- 25 people
- Sponsor
- IRCCS San Raffaele
Hemophilia, Embolization
Embolization in Hereditary Coagulopathies
This is a longitudinal, prospective study, which will include 30 subjects with hereditary coagulopathies, with arthropathy, chronic synovitis resulting from hemarthrosis of the elbows, knees and/or ankles followed up at the…
- Conditions
- Hemophilia, Embolization, Hemarthrosis, Clotting Factor Deficiency
- Trial name
- EHCO (NCT05629130)
- Testing
- embolization with spherical microparticles embosphere
- Where
- Brazil
- Volunteers
- 30 people
- Sponsor
- University of Sao Paulo General Hospital
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
- Trial name
- NCT05933200
- Testing
- Triheptanoin, MCT Oil
- Where
- 15 sites in Czechia, Germany and 5 more
- Volunteers
- 69 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Asthma
IVIG Treatment for Asthmatic Patients With IgG Subclass Deficiency
Immunoglobulin G deficiency subtypes in patients with asthma six months once a month intravenous immunoglobulin (400mg/kg/4weeks) infection in treatment-related effect on reducing the frequency of asthma exacerbations appreciate.
- Trial name
- IVIG (NCT01992328)
- Testing
- Immune Globulin
- Volunteers
- 30 people
- Sponsor
- Ajou University School of Medicine
Afibrinogenemia, Hypofibrinogenemia
Fibrinogen Concentrate (Human) - Efficacy and Safety Study
This is a multinational, multicenter, prospective, open-label historically controlled Phase IIIb non-inferiority clinical trial on the efficacy and safety of Fibrinogen Concentrate (Human).
- Conditions
- Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency
- Trial name
- NCT00916656
- Testing
- Fibrinogen Concentrate, Human (FCH), Cryoprecipitate
- Sponsor
- CSL Behring
Hemophilia A
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts.
- Trial name
- NCT00157040
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Growth Hormone Deficiency
304-C03/L3301n: Effects of Growth Hormone on Glucose and Protein Metabolism in Children With Growth Hormone Deficiency
The purpose of the proposed study is to investigate the effects of rhGH treatment on glucose, protein and fat metabolism in GHD children.
- Trial name
- NCT00362063
- Testing
- growth hormone (Nutropin)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
Primary Immune Deficiencies, Autoimmune Diseases
Investigation of Immune Disorders and Deficiencies
The immune system is an intricate system comprised of specialized cells, proteins, tissues and organs. Proper functioning is critical to the body's ability to defend itself against harmful pathogens.
- Conditions
- Primary Immune Deficiencies, Autoimmune Diseases, Inflammatory Diseases, Common Variable Immune Deficiencies
- Trial name
- NCT01981785
- Where
- United States
- Volunteers
- 343 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Bloom Syndrome
Biological Significance of the Bloom's Syndrome Protein
Since 1960, persons with the very rare disorder Bloom's syndrome (BS) have been followed clinically, documenting clinical matters as obtained from their doctors.
- Trial name
- NCT00021437
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
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
Nutritional Deficiency
Bioavailability of Zinc and Iron From a Whey-based Protein Supplement Consumed With a Habitual Plant-based Diet
Bioavailability of iron and zinc from habitual plant-based diets consumed by young children in Mexico is low due to the high phytate content.
- Trial name
- NCT02208622
- Testing
- Study 1: Whey Supplement Day 1, Study 1: Whey Supplement Day 2, Study 2: Whey Supplement
- Where
- 2 sites in United States, Mexico
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
HIV Infections, Hepatitis C
Treatment of Hepatitis C in Hemophilic Patients With HIV
Pegylated interferon (PEG-interferon) and ribavirin are accepted treatments for hepatitis C virus (HCV) infection. However, HCV infection progresses differently in patients who are coinfected with HIV and in hemophiliacs.
- Conditions
- HIV Infections, Hepatitis C, Hemophilia
- Trial name
- NCT00055341
- Testing
- Peginterferon alfa-2a, Ribavirin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immune Deficiency Disease
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
- Trial name
- NCT00634569
- Testing
- Flebogamma 5% DIF
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Instituto Grifols, S.A.
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
CD55 - Cluster of Differentiation Antigen 55 Deficiency, Primary Intestinal Lymphangiectasis
Transcriptome and Metabolic Analyses of CHAPLE Disease
CHAPLE syndrome (complement hyperactivation, angiopathic thrombosis, protein losing enteropathy) is a newly discovered genetic disorder, which is caused by deleterious mutations in the CD55 gene.
- Conditions
- CD55 - Cluster of Differentiation Antigen 55 Deficiency, Primary Intestinal Lymphangiectasis, Protein-Losing Enteropathies, Complement Regulatory Factor Defect
- Trial name
- CHAPLEOMIC (NCT03950804)
- Testing
- Eculizumab Injection
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Marmara University
HIV Infections, Hemophilia A
The Effects of AZT and Ibuprofen on HIV-Infected Patients With Hemophilia
To determine if platelet dysfunction and/or pharmacologic drug interaction occurs in patients taking both AZT and ibuprofen, which might account for enhanced bleeding tendency.
- Trial name
- NCT00002276
- Testing
- Ibuprofen, Zidovudine
- Where
- United States
- Sponsor
- University of Pittsburgh
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants.
- Trial name
- PROTECT-ALT (NCT06684314)
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Taiwan
- Volunteers
- 79 people
- Sponsor
- Sanofi