Clinical trials: Protein Deficiency
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
This clinical trial studies the side effects and best dose of giving fludarabine and total-body irradiation (TBI) together followed by a donor stem cell transplant and cyclosporine and mycophenolate mofetil in treating human…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00112593
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Fred Hutchinson Cancer Center
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
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
Anemia, Iron-deficiency
Improvement of Fatigue Symptoms in the Iron Deficiency Anemia With Iron Succinylate Therapy
The treatment of anemia depends on its cause. Patients with underlying iron-deficient anemia should be treated or referred to a specialist (eg gynecologist, gastroenterologist) for treatment.deficiency anemia are global health…
- Conditions
- Anemia, Iron-deficiency, Iron Deficiency Anemia, Fatigue
- Trial name
- IFAISTOS (NCT03873584)
- Testing
- Fatigue
- Sponsor
- Elpen Pharmaceutical Co. Inc.
HIV Infections
Extended Follow-Up of the ISS T-003 Trial Volunteers (ISS T-003 EF-UP2020)
A further extension of the follow-up of the volunteers of the ISS T-003 trial is being conducted in order to evaluate the persistence of vaccine immunogenicity as well as of the immunological and virological effects induced by…
- Trial name
- T003EFUP2020 (NCT05680948)
- Where
- South Africa
- Volunteers
- 179 people
- Sponsor
- Istituto Superiore di Sanità
CDG Ib
Study of ORL-1M (D-mannose) in Patients With CDG-Ib
Study of ORL-1M in Patients With CDG-Ib
- Trial name
- NCT03404869
- Testing
- ORL-1M - D-mannose
- Where
- Turkey (Türkiye)
- Volunteers
- 5 people
- Sponsor
- Orpha Labs
ESRD (End-Stage Renal Disease), Dialysis
The Effects of a Dialysis-Specific Formula Rich in Branched-Chain Amino Acids, Omega-3, and Dietary Fiber on Nutritional Status
The aim of this study is to evaluate the effects of a specialized oral nutritional supplement (SF) enriched with BCAAs, omega-3 fatty acids, and dietary fiber on improving the nutritional status of dialysis patients with mild to…
- Trial name
- NCT07060040
- Testing
- BOSCOGEN 18% Protein ONS: Energy 480 kcal, Protein 21.6 g, BCAA 6000 mg, Omega-3 fatty acid 1.8 g, fiber 5 g, Fresubin dialysis ONS (200 ml): Energy 300 kcal, Protein 20 g, BCAA - mg, Omega-3 fatty acid - g, fiber 0.2 g
- Volunteers
- 100 people
- Sponsor
- Buddhist Tzu Chi General Hospital
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
HIV Infections, Hemophilia A
Safety and Effectiveness of Azidothymidine (AZT) in HIV-Positive Patients With Hemophilia
The purpose of this study is to see if giving azidothymidine (AZT) to HIV-positive patients with hemophilia is safe and if it is effective in lowering HIV levels and boosting the immune system.
- Trial name
- NCT00000705
- Testing
- Zidovudine
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3
Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.
The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome.
- Trial name
- THERIPEX (NCT07697118)
- Testing
- FOXP3-T4 drug product, ILT-101
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Inherited Bleeding Disorders
Study to Evaluate the Use of Ultrasound in the Diagnosis of Hemophilic Joint Disease
The purpose of this study is to see whether power Doppler sonography (PDS) can be used to diagnose synovitis in patients with inherited bleeding disorders.
- Trial name
- NCT00212485
- Testing
- Power Doppler Sonography, MRI
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- New York Presbyterian Hospital
Diagnosis of GHD With IGF-I/IGFBP-3 Ratio
Growth Hormone (GH) Deficiency & Insulin Like Growth Factor 1 (IGF-I)/ Insulin Like Growth Factor Binding Protein 3 (IGFBP-3) Ratio
A 35-month cross-sectional observational study was conducted at an outpatient university hospital in Amman, Jordan. We obtained a comprehensive medical and laboratory evaluation for 134 short-statured children (64 boys and 70…
- Trial name
- NCT02424240
- Testing
- IGF-1/IGFBP-3 ratio
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- University of Jordan
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
Infection, Immune Deficiency
Personalized Immunotherapeutic for Antibiotic-resistant Infection
M. A. suffers from hypogammaglobulinemia that has been complicated by refractory Mycoplasma hominis septic arthritis. He has been receiving the antibiotic valnemulin under Emergency Investigational New Drug (eIND) 114686…
- Conditions
- Infection, Immune Deficiency, Hypogammaglobulinemia, Septic Arthritis
- Trial name
- NCT02508584
- Testing
- anti-mycoplasma hominis antibodies
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Brigham and Women's Hospital
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
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
Common Variable Immunodeficiency, Agammaglobulinemia
Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin
Objective: Measure serum IgG antibody to Streptococcus pneumoniae serotypes 1, 3, 5, 6B, 9V e 14, Haemophilus influenzae type b and tetanus toxoid in patients with primary antibody deficiencies who were treated with subcutaneous…
- Trial name
- NCT00661401
- Testing
- gammaglobulin
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- Federal University of São Paulo
Severe Acute Malnutrition, Kwashiorkor
Effectiveness of Alternative Diets During the Stabilization Phase on Children With Complicated SAM
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
- NCT05020847
- Testing
- Standard F75, Alternative F75 with CMV, Alternative F75 without CMV
- Where
- Burkina Faso
- Volunteers
- 297 people
- Sponsor
- University Ghent
Lynch Syndrome
Diagnosis of Lynch Syndrome Based on Next-generation Sequencing in Colorectal Cancer
The purpose of this study is to determine the proportion of patients diagnosed with Lynch syndrome in colorectal cancer patients with the loss of staining by immunohistochemistry (IHC) of any of the mismatch repair (MMR)…
- Trial name
- NCT03047226
- Testing
- next-generation sequencing
- Where
- 7 sites in China
- Volunteers
- 311 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Hemophilia A Patient
Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly…
- Trial name
- E-PROTECT (NCT07830693)
- Testing
- Study questionnaires, Clinical/pharmacological data collection
- Where
- 8 sites in France
- Volunteers
- 553 people
- Sponsor
- Nantes University Hospital