Clinical trials: Protein Deficiency
Hypophosphatasia
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042).
- Trial name
- NCT01205152
- Testing
- asfotase alfa
- Where
- 8 sites in United States, United Arab Emirates and 1 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pregnancy, Protein-Energy Malnutrition
NutFish and Nutrient Supplementation in Pregnancy Class to Improve Maternal and Birth Outcomes
This study evaluates the intervention of Nut Fish based food, micro nutrients supplementation and pregnancy class to improve maternal and birth outcome. This is cluster randomized trial with two arms.
- Conditions
- Pregnancy, Protein-Energy Malnutrition, Anemia
- Trial name
- NCT02959125
- Testing
- NutFish Based Food Supplementation, Multiple micronutrients, Health Education in Pregnancy Class
- Where
- 2 sites in Indonesia
- Volunteers
- 72 people
- Sponsor
- Semarang Health Polytechnic
Hemophilia A, Hemophilia B
Correlation Between Vitamin D Status and Bone Mineral Density in Patients With Hemophilia
Study will look at baseline Vitamin D levels, calcium, albumin, liver enzymes, testosterone, osteocalcin, urine N telopeptides, bone mineral density, nutritional assessment and physical activity assessment of boys with hemophilia…
- Conditions
- Hemophilia A, Hemophilia B, Vitamin D Deficiency
- Trial name
- NCT01090206
- Testing
- Vitamin D and calcium
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Virginia Commonwealth University
XLA, CVID
Immune Regulation in Patients With Common Variable Immunodeficiency and Related Inborn Errors of Immunity (IEI)
This study aims to understand the causes and progression of Common Variable Immunodeficiency (CVID) and related inborn errors of immunity (IEI).
- Conditions
- XLA, CVID, Yao Syndrome, Blau Syndrome
- Trial name
- NCT00001244
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Acute Malnutrition, Persistent Diarrhea
Trial of Rice-Based F-75 in Severe Malnutrition & Persistent Diarrhea in Children (SAM-RICE Study)
The goal of this study is to find out if a rice-based version of the F-75 therapeutic food helps children with severe acute malnutrition (SAM) and persistent diarrhea recover better than the standard commercial F-75.
- Conditions
- Severe Acute Malnutrition, Persistent Diarrhea, Children
- Trial name
- SAM-RICE (NCT07070856)
- Testing
- WHO-recommended standard therapeutic milk used for stabilization phase in SAM), HO-recommended Rice based therapeutic milk used for stabilization phase in SAM)
- Where
- 2 sites in Pakistan
- Volunteers
- 320 people
- Sponsor
- Children's Hospital and Institute of Child Health, Multan
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
RDS of Prematurity, Surfactant Protein B Deficiency
Administration of Surfactant Through an Instillation Device Infasurf® (Calfactant) in Neonates- A Pilot Study
A pilot study to explore and determine feasibility and safety of the administration of surfactant through a new supraglottic device in patients from 500 to 5000 grams with RDS.
- Conditions
- RDS of Prematurity, Surfactant Protein B Deficiency, Chronic Lung Disease
- Trial name
- NCT04056741
- Testing
- surfactant airway device
- Where
- 4 sites in United States
- Volunteers
- 55 people
- Sponsor
- ONY
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency
Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy.
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- Triheptanoin (NCT01379625)
- Testing
- Triheptanoin
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- Oregon Health and Science University
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)
Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement.
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT01461304
- Testing
- triheptanoin
- Where
- United States
- Sponsor
- Jerry Vockley, MD, PhD
Factor VIII Deficiency, Congenital
Hemlibra in Mild Hemophilia A
This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild…
- Trial name
- NCT04567511
- Testing
- Emicizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Indiana Hemophilia &Thrombosis Center, Inc.
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in…
- Conditions
- Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency
- Trial name
- NCT02214160
- Testing
- UX007
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 94 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
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
Hemophilia A
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation…
- Trial name
- NCT00157053
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 24 sites in United States, Austria and 7 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively…
- Trial name
- JOSC (NCT01000844)
- Volunteers
- 11 people
- Sponsor
- University of Colorado, Denver
Malnutrition (Calorie), Protein-energy Malnutrition
Composite Flour Cookies for Improving Nutrition in Malnourished Children
Malnutrition is a major public health concern among children in developing countries, leading to impaired growth and increased risk of morbidity.
- Trial name
- CFC-MAL (NCT07504133)
- Testing
- Composite Flour Cookies
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- Mairaj Noor
HIV Infections
A Study of Zidovudine in HIV-Infected Patients Who Have Hemophilia
Study A: To determine whether treatment with zidovudine (ZDV) will delay or change the disease process in hemophilic patients who have HIV infection with no symptoms.
- Trial name
- NCT00001104
- Testing
- Zidovudine
- Where
- 23 sites in United States
- Volunteers
- 538 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency
High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT01494051
- Testing
- Diet counseling
- Volunteers
- 13 people
- Sponsor
- Oregon Health and Science University
HIV Infections, Pregnancy
Active Immunization of HIV-1 Infected, Pregnant Women With CD4 Lymphocyte Counts >= 400/mm3: A Phase I Study of Safety and Immunogenicity of MN rgp120/HIV-1 Vaccine (NOTE: Some Patients Receive Placebo)
To evaluate the safety of rgp120/HIV-1MN vaccine in HIV-1 infected pregnant women with CD4 counts >= 400 cells/mm3. To evaluate the immunogenicity of this vaccine in pregnant women and the passive acquisition of vaccine-specific…
- Trial name
- NCT00001041
- Testing
- rgp120/HIV-1MN
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)