Clinical trials: Protein Deficiency
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
HIV Infections
A Phase I Study of the Safety and Pharmacokinetics of Recombinant CD4 (rCD4) in Infants and Children Infected With or at Risk for HIV Infection
AMENDED: As of 10/19/90 only Children 0 to 3 months are being enrolled. Original design: To determine whether the experimental drug recombinant CD4 (rCD4), which is produced through genetic engineering technology, is safe and…
- Trial name
- NCT00000984
- Testing
- CD4 Antigens
- Where
- 6 sites in United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
CD55-deficient Protein-losing Enteropathy
Compassionate Use of Pozelimab
Provide compassionate use of pozelimab
- Trial name
- NCT05232110
- Testing
- Pozelimab
- Sponsor
- Regeneron Pharmaceuticals
Mevalonate Kinase Deficiency
Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency
Mevalonate kinase deficiency (HyperIgD syndrome, HIDS) is an inborn error of immunity caused by a block in the mevalonate pathway.
- Trial name
- NCT06497829
- Testing
- geranylgeraniol
- Where
- Czechia
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University Hospital, Motol
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
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
Factor XIII Deficiency
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00945906
- Testing
- FXIII Concentrate (Human) (FXIII)
- Where
- 25 sites in United States
- Volunteers
- 61 people
- Sponsor
- CSL Behring
Marasmus, Kwashiorkor
Mothers Understand And Can do it - Mid Upper Arm Circumference Screening for Malnutrition Performed by Mothers
PRIMARY OBJECTIVE : To determine whether mothers, given minimal group training, are capable of using a MUAC (mid-upper arm circumference) bracelet to screen their children for malnutrition and categorise them into one of three…
- Conditions
- Marasmus, Kwashiorkor, Malnutrition; Degree, Moderate, Malnutrition
- Trial name
- MUAC (NCT01790815)
- Where
- Niger
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Alliance for International Medical Action
Vitamin D Deficiency
Effects of Vitamin D Dose and Genotype of the Binding Protein in Infants and Children
The purpose of this study is to determine if the vitamin D binding protein genotype influences circulating vitamin D levels and if it may have functional consequences on vitamin D activity.
- Trial name
- VitaD (NCT01050387)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 193 people, healthy volunteers welcome
- Sponsor
- Yale University
Lymphoma, Non-Hodgkin Lymphoma
AZD3470 as Monotherapy or in Combination With Anticancer Agent(s) in Participants With Haematologic Malignancies.
This study is designed to evaluate the safety, tolerability, PK, pharmacodynamics, and preliminary efficacy following oral administration of AZD3470 as a monotherapy, and in combination with other anticancer agents in…
- Conditions
- Lymphoma, Non-Hodgkin Lymphoma, Hodgkin Lymphoma, Peripheral T-cell Lymphoma (PTCL)
- Trial name
- PRIMAVERA (NCT06137144)
- Testing
- AZD3470, Pembrolizumab
- Where
- 38 sites in United States, Australia and 9 more
- Volunteers
- 161 people
- Sponsor
- AstraZeneca
X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
OBJECTIVES: I. Identify the molecular defects responsible for primary immunodeficiency disorders. II. Explore the mutations within each syndrome to better understand the genetics of these disorders. III.
- Conditions
- X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Deficiency Syndrome
- Trial name
- NCT00004341
- Where
- United States
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
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
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Factor VIII Deficiency
A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture
The purpose of this research study is to see if factor levels and inhibitor levels in Hemophilia A and B subjects are accurate when they are drawn from a central venous line (CVL) instead of from a peripheral stick.
- Conditions
- Hemophilia A, Factor VIII Deficiency, Hemophilia, Hemophilia B
- Trial name
- CVL#2 (NCT02402829)
- Testing
- Peripheral Vein Blood draw
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Mercy Hospital Kansas City
Immune System Diseases, Premature Infant Disease
Oral Colostrum and Its Effect on Immune System
The purpose of this project is to increase the serum immunological defenses of premature infants less then 32 weeks of gestation by administrating colostrum in the oropharyngeal mucosa versus placebo
- Conditions
- Immune System Diseases, Premature Infant Disease, Immunoglobulin Deficiency
- Trial name
- NCT03578341
- Testing
- Orally Colostrum
- Where
- 2 sites in Mexico
- Volunteers
- 96 people
- Sponsor
- Instituto Nacional de Perinatologia Isidro Espinosa de los Reyes
Congenital Protein C Deficiency
A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
Pharmacokinetic Part: This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time.
- Trial name
- NCT04984889
- Testing
- TAK-662
- Where
- 4 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda
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