Thymus hypoplasia Thymic Hypoplasia
Conditions
Interventions
Patients will receive treatment with Biomodulin T via IM. The doses of one, two or three bulb per week will be differentiated according to the degree of Hypoplasia: Severe Thymic Hypoplasia (STH), Mod
Injections, Intramuscular
Sponsors
Center National of Bioproducts (BioCen)
Eligibility
Sex/Gender
All
Age
1 Years to 5 Years
Inclusion criteria
Inclusion criteria: 1. Patients with radiological confirmation by Thymus Ultrasound of Severe, Moderate or Light Thymic Hypoplasia with or without clinical manifestations of immunodeficiencies 2. Both sexes between the ages of one and five years. 3. That they are virgins of immunostimulating or immunosuppressive treatment, at least during the previous 6 months. 4. Voluntariness of the patient's parent or guardian and giving written informed consent.
Exclusion criteria
Exclusion criteria: 1. Patients with a history of severe allergic reactions and / or severe generalized eczema. 2. Patients with any other type of non-cellular immunodeficiency with exception of IgA partial deficiency. 3. Di George Syndrome. 4. Patients who have been treated with steroids in the previous 45 days. 5. Patients with a diagnosis of tumor or autoimmune disease of any kind. 6. Patients with chronic diseases of the type Diabetes Mellitus Type I or any other endocrinopathy. 7. Patients with severe congenital malformations that cause disability or require medical or surgical treatment. 8. Patients with hemoglobin lower than 9 g/L.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Thymic Area (Ultrasound measurement of thymus in mm2). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 2. Degree of thymic hypoplasia (Classification of degree of thymic hypoplasia according to scale in light, moderate or severe). Measurement time: At baseline, 16 weeks and 32 weeks if patient receives the third cycle. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Infections (Number of infections in the period). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 2. Nutritional Status (Body Mass Index-BMI). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 3. Lymphocyte Subpopulations (Percent of Lymphocyte Markers by Flow Cytometry- CD3+/CD4+, CD3+/CD8+, CD19+, CD56+). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle . 4. Quality of life (PedsQL Version 4.0 in Spanish Quality of Life questionnaire). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 5. Adverse events-AE (Occurrence of some AE (Yes, No), Description of the AE (Name of adverse event),According to available previous information (Unexpected, Expected), According to its localization the expected AE (World Allergy Organization Criterion: local and systemic AE (0= Nonspecific Symptoms, I= Mild systemic reactions, II= Moderate systemic reactions, III= Severe (nonlife-threatening) systemic reactions, IV= Anaphylactic shock)), Duration of the AE (Time between the start and the end of the AE), Graveness of the AE (Serious, No serious), Attitude regarding the treatment in study (Without changes, Dose modification, Temporary interruption, Definitive interruption), Result of the AE (Recovered, Improved, Persists, Sequels), Causality Relationship of the AE (1.Very likely, 2.Likely, 3.Possible, 4.Unlikely, 5.Not related, 6.Not evaluated)). Measurement time: weekly during the first month and, monthly until month 12. | — |
Countries
Cuba
Contacts
Public ContactOdalis de la Guardia Peña
Institute of Hematology and Immunology
Outcome results
None listed