E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
All conditions in which a T cell immunodeficiency is present. Examples are genetic immunodeficiencies, chronic viral infections like HIV, after chemotherapy or bone marrow transplantation |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10021463 |
E.1.2 | Term | Immunodeficiency with predominant T-cell defect, unspecified |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study will be to learn if TSH is able to induce the generation of new T cells by thymopoiesis. |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives of this study will be to: • Evaluate involvement of TSH in lipid metabolism • Evaluate involvement of TSH in bone metabolism • Evaluate involvement of TSH in CK levels Evaluate involvement of TSH in metabolic pathways (metabolomics)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients must meet the following criteria to be included: Have the capacity to understand and willing to sign an informed consent form Have been medically treated for primary hypothyroidism for the last 6 months with only adequate thyroxin substitution therapy Have medically controlled disease T3 and T4 blood levels within the normal range for the past 6 months TSH within the normal range for the past 6 months TSH>20 mU/l at diagnosis Presence of anti TPO antibodies Age 20-45 years
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E.4 | Principal exclusion criteria |
Patients will be excluded from the study meeting the following criteria: Uncontrolled hypothyroidism Presence of antibodies to the TSH receptor History of M. Graves or thyroiditis Presence of struma Enlarged tyroid gland measured with ultrasound Serious infections in the last 3 months Have current symptoms of cardiac disease Have current signs or symptoms of severe, progressive or uncontrolled renal, hepatic, hematologic, gastrointestinal, endocrine, pulmonary, cardiac, neurologic, or cerebral disease. Clinically relevant abnormal findings during routine physical examination, screening blood samples of hematology, biochemistry, urinanalysis and/or known ECG abnormalities. Alcohol abuse Known hematologic malignancy Known thyroid malignancy Other autoimmune disorders than hypothyroidism Thymectomy in the medical history T cell affecting co-medication Pregnancy
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint consist of a change in thymic output, peripheral cell numbers or ratio’s of peripheral T cell subpopulations in response to treatment with rhTSH. T cell subpopulations will be defined using flow cytometry. Moreover thymic output will be measured using TREC analysis. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |