E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | 10018048 |
E.1.2 | Term | Gaucher's disease |
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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 is to evaluate the long-term safety of GA-GCB when administered every other week (EOW) intravenously in patients with type 1 gaucher disease. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of this study are to evaluate the effects of GA-GCB administration on clinical parameters of Gaucher disease, including: -Hemoglobin concentration -Platelet count -Liver volume by MRI -Spleen volume by MRI (in patients who have not undergone splenectomy)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients must meet all of the following criteria to be considered eligible for enrollment: 1. The patient has completed through Week 53 of study TKT032 or TKT034, or through Week 41 of study HGT-GCB-039. 2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy tests will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner’s pregnancy to the investigator. 3. The patient, the patient’s parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC). 4. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
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E.4 | Principal exclusion criteria |
Patients who meet any of the following criteria are not eligible for this study: 1. The patient has received treatment with any investigational drug, other than GA-GCB, or device within the 30 days prior to study entry; such use during the study is not permitted. Treatment with imiglucerase, if provided as a part of participation in HGT-GCB-039, is allowed. 2. The patient is pregnant or lactating. 3. The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study. 4. The patient has a significant co-morbidity(ies) that might affect study data or confound the study results (e.g., malignancies, hepatic cirrhosis, autoimmune liver disease, significant infectious disease, etc.). 5. The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, is otherwise unlikely to complete the study, or continued treatment with GA-GCB is not in the best interest of the patient, as determined by the Investigator.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint of this study is safety as assessed by adverse events (including infusion-related adverse events), concomitant medication use, vital sign measurement, physical examinations, and clinical laboratory tests (hematology, serum chemistry, urinalysis, and anti-GA-GCB antibody formation). |
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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 | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | Yes |
E.8.1.3 | Single blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.3 |
The trial involves single site in the Member State concerned
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E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 3 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |