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 | PT |
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 objective of this study is to assess the safety and efficacy of prGCD in patients with Gaucher disease currently being treated with Imiglucerase (Cerezyme®) enzyme replacement therapy (ERT). |
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E.2.2 | Secondary objectives of the trial |
there are no secondary objectives |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Males and females, 18 years or older 2. Female patients of child-bearing potential and male patients with female partners of child-bearing potential must agree to use two medically acceptable methods of contraception, one of which must be a barrier method, e.g. oral contraceptive and condom, intra-uterine device (IUD) and condom, diaphragm with spermicidal and condom for at least 42 days after the treatment has finished. 3. Diagnosis of Gaucher disease with leukocyte GCD activity level ≤3 nmol/mg*hr (≤30 % of the mean activity of the reference range) 4.Stable Gaucher disease, defined as: a. Hemoglobin during Stability Evaluation Period is stable with no value more than 15% below or above the mean value b. Platelets count during Stability Evaluation Period is stable with no values more than 40% below or above the mean value if the mean value is >120,000, or more than 20% below or above the mean value if the mean value is ≤ 120,000 c. No major surgery in the last year d. No blood transfusion or major bleeding episode in the last year e. No acute avascular necrosis event in the last year f. No evidence of spleen or liver increasing enlargement while being treated with enzyme replacement therapy by palpation, ultrasound, or MRI over the last year 5. Receiving imiglucerase therapy for at least 2 years and on a stable maintenance regimen (dose and regimen unchanged, except for situation of drug shortage) for at least last six months 6. Able to provide written informed consent
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E.4 | Principal exclusion criteria |
1. Currently taking another experimental drug for any condition 2. Pregnant or nursing or planning to become pregnant 3. History of allergy to carrots 4. Presence of anti-glucocerebrosidase (GCD) antibodies 5. Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions 6. Presence of HIV and/or HBsAg and/or hepatitis C infection 7. Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency 8. Presence of any significant comorbidity that could confound the interpretation of the clinical response to prGCD 9. Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient’s compliance with the requirements of the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Adverse events 2. Clinical laboratory (hematology, biochemistry, urinalysis) 3. Anti human prGCD antibodies 4. Electrocardiogram 5. Echocardiogram 6. Pulmonary function test 7. Spleen volume 8. Liver volume 9.Platelet count 10. Hemoglobin 11. Biomarkers [chitotriosidase and pulmonary and activation-regulated chemokine (PARC/CCL18)]
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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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | 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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
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 | Yes |
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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the end of the trial is 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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |