| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
|
| 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 |
|
| 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 |
To evaluate the safety of every other week dosing of GA-GCB in patients with type 1 Gaucher disease who were previously treated with
imiglucerase. |
|
| E.2.2 | Secondary objectives of the trial |
To evaluate changes from Baseline in hemoglobin concentration after every other
week dosing of GA-GCB
To evaluate changes from Baseline in platelet count after every other week dosing of GA-GCB
To evaluate changes from Baseline in liver and spleen volumes by MRI after every other week dosing of GA-GCB |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (GCB) activity in leukocytes or by genotype analysis.
The patient has received consistent treatment with imiglucerase for a minimum of 30 consecutive months. Consistent treatment is defined as a prescribed dosing interval of every other week at a dose ≤ 60 U/kg and ≥ 15 U/kg. Patients must have received the same dose during the 6 months prior to study enrollment. Minor dosing interval variance is allowed as reflected in standard clinical practice.
The patient is at least 2 years of age.
Female patients of child bearing potential must agree to use a medically acceptable
method of contraception at all times during the study and must have negative results
to a pregnancy test performed at Screening and as required throughout their
participation in the study. Male patients must use a medically acceptable method of birth control throughout their participation in the study and must report their partner’s pregnancy.
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).
The patient must be sufficiently cooperative to participate in this clinical study as
judged by the Investigator. |
|
| E.4 | Principal exclusion criteria |
The patient has both a hemoglobin concentration ≤ 10 g/dL and a platelet count ≤ 80,000 platelets/cu mm.
The patient has unstable hemoglobin concentration during the six months prior to Screening. Stable hemoglobin concentration is defined as the average of all values in the past six months falling within a range of + 1 g/dL of the Screening value. If at least three consecutive values, including Screening, in the previous 6 months are not available, the three most recent values will be considered. At least one of the values must be six months prior to Screening. In addition, the difference between the highest and lowest values cannot exceed 1.5 g/dL. Values within the first 12 months of treatment initiation with ERT will be excluded.
The patient has unstable platelet count during the six months prior to Screening. Stable platelet count is defined as the average of all values in the past six months falling within a range of + 20 % of the Screening value. If at least three consecutive values, including Screening, in the previous 6 months are not available, the three most recent values will be considered. At least one of the values must be six months prior to Screening. In addition, the difference between the highest and lowest values cannot exceed 20% of the highest value. Values within the first 12 months of treatment initiation with ERT will be excluded.
The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease.
The patient has received treatment with any investigational drug or device within the
30 days prior to study entry; such use during the study is not permitted.
The patient is known to be positive for human immunodeficiency virus (HIV), i.e.,
has a documented positive result. Patients who do not have a documented positive
result will be tested for HIV during Screening.
The patient is known to be positive for hepatitis B or hepatitis C, i.e., has a documented
positive result. Patients who do not have a documented positive result will be tested
for hepatitis during Screening.
The patient presents with sustained iron, folic acid or vitamin B12 deficiencyrelated anemia during Screening (i.e., non-Gaucher disease-related anemia).
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.
The patient has a significant comorbidity that might affect study data or confound the
study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease,
etc.).
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, or is otherwise
unlikely to complete the study, as determined by the Investigator.
The patient has had inconsistent treatment with imiglucerase in the last 6 months.
The patient has experienced an anaphylactic or anaphylactoid reaction during treatment with
imiglucerase.
The patient has received miglustat during the 6 months prior to study enrollment.
The patient is currently receiving red blood cell growth factor, (e.g., erythropoietin) or chronic systemic corticosteroids in
the last 6 months. (NOTE: Inhaled corticosteroid therapy, e.g., treatment for asthma, is acceptable. Use of intermittent corticosteroids as premedication to
prevent infusion reactions is allowed.)
The patient has active, clinically significant spleen infarction. within 12 months of screening which was radiologically confirmed (in absence of significant clinical symptoms, radiological evidence will suffice). Splenectomized patients are not excluded.
The patient has worsening bone necrosis. within 12 months of screening. Inactive or stable bone necrosis is not excluded.
The patient is pregnant or lactating. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
| To evaluate the safety of transitioning patients previously receiving enzyme replacement therapy with imiglucerase to GA-GCB therapy administered as the same number of units as their imiglucerase therapy. Safety assessments include adverse events (including infusion-related adverse events), concomitant medication use, vital signs, 12-lead ECG, physical examinations, and clinical laboratory tests (hematology, serum chemistry, urinalysis, and anti-GA-GCB antibody testing). |
|
| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | Information not present in EudraCT |
| E.6.2 | Prophylaxis | Information not present in EudraCT |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | Yes |
| E.6.5 | Efficacy | Yes |
| E.6.6 | Pharmacokinetic | Information not present in EudraCT |
| E.6.7 | Pharmacodynamic | Information not present in EudraCT |
| E.6.8 | Bioequivalence | Information not present in EudraCT |
| E.6.9 | Dose response | Information not present in EudraCT |
| E.6.10 | Pharmacogenetic | Information not present in EudraCT |
| E.6.11 | Pharmacogenomic | Information not present in EudraCT |
| E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
| E.6.13 | Others | Information not present in EudraCT |
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
| E.7.1.1 | First administration to humans | Information not present in EudraCT |
| E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
| E.7.1.3 | Other | Information not present in EudraCT |
| E.7.1.3.1 | Other trial type description | |
| E.7.2 | Therapeutic exploratory (Phase II) | Yes |
| E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
| E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
| E.8 Design of the trial |
| E.8.1 | Controlled | No |
| E.8.1.1 | Randomised | Information not present in EudraCT |
| E.8.1.2 | Open | Information not present in EudraCT |
| E.8.1.3 | Single blind | Information not present in EudraCT |
| E.8.1.4 | Double blind | Information not present in EudraCT |
| E.8.1.5 | Parallel group | Information not present in EudraCT |
| E.8.1.6 | Cross over | Information not present in EudraCT |
| E.8.1.7 | Other | Information not present in EudraCT |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
| E.8.2.2 | Placebo | Information not present in EudraCT |
| E.8.2.3 | Other | Information not present in EudraCT |
| E.8.3 |
The trial involves single site in the Member State concerned
| No |
| 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 | 6 |
| 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 |
|
| 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
|
|
| 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 | 8 |
| E.8.9.1 | In the Member State concerned days | |
| E.8.9.2 | In all countries concerned by the trial years | 1 |
| E.8.9.2 | In all countries concerned by the trial months | 10 |
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