A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND
- Conditions
- Patients with type 1 Gaucher disease who previously received imigluceraseMedDRA version: 9.1Level: LLTClassification code 10018048Term: Gaucher's disease
- Registration Number
- EUCTR2006-006304-11-IT
- Lead Sponsor
- SHIRE HUMAN GENETIC THERAPIES, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 40
The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (GCB) activity in leukocytes or by genotype analysis. 2. The patient has received imiglucerase at a dose < or equal to 60 U/kg and > or equal to 15 U/kg every other week for a minimum of 30 consecutive months. Patients must have received the same dose and dose regimen during the 6 months prior to study enrollment. (Note: Patients who are anti-imiglucerase antibody positive will be allowed to enter this study). 3. The patient is at least 2 years of age. 4. 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. 5. 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). 6. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
The patient has a hemoglobin concentration < o uguale a 10 g/dL and a platelet count < or equal to 80 x 103 platelets/mm3. 2. The difference between the patient?s highest and lowest hemoglobin concentration collected over 3 consecutive evaluations is > 1 g/dL, AND/OR, the difference between the patient?s highest and lowest platelet count collected over 3 consecutive evaluations is > 20%. (Note: At least 1 of these values must have been collected at least 6 months prior to the Screening evaluation and 1 value must be collected during Screening). 3. The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease. 4. 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. 5. 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. 6. The patient is known to be positive for hepatitis B and/or C, i.e., has a documented positive result. Patients who do not have a documented positive result will be tested for hepatitis during Screening. 7. The patient presents with sustained iron, folic acid and/or vitamin B12 deficiency-related anemia during Screening (i.e., non-Gaucher disease-related anemia). 8. 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. 9. 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.). 10. 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. 11. The patient has had inconsistent treatment with imiglucerase in the last 6 months such as a drug holiday. 12. The patient has experienced an anaphylactic reaction during treatment with imiglucerase. 13. The patient has received miglustat during the 6 months prior to study enrollment. 14. The patient is currently receiving erythropoietin or chronic systemic corticosteroids in the last 6 months. (NOTE: Use of intermittent corticosteroids as premedication to prevent infusion reactions is allowed.) 15. The patient has experienced spleen infarction. 16. The patient has bone necrosis.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method