Gaucher Disease
Conditions
Keywords
Gaucher disease, imiglucerase, glucocerebrosidase, enzyme replacement therapy, lysosomal storage disorder
Brief summary
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
Detailed description
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT. Eligible patients will enter a 12-week Stability Evaluation Period to establish the stability of their disease. Patients with stable disease will then be switched from their imiglucerase treatment to receive intravenous (IV) infusions of taliglucerase alfa every two weeks for a total of 20 IV infusions. The dose of taliglucerase alfa will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital), infusion center, or at home. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.
Interventions
Intravenous infusion every 2 weeks
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Males and females, 2 years or older * Confirmed diagnosis of Gaucher disease by the enzymatic activity assay * Stable Gaucher disease * Treatment with imiglucerase (Cerezyme®) for at least 2 years and on a stable maintenance regimen (dose and regimen unchanged, except for situation of drug shortage) for at least the last six months * Able to provide written informed consent
Exclusion criteria
* Currently taking another experimental drug for any condition * History of allergy to carrots * History of allergy to beta lactam antibiotics * Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions * Presence of HIV and/or HBsAg and/or hepatitis C infection * Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency * Presence of any significant comorbidity that could confound the interpretation of the clinical response to taliglucerase alfa * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin | Every 3 months from Baseline to Month 9 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Platelet Count | Every 3 months from Baseline to Month 9 | — |
| Spleen Volume | Baseline and 9 Months | Spleen volume measured by MRI in mL |
| Liver Volume | Baseline and 9 months | Liver volume measured by MRI |
Countries
Australia, Canada, Israel, Spain, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Taliglucerase Alfa Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks | 31 |
| Total | 31 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Taliglucerase Alfa |
|---|---|
| Age, Categorical <=18 years | 5 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 25 Participants |
| Age, Continuous | 41.6 years STANDARD_DEVIATION 17.4 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 30 Participants |
| Region of Enrollment Australia | 3 participants |
| Region of Enrollment Canada | 1 participants |
| Region of Enrollment Israel | 9 participants |
| Region of Enrollment Serbia | 4 participants |
| Region of Enrollment Singapore | 1 participants |
| Region of Enrollment Spain | 4 participants |
| Region of Enrollment United Kingdom | 2 participants |
| Region of Enrollment United States | 7 participants |
| Religion JEWISH - ASHKENAZI | 14 participants |
| Religion JEWISH - NONASHKENAZI | 0 participants |
| Religion NON JEWISH | 15 participants |
| Religion UNREPORTED | 2 participants |
| Sex: Female, Male Female | 14 Participants |
| Sex: Female, Male Male | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 29 / 31 |
| serious Total, serious adverse events | 3 / 31 |
Outcome results
Primary
Hemoglobin
Time frame: Every 3 months from Baseline to Month 9
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Taliglucerase Alfa | Hemoglobin | Baseline | 13.5 g/dL | Standard Deviation 1.4 |
| Taliglucerase Alfa | Hemoglobin | Month 3 | 13.3 g/dL | Standard Deviation 1.5 |
| Taliglucerase Alfa | Hemoglobin | Month 6 | 13.3 g/dL | Standard Deviation 1.6 |
| Taliglucerase Alfa | Hemoglobin | Month 9 | 13.4 g/dL | Standard Deviation 1.5 |
Other Pre-specified
Liver Volume
Liver volume measured by MRI
Time frame: Baseline and 9 months
Population: 2 patients were MRI phobic
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Taliglucerase Alfa | Liver Volume | Baseline | 1766 mL | Standard Deviation 474.5 |
| Taliglucerase Alfa | Liver Volume | Month 9 | 1716 mL | Standard Deviation 455.6 |
Other Pre-specified
Platelet Count
Time frame: Every 3 months from Baseline to Month 9
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Taliglucerase Alfa | Platelet Count | Baseline | 161137 platelets/mm^3 | Standard Deviation 73387 |
| Taliglucerase Alfa | Platelet Count | Month 3 | 150800 platelets/mm^3 | Standard Deviation 66038 |
| Taliglucerase Alfa | Platelet Count | Month 6 | 157586 platelets/mm^3 | Standard Deviation 78357 |
| Taliglucerase Alfa | Platelet Count | Month 9 | 161167 platelets/mm^3 | Standard Deviation 80820 |
Other Pre-specified
Spleen Volume
Spleen volume measured by MRI in mL
Time frame: Baseline and 9 Months
Population: 25 patients had spleen volume measured by MRI, 2 were MRI phobic and 3 were splenectomized
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Taliglucerase Alfa | Spleen Volume | Baseline | 720.5 mL | Standard Deviation 579.5 |
| Taliglucerase Alfa | Spleen Volume | Month 9 | 654.7 mL | Standard Deviation 535.9 |