Phase 3
N=20
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Gaucher Disease
Bottom Line
View on ClinicalTrials.gov: NCT05529992 ↗Enrolled (actual)
20
Serious AEs
20.0%
Results posted
Feb 2025
Primary outcome: Primary: Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE) — 20 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Velaglucerase Alfa (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Takeda
- Primary completion
- Aug 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE) |
20 | — |
| SECONDARY Percentage of Participants With TEAEs |
95 | — |
| SECONDARY Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event |
5 | — |
| SECONDARY Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53 |
15.8; 10.5 | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53 |
2; 3; 2; 2; 5; 4 | — |
| SECONDARY Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis |
1; 0; 1; 1; 7; 3 | — |
| SECONDARY Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53 |
15; 1; 18; 9; 11; 5 | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Measurements at Week 53 |
1 | — |
| SECONDARY Change From Baseline to Week 53 in Hemoglobin Concentration |
2.34 | — |
| SECONDARY Change From Baseline to Week 53 in Platelet Count |
42.1 | — |
| SECONDARY Change From Baseline to Week 53 in Normalized Liver Volume |
-1.11 | — |
| SECONDARY Change From Baseline to Week 53 in Normalized Spleen Volume |
-3.09 | — |
| SECONDARY Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores |
7.015; 2.858 | — |
| SECONDARY Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores |
-0.02; 0.38 | — |
| SECONDARY Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1 |
5220 | — |
| SECONDARY Serum Concentration for VPRIV at Week 37 |
3450 | — |
| SECONDARY Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV |
49.00 | — |
| SECONDARY AUCinf: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for VPRIV |
235000 | — |
| SECONDARY Terminal Phase Elimination Half-life (T1/2) for VPRIV |
10.12 | — |
| SECONDARY Oral Clearance (CL) for VPRIV |
7.27 | — |
| SECONDARY Apparent Steady-state Volume of Distribution (Vss) for VPRIV |
96.3 | — |
| SECONDARY Percent Change From Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C Motif] Ligand 18 |
-58.94 | — |
| SECONDARY Percent Change From Baseline to Week 53 in Biomarker: Glucopsychosine |
-63.88 | — |
Summary
The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT).
Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.
Eligibility Criteria
Inclusion:
- Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:
- Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
- Decreased GCB activity level that is >30% of normal, but with confirmation of genetic mutation test
- Is at least 2 years of age, inclusive, at screening
- Is naive to treatment for Gaucher disease (Has not received treatment for Gaucher disease [investigational or approved products] within the 12 months prior to screening) OR Is receiving or has recently received Imiglucerase ERT (Has received Imiglucerase treatment within the 12 months prior to screening and not within the 14 days prior to screening)
- Has Gaucher disease-related hematological abnormalities, defined as
- Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
- A platelet count of 5 times normal) AND/OR
- Participant has hepatomegaly, assessed by palpation or by abdominal radiology scan (MRI or CT scan); Participants who have undergone splenectomy must have satisfied these criteria for this study.
Exclusion:
- Has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease as assessed by the investigator
- Has had a splenectomy or an active, clinically significant spleen infarction within the 12 months prior to screening
- Has received treatment with any investigational drug or device within 30 days prior to screening, or within 5 half-lives of that investigational product, whichever is greater; such treatment during the study will not be permitted
- Is currently receiving red blood cell growth factor (eg, erythropoietin), chronic systemic corticosteroids, or has been on such treatment within the 6 months prior to screening
- Presents with non-Gaucher disease related exacerbated anemia at screening
- Has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)
Data sourced from ClinicalTrials.gov (NCT05529992). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.