Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 11 January 2021
• Main ID: NCT02574286
• Date of registration: 09/10/2015
• Prospective Registration: Yes
• Primary sponsor: Shire
• Public title: Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
• Scientific title: An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease
• Date of first enrolment: June 29, 2016
• Target sample size: 21
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02574286
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Israel, Spain, United Kingdom, United States

Contacts

• Name: Study Director
• Affiliation: Shire

Key inclusion & exclusion criteria

Inclusion Criteria:

- The participant has a documented diagnosis of type 1 Gaucher disease, as documented by
deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts.
Diagnosis by only dry blood spot test is insufficient. Diagnosis may be based on
results obtained prior to screening if documented in the participant's medical
history.

- Participants must have a LS BMD Z-score less than (<) -1 or BMD T-score of < -1 as
measured by DXA during the screening phase.

- Participant is treatment-naive, that is (ie,) has not received ERT or SRT in the 12
months prior to enrollment.

- The participant is greater than or equal to (>=) 18 and less than or equal to (<=) 70
years of age.

- Female participants of childbearing potential must agree to use a medically acceptable
method of contraception at all times during the study.

- The participant, or participant's legally authorized representative(s), if applicable,
understands the nature, scope, and possible consequences of the study and has provided
written informed consent that has been approved by the Institutional Review
Board/Independent Ethics Committee (IRB/IEC).

- The participant must be sufficiently cooperative to participate in this clinical study
as judged by the investigator.

Exclusion criteria

- Neurological symptoms indicating that the participant may have type 3 Gaucher disease.

- A significant comorbidity, which, as determined by the investigator, might affect
study data or confound the study results (eg, malignancies, primary biliary cirrhosis,
autoimmune liver disease, etc).

- Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with
erythropoietin (or erythropoietin-like substances) during the past year.

- Structural, joint-associated bone damage of such extent and severity that the
investigator deems it could impact participation in the study and assessment of
relevant study endpoints (example, pain).

- The participant is pregnant or lactating.

- The participant has had a splenectomy. (This criterion is not meant to exclude
participants who have accessory spleens.)

- The participant is enrolled in another clinical study that involves clinical
investigations or use of any investigational product (drug or device) within 30 days
prior to study enrollment or at any time during the study.

- Severe vitamin D deficiency to the level that would be expected to result in
osteomalacia (vitamin D < 10 nanograms per milliliter [ng/mL] [25 nanomoles per liter
{nmol/L}]). If there is mild vitamin D insufficiency at screening (vitamin D greater
than [>] 10 and < 30 ng/mL) treat with 4000 IU vitamin D per day for 1 month and
rescreen.

- The participant has previously interrupted ERT for safety reasons.

- The participant has had hypersensitivity to the active substance or to any of the
excipients.



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease

Intervention(s)

Drug: Velaglucerase alfa

Dietary Supplement: Vitamin D

Primary Outcome(s)

Change From Baseline in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-Score to Week 103/End of Study [Time Frame: Baseline, Week 103 (end of study)]

Secondary Outcome(s)

Change From Baseline in Severity of Bone Pain [Time Frame: Baseline, Week 51 and 103 (end of study)]

Number of Participants with Adverse Events (AEs) [Time Frame: From start of study drug infusion up to 37 days after the last study drug infusion (106 weeks)]

Change From Baseline in Normalized Spleen Volume [Time Frame: Baseline, Week 51 and 103 (end of study).]

Change From Baseline in Impact of Bone Pain [Time Frame: Baseline, Week 51 and 103 (end of study)]

Change From Baseline in Overall Fatigue [Time Frame: Baseline, Week 51 and 103 (end of study)]

Change From Baseline in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score at Week 51 [Time Frame: Baseline, Week 51]

Shifts In World Health Organization (WHO) Bone Mineral Density (BMD) Classifications (Normal Bone Density, Osteopenia, Osteoporosis) Based on Lumbar Spine (LS) T-Scores [Time Frame: Baseline, Week 51, and 103 (end of study)]

Change From Baseline in Normalized Liver Volume [Time Frame: Baseline, Week 51 and 103 (end of study)]

Change From Baseline Over Time in Hemoglobin Concentration [Time Frame: Baseline, Week 13, 25, 37, 51, 65, 77, 89, and 103 (end of study)]

Change From Baseline in Bone Marrow Burden (BMB) Score [Time Frame: Baseline, Weeks 51, and 103 (end of study)]

Change From Baseline in Lumbar Spine (LS) Bone Mineral Density (BMD) [Time Frame: Baseline, Week 51 and 103 (end of study)]

Change From Baseline Over Time in Platelet Count [Time Frame: Baseline, Week 13, 25, 37, 51, 65, 77, 89, and 103 (end of study)]

Secondary ID(s)

SHP-GCB-402

2015-001578-17

Source(s) of Monetary Support

Please refer to primary and secondary sponsors