An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease

Phase 1

• Conditions: Gaucher Disease; MedDRA version: 20.0Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2015-001578-17-GB
• Lead Sponsor: Shire Human Genetic Therapies, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-12-21
• Last Update Posted: 18 March 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 19

Inclusion Criteria

Each patient must meet the following criteria to be enrolled in this study.
1. The patient 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 patient’s medical history.
2. Patients must have a LS BMD Z-score < -1 or BMD T-score of < -1 as measured by DXA during the screening phase.
3. Patient is treatment-naïve, ie, has not received ERT or SRT in the 12 months prior to enrollment.
4. The patient is =18 and =70 years of age.
5. Female patients of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study. (See Section 6.8.3 for acceptable methods of contraception).
6. The patient, or patient’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).
7. The patient must be sufficiently cooperative to participate in this clinical study as judged by the investigator.
Are the trial subjects under 18? no
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 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

Patients who meet any of the following criteria will be excluded from the study.
1. Neurological symptoms indicating that the patient may have type 3 Gaucher disease.
2. 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).
3. Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with erythropoietin (or erythropoietin-like substances) during the past year.
4. 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 (eg, pain).
5. The patient is pregnant or lactating.
6. The patient has had a splenectomy.
7. The patient 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.
8. Severe vitamin D deficiency to the level that would be expected to result in osteomalacia (vitamin D < 10 ng/mL [25 nmol/L]). If there is mild vitamin D insufficiency at screening (vitamin D > 10 and < 30 ng/mL) treat with 4000 IU vitamin D per day for 1 month and rescreen.
9. The patient has previously interrupted ERT for safety reasons.
10. The patient has had hypersensitivity to the active substance or to any of the excipients.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified