A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

• Status: Recruiting
• Trial end date: 2022-08-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ari Zimran
• Collaborator: Pfizer

Criteria

Inclusion Criteria:

1. Male or female of any age; however, if female:

• must be using contraception if of childbearing potential or must be surgically sterile

• must not be lactating

2. Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor.

3. Splenomegaly at least 5 x multiples of normal (MN).

4. Treatment-naïve.

Exclusion Criteria:

Eligible subjects may not have any of the following exclusion criteria:

1. Type 2 GD.

2. Presence of myoclonic seizures.

3. At least one allele of:

• N370S (N409S in recent nomenclature)

• R496H (R535H in recent nomenclature)

4. Presence of calcification in heart valves or arteries in echocardiography.

5. Presence of untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism. (Resolved anemia is not an exclusion criterion.)

6. Presence of human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and/or hepatitis C infections.

7. Splenectomy and bone marrow transplantation.

8. Presence of any medical, emotional, behavioural, or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

9. Any other disorder that may interfere with the results of the efficacy endpoints.

10. Pregnancy or breastfeeding.

11. Currently taking another investigational drug for any condition or any therapeutic drug for Gaucher disease.

12. The subject and/or subject's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study.

13. Medical history of any food/drugs allergy.