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An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712

An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712

1

One participant in Japan with ASMD who completed Study DFI12712 was enrolled in this study and treated with olipudase alfa.

Planned: 1 Treated: 1 Evaluated: 1

No AEs were reported in this study.

Refer to "Adverse events" because the Primary Outcome of the study is AE. Secondary outocome is not applicable.

One Japanese participant with ASMD who completed DFI12712 was enrolled in this study and was treated with olipudase alfa. The participant completed the study. No AE was reported, and no new safety concerns were identified in this study.

No

https://jrct.mhlw.go.jp/latest-detail/jRCT2031210279

Tanaka Tomoyuki

Sanofi K.K.

Tokyo Opera City Tower, 3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo 163-1488, Japan

clinical-trials-jp@sanofi.com

Clinical Study Unit

Sanofi K.K.

Tokyo Opera City Tower, 3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo 163-1488, Japan

+81-3-6301-3670

clinical-trials-jp@sanofi.com

Complete

Sept. 01, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

The patient is eligible to be included in the study only if the following criteria apply:
1) Adult patient who has been enrolled in, and has completed, Study DFI12712 (ASCEND), a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled, repeat dose study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with ASMD.
2) Patient who is willing and able to provide signed written informed consent.
3) If female and of childbearing potential, patient with a negative urine pregnancy test (beta-HCG) result.
4) If female and of childbearing potential or male and sexually active, patient who is willing to abstain from heterosexual intercourse in accordance with her or his preferred and usual life style, or to use 2 acceptable, effective contraceptive methods, while participating in this study and for 15 days after the last infusion of olipudase alfa.

A patient is excluded from the study if any of the following criteria apply:
1) Any patient with a diagnosis of ASMD who has not participated in Study DFI12712.
2) Any new condition or worsening of an existing condition that, in the opinion of the Investigator, would make the patient unsuitable for enrollment, or could interfere with the patient's participation in or completion of the study.
3) Any systemic hypersensitivity reaction to olipudase alfa in Study DFI12712 that, in the opinion of the Investigator, could indicate that continued treatment with olipudase alfa may present an unreasonable risk.
4) Any patient who, in the opinion of the Investigator, is unable to comply with study requirements.
5) Unwillingness or inability to abstain from alcohol for 1 day prior to and 3 days after each olipudase alfa infusion, for the duration of the study period.
6) Any of the following medical conditions:
- Active, serious intercurrent illness which will preclude enrollment until recovery is complete.
- Any other serious medical or psychiatric condition that may preclude participation, or circumstances that may interfere with compliance in this study.
- Requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.
- Pregnancy or breastfeeding.

Both

Acid sphingomyelinase deficiency

Drug: Olipudase alfa (GZ402665)
Pharmaceutical form: lyophilized powder, Route of administration: Intravenous (IV) infusion.

Adverse event

none