Nervenheilkunde 2019; 38(05): 300
DOI: 10.1055/s-0039-1685081
Poster
Spinale Muskelatrophien
Georg Thieme Verlag KG Stuttgart · New York

Genetic Replacement Therapy (GRT) Clinical Trial with AVXS-101 in Presymptomatic Spinal Muscular Atrophy (SMA): Design of the Phase III Trial and Baseline Demographic Data

M Schultz
1   University of Wisconsin School of Medicine and Public Health, Department of Neurology, Madison, Vereinigte Staaten
,
K Swoboda
2   Massachusetts General Hospital, Department of Neurology, Boston, Vereinigte Staaten
,
E Kernbauer
3   Avexis, Inc., Bannockburn, Vereinigte Staaten
,
F Ogrinc
3   Avexis, Inc., Bannockburn, Vereinigte Staaten
,
DE Feltner
3   Avexis, Inc., Bannockburn, Vereinigte Staaten
,
BE McGill
3   Avexis, Inc., Bannockburn, Vereinigte Staaten
,
DM Sproule
3   Avexis, Inc., Bannockburn, Vereinigte Staaten
,
KA Strauss
4   Clinic for Special Children, Strasburg, Frankreich
› Author Affiliations
Further Information

Publication History

Publication Date:
06 May 2019 (online)

Also available at
 
 

    Introduction:

    The GRT onasemnogen Abeparvovec (AVXS-101) for treatment. d. genetic cause of SMA (loss of the survival motor neuron gene SMN) improved outcomes in patients with SMA type I (NCT02122952). Design and demo graf. Data at the beginning of the Phase III SPR1NT study (NCT03505099). effectiveness and safety v. AVXS-101 at presymptom. Newborns are presented.

    Methods:

    SPR1NT is a multicentre, open, single arm. Study with ≥27 SMA patients with 2 – 3 copies of SMN2 (2 ×–3 × SMN2). Präsympt. Patients ≤6 wks. received 1 single dose AVXS-101 (therapeutic dose: 1.1 × 10 14 VG/kg). Safety and effectiveness are 18 mo. (2 × SMN2) or 24 mo. (3 × SMN2) assessed until end of study. Primary results are ≥30 s sitting (2 × SMN2) or standing (3 × SMN2) at the end of the study.

    Results:

    Until Aug. 2018, AVXS-101 3 was presympted. Säugl. (2 female, 1 male) with SMA type I and 2 × SMN2 given ≤33 days after birth. One child was diagnosed prenatal, 2 after birth on the 1st and 9th day. The presented baseline dose levels include weight, motor function (CHOP-INTEND + Bayley-III) and AAV9 antibody titer of the mother.

    Discussion:

    In d. Phase III SPR1NT trial of AVXS-101 will demonstrate efficacy and safety of a single-dose iv AVXS-101 in pre-symptomatic administration. SMA patients examined. Early treatment is important to prevent motor neuron loss, promote motor function and motor. To reach milestones.


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