AveXis klinische Gen-Studien in Europa: STR1VE EU, SPRINT, REACH

und es geht in den nächsten Meilenstein in der Behandlung von SMA. AveXis möchte in den nächsten sechs Monaten die ersten Gentherapie-Studien in Europa starten.

SMA Europe hatte AveXis gebeten, es über die Entwicklung seiner klinischen Studien mit der SMA Gentherapie zu aktualisieren. In Europa ist geplant, so berichtet  Initiative-SMA


Überblick: STR1VE EU wird voraussichtlich im ersten Halbjahr 2018 beginnen und SMA-Typ-I-Patienten in mehreren EU-Zentren rekrutieren.
Verabreichung: In der Studie STRIVE EU wird AVXS-101 mit einer einzigen intravenösen Infusion verabreicht.
Wer: Über die STRIVE EU- wird 30 Patienten mit SMA vom Typ I, die zum Zeitpunkt der Gentherapie weniger als 6 Monate alt sind.


Übersicht: SPRINT wird voraussichtlich in der ersten Hälfte des Jahres 2018 beginnen und umfasst präsymptomatische Patienten mit SMA Typ I, II und III.
Anwendung und Dosierung: In der SPRINT-Studie wird AVXS-101 mit einer einzigen intravenösen Infusion verabreicht.
Wer: Die SPRINT-Studie soll 44 Patienten mit 2, 3 und 4 Kopien des SMN2-Gens eingeschlossen werden. Patienten sollten weniger als 6 Wochen alt sein und keine Symptome von SMA haben.


Überblick: Es wird erwartet, dass die REACH-Studie Patienten mit SMA Typ I, II und III später im Jahr 2018 oder Anfang 2019 einbeziehen.

Anwendung und Dosierung: In der REACH-Studie wird AVXS-101 durch einmalige intrathekale Injektion verabreicht. Die Daten aus STRONG-Studien (die erste Studie, die AVXS-101 mit einer intrathekalen Injektion liefern wird) werden helfen, die endgültige Studie zu bestimmen

Wer: Es wird erwartet, dass ungefähr 50 Patienten mit SMA Typ I, II und III werden im Alter von 6  Monate bis ca. 18 Jahren in die REACH-Studie aufgenommen werden.

Clinical Trials with AVXS-101

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Liz Andersson

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4 Responses to AveXis klinische Gen-Studien in Europa: STR1VE EU, SPRINT, REACH

  1. “Throughout the first quarter and in recent weeks, we have made significant progress in executing against our strategic plan and are quite pleased that Novartis, with its pending acquisition of the company, recognizes the value that the AveXis team has created,” said Sean Nolan, President and Chief Executive Officer of AveXis. “Data presented recently at the American Academy of Neurology from both the Type 1 Phase 1 and STR1VE studies highlight the clinically transformative, durable and consistent response of AVXS-101. We remain steadfast in our goal to make our gene therapy available as quickly and safely as possible for families suffering from SMA.”


    Presented Data at the 2018 Annual Meeting of the American Academy of Neurology:
    On April 24, 2018, AveXis reported initial results from the U.S. pivotal trial (STR1VE) and 24-month follow-up data from the Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1.

    STR1VE Data as of April 11, 2018
    Eleven patients were enrolled in the trial, and six patients were symptomatic and at least one-month post gene therapy treatment.
    All symptomatic patients (6 of 6) were alive and event-free. AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated. At the time of gene transfer, the mean age was 3.2 months, with the oldest patient being 5.0 months of age.

    In the six patients who were at least one-month post gene transfer, a cumulative total of 25 adverse events (AEs) were reported. Two patients experienced transient elevations in transaminases greater than 3x ULN that were not clinically significant and all resolved with prednisolone treatment without any clinical manifestations or sequelae. There were no serious adverse events (SAEs) reported.

    Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) scores increased by an average of 7.8 at one month after gene transfer (in six patients) and 17.3 at three months after gene transfer (in three patients), reflecting improvement in motor function. These data correlate to CHOP-INTEND achievement by the proposed therapeutic dose cohort (Cohort 2) in the Phase 1 trial, which experienced mean increases of 9.8 points at one month and 15.4 points at three months. Early CHOP-INTEND increases have been observed to be associated with eventual milestone achievement.

    24-Month Follow-Up Data from Phase 1 Trial
    Twenty-four months following gene transfer, 15 of 15 (100%) patients were alive and without need for permanent ventilation.

    The median age at last follow-up was 27.8 months and 30.7 months for patients in Cohort 2 and low-dose cohort (Cohort 1), respectively. Natural history indicates only eight percent of untreated patients with SMA Type 1 survive event-free at 20 months of age. After the 24-month follow-up, to date, 11 patients have enrolled in the Long-Term Follow-Up (LTFU) trial for ongoing evaluation.

    Patients in Cohort 2 continued to achieve new milestones during the LTFU trial.
    Two additional patients achieved the ability to sit unassisted for 30 seconds or more. Eleven of 12 (92%) patients could sit unassisted.
    Two additional patients achieved the ability to stand with assistance. Four of 12 (33%) patients could stand with assistance.

    Three of the four patients achieving these new milestones were on AVXS-101 alone (one sitting and two standing with assistance).
    The oldest child from Cohort 2 at the time of last visit in the LTFU study was 46.2 months old and 40.6 months post gene therapy.

    AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated, with no new treatment-related safety or tolerability concerns identified at the 24-month follow-up.

    Dosed Fourth Patient in Phase 1 Trial of AVXS-101 in SMA Type 2 (STRONG): Following review of safety data from the first three patients dosed in Cohort 1 using the lower dose (6.0 x 1013 vg), the first patient has been dosed in Cohort 2, the higher dose cohort (1.2 X 1014 vg). Four total patients with SMA Type 2 have now been treated with AVXS-101 in the STRONG study. Three patients less than 60 months of age will be enrolled in Cohort 2 and, if safety is established according to the Data Safety Monitoring Board, an additional 21 patients will be enrolled in Cohort 2 until there are a total of 12 patients less than 24 months, and 12 patients at least 24 months but less than 60 months of age.

    Initiated Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3 Trial (SPRINT): On April 25, 2018, AveXis announced that the first patient has been dosed in a multi-national Phase 3 trial evaluating AVXS-101 in pre-symptomatic infants less than six weeks of age with SMA Types 1, 2 and 3.

    Awarded SAKIGAKE Designation for SMA Type 1: On March 27, 2018, AveXis announced that Japan’s Ministry of Health, Labour and Welfare awarded the company’s initial product candidate, AVXS-101, SAKIGAKE Designation for the treatment of SMA Type 1. The designation was based on data from the Phase 1 clinical trial of AveXis’ proprietary gene therapy. SAKIGAKE is intended to promote research and development in Japan for innovative new medical products that satisfy certain criteria, such as the severity of the intended indication, by providing prioritized consultation review during the early stages of development and by shortening the target review period for license applications from 12 months to as few as six months. The benefits of SAKIGAKE Designation are similar to the Breakthrough Therapy Designation in the United States and access into the PRIority MEdicines (PRIME) scheme in the EU, both of which have already been granted to AVXS-101.

    Entered into Licensing Agreement with Généthon: On March 13, 2018, AveXis and Généthon announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of the SMN gene using the AAV9 vector into the central nervous system for the treatment of SMA. This agreement strengthens the AVXS-101 intellectual property estate by providing greater freedom to operate in the U.S., Europe and Japan. Under the terms of the agreement, Généthon granted AveXis a license to patents in the U.S., Europe and Japan for the AAV9 SMN product and in vivo gene therapy delivery of AAV9 vector into the CNS using intrathecal or intravenous routes of administration for the treatment of SMA.

    Expanded Relationship with REGENXBIO via Amended SMA License: On January 8, 2018, AveXis and REGENXBIO Inc. (REGENXBIO) announced that under the terms of an amended license agreement for SMA, REGENXBIO granted AveXis exclusive, worldwide rights to all vectors in REGENXBIO’s NAV Technology Platform for the treatment of SMA in addition to adding and amending certain terms of their 2014 license agreement for SMA, including the modification of the assignment provision to permit assignment without REGENXBIO’s consent in the event of a change of control of AveXis.

  2. MiaMia says:


  3. Mia hat hier

    die Deutschen Zentren geschrieben die Probanden zu Studie mit Avxis 101 durchführen. No ist die Registration nicht offen, kann sich aber nur noch um wenige Wochen handeln

  4. MiaMia says:

    Last Update Posted : September 3, 2018
    Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STRIVE-EU)
    The trial includes a screening period, a gene replacement therapy period, and a follow-up period. During the screening period (Days -30 to -2), patients whose parent(s)/legal guardian(s) provide informed consent will complete screening procedures to determine eligibility for trial enrollment. Patients who meet the entry criteria will enter the in-patient gene replacement therapy period (Day -1 to Day 3). On Day -1, patients will be admitted to the hospital for pre-treatment baseline procedures. On Day 1, patients will receive a one-time intravenous (IV) infusion of AVXS-101, and will undergo in-patient safety monitoring over the next 48 hours. Patients may be discharged 48 hours after the infusion, based on Investigator judgment.