ABO-101 Gene Therapy | MPS IIIB | Phase I-II | Abeona

March 9, 2020

Page last updated: Feb. 23, 2024

Page reviewed by: Dr. Cara O’Neill, FAAP

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Summary Information

Study Title: “Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB” ClinicalTrials.gov ID NCT03315182

Trial Listing: Read this clinical trial’s information on ClinicalTrials.gov, for more details about the study.

Status: Completed

Study Start: October 2017

Study Ended: April 2022

Number of patients enrolled: 11

Study design: Open label dose escalation study

Study Description:
This study aimed to evaluate the safety and early efficacy of the intravenous gene therapy product ABO-101 for children with Sanfilippo type B. This therapy was developed by Haiyan Fu, PhD, and Doug McCarty, PhD, during their tenures at Ohio State University/Nationwide Children’s Hospital. This dose escalation trial that began with the minimal efficacious dose as determined by preclinical studies and approved by the FDA. During the course of the trial, as safety was shown, the dose was escalated according to the clinical protocol.

The therapy was then licensed to Abeona Therapeutics for further development. The program was discontinued in 2022 by Abeona Therapeutics due to a lack of drug supply and for business reasons unrelated to the product safety profile and/or signs of efficacy.

Our community is grateful to the patients and families who choose to participate in clinical trials. Their participation is essential to the advancement of science that brings us closer to an approved treatment for Sanfilippo syndrome.

Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein. A tapering course of prophylactic oral prednisone or prednisolone will be administered for a period of at least two months.

Dosing cohorts (groups):

  • Cohort 1 (Low Dose): 2 x 10e13 vg/kg (vector genomes per kilogram of body weight). Two patients were enrolled in this cohort.
  • Cohort 2 (Medium Dose): 5 x 10e13 vg/kg. Four to Five planned enrollees in this cohort.
  • Cohort 3 (High Dose): 1 x 10e14 vg/kg. Four to Eight participants were planned in this cohort.

As the study progressed, the inclusion criteria were revised to a narrowed group of children: ages 0-2 years of age or >2 years of age with a developmental quotient of >60. This youngest group of patients receiving the highest dose of gene therapy are considered to be part of the updated study name “Transpher A.”

Long-Term Follow-up

Study Title: “A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101” (ClinicalTrials.gov ID NCT04655911)

Study Status: TERMINATED

Abeona (the sponsor) has decided to discontinue development activities for Product AB0-101 due to a lack of drug supply and for business reasons unrelated to the product safety profile and/or signs of efficacy

Study Start: October 2020

Study Termination: April 2022

Patients enrolled: 1

Study description: This was a multicenter, non-interventional, long-term follow-up (LTFU) study in participants who have been treated with ABO-101 in a prior trial. Eligible participants were planned to undergo clinical evaluations at prespecified intervals for 3 years from the last visit in the prior clinical trial (up to 5 years post-treatment).

Associated publications with this experimental therapeutic

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