A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

A Long-term Follow-up Study of Patients With MPS IIIB From Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU)

This is 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 will undergo clinical evaluations at prespecified intervals for 3 years from the last visit in the prior clinical trial (up to 5 years post-treatment).

Study Overview

• Status: Terminated
• Conditions: Mucopolysaccharidosis III-B
• Intervention / Treatment: Biological: ABO-101

Detailed Description

This is a multicenter, noninterventional, long-term follow-up study of patients with (Mucopolysaccharidosis IIIB) MPS IIIB who have completed a prior clinical trial involving the administration of ABO-101. This study is designed to provide LTFU in accordance with the FDA and European Medicines Agency (EMA) guidelines for patients treated with gene therapy products. The duration of the current study is 3 years for a total of up to 5 years post-treatment for these participants who rollover from a prior clinical trial of ABO-101. Participants will have a maximum of 5 annual scheduled visits with assessments as specified in the schedule of assessments.

• Study Type: Observational
• Enrollment (Actual): 1

Contacts and Locations

Study Locations

• France
  • Paris, France, 75571
    • Armand-Trousseau Hospital
• Germany
  • Hamburg, Germany, 20246
    • University Hospital Hamburg-Eppendorf
• United States
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

This long-term follow-up study will enroll up to 24patients with MPS IIIB from prior ABO-101 clinical trials. Participants from the previous trials are of any racial, ethnic, or gender background, and could be in the early, middle or advanced phase of the disease

Description

Inclusion Criteria:

• Participants that have completed a prior clinical trial involving the administration of ABO-101
• Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule

Exclusion Criteria:

• Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-101 during the duration of this study
• Any other situation that precludes the participant from undergoing procedures required in this study

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
ABO-101; Participants from prior interventional trials involving the administration of ABO-101.	Biological: ABO-101; Gene therapy rAAV9.CMV.hNAGLU

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)	60 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Antibody formation (humoral) against the AAV9 capsid	Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.	60 Months
Age Equivalent Compared to Natural History Study Data	Change from baseline in the Age Equivalent after treatment compared to Natural History Study data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery far Children; Second Edition, based on chronological and developmental age	AII study visits (30, 36, 42, 48, and 60 Months)
Developmental Quotient Compared to Natural History Study Data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children	Change from baseline in the Developmental Quotient (DQ) after treatment compared to Natural History Study data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children; Second Edition, based on chronological and developmental age	AII study visits (30, 36, 42, 48, and 60 Months)
Cognitive Age Equivalent Compared to Natural History Study Data	Change from baseline in the Cognitive Age Equivalent after treatment compared to Natural History Study, calculated using the Bayley Scales of lnfant and Toddler Development - Third edition or the Kaufman Assessment Battery far Children. Second Edition, based on developmental age	AII study visits (30, 36, 42, 48, and 60 Months)
Developmental Quotient Compared to Natural History Study Data calculated using the Bayley Scales of lnfant and Toddler Development or the Kaufman Assessment Battery for Children	Change from baseline in the Developmental Quotient after treatment compared to Natural History Study, calculated using the Bayley Scales of lnfant and Toddler Development - Third edition or the Kaufman Assessment Battery far Children. Second Edition, based on developmental age	AII study visits (30, 36, 42, 48, and 60 Months)
Adaptive Age Equivalent Compared to Natural History Study Data	Change from baseline in the Adaptive Age Equivalent score after treatment compared to Natural History Study data, as assessed by parent report using the Vineland Adaptive Behavior Scale II Survey farm	AII study visits (30, 36, 42, 48, and 60 Months)
Pediatric Quality of Life lnventory (PedsQL ™)	Quality of life based on Pediatric Quality of Life lnventory (PedsQL ™) applicable for participants who completed these evaluations in the prior clinical trial.	AII study visits (30, 36, 42, 48, and 60 Months)
Parenting Stress lndex, 4th Edition (PSl-4)	Quality of life based on Parenting Stress lndex, 4th Edition (PSl-4).	AII study visits (30, 36, 42, 48, and 60 Months)
Parental Global lmpression Scale	Evolution on Parental Global lmpression Score, applicable for patients who completed these evaluations in the prior clinical trial.	AII study visits (30, 36, 42, 48, and 60 Months)
Clinical Global lmpression lmprovement Scale	Evolution of Clinical Global lmpression lmprovement Score applicable for patients who completed these evaluations in the prior clinical trial.	AII study visits (30, 36, 42, 48, and 60 Months)
Parent Symptom Scale Questionnaire	Evolution on Parent Symptom Score Questionnaire applicable for patients who completed these evaluations in the prior clinical trial.	AII study visits (30, 36, 42, 48, and 60 Months)
T-cell responses against the AAV9 capsid	Long-term immunological responses defined as T-cell responses against the AAV9 capsid	60 Months
T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product	Long-term immunological responses defined as T-cell responses against the α-N- acetylglucosaminidase transgene product.	60 Months
Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product	Long-term immunological responses defined as antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU)	60 Months
Viral Load	Long-term viral load, in applicable cases.	AII study visits (30, 36, 42, 48, and 60 Months)

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sanfilippo Behavior Rating Scale	Change from baseline in the Sanfilippo Behavior Rating Scale (SBRS) scale after treatment compared to Natural History Study data, as assessed by parent report using the Sanfilippo Behavior Rating Scale form. Minimum value is 0 and maximum value is 6 where higher score indicates a worse outcome	30, 36, 42, 48, and 60 Months
Children's Sleep Habits Questionnaire (CSHQ)	Quality of life based on sleep-pattern evaluation based on modified Children's Sleep Habits Questionnaire (CSHQ).Minimum value is 1 and maximum value is 111 where higher scores indicates a worse outcome	30, 36, 42, 48, and 60 Months
Neutralizing antibody against the AAV9 capsid	Long-term immunological responses defined as neutralizing antibody formation against the AAV9 capsid	60 Months

Collaborators and Investigators

• Sponsor: Abeona Therapeutics, Inc

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 28, 2020
• Primary Completion (ACTUAL): April 7, 2022
• Study Completion (ACTUAL): April 7, 2022

Study Registration Dates

• First Submitted: November 17, 2020
• First Submitted That Met QC Criteria: November 30, 2020
• First Posted (ACTUAL): December 7, 2020

Study Record Updates

• Last Update Posted (ACTUAL): May 31, 2022
• Last Update Submitted That Met QC Criteria: May 25, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: MPS IIIB; Mucopolysaccharidosis IIIB; Sanfilippo B
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses; Mucopolysaccharidosis III
• Other Study ID Numbers: LTFU-ABO-101; 2019-002936-97 (EUDRACT_NUMBER)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: There is no plan to share data

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No