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

NCT04655911 | Terminated DMC FDA Drug

• 2 other identifiers: LTFU-ABO-1012019-002936-97
• Study Type: observational
• Target: 1
• Locations: 3 countries
• Sites: 3

Brief Summary

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).

Conditions

Mucopolysaccharidosis III-B

Keywords

MPS IIIB; Mucopolysaccharidosis IIIB; Sanfilippo B

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (15)

• Age Equivalent Compared to Natural History Study Data

  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

  AII study visits (30, 36, 42, 48, and 60 Months)

• Cognitive Age Equivalent Compared to Natural History Study Data

  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

  AII study visits (30, 36, 42, 48, and 60 Months)

• Adaptive Age Equivalent Compared to Natural History Study Data

  AII study visits (30, 36, 42, 48, and 60 Months)

Other Outcomes (3)

• Sanfilippo Behavior Rating Scale

  30, 36, 42, 48, and 60 Months

• Children's Sleep Habits Questionnaire (CSHQ)

  30, 36, 42, 48, and 60 Months

• Neutralizing antibody against the AAV9 capsid

  60 Months

Study Arms (1)

ABO-101

Participants from prior interventional trials involving the administration of ABO-101.

Biological: ABO-101

Interventions

ABO-101 BIOLOGICAL

Gene therapy rAAV9.CMV.hNAGLU

ABO-101

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Abeona Therapeutics, Inc: lead

Study Sites (3)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Armand-Trousseau Hospital

Paris, 75571, France

Location

University Hospital Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

MeSH Terms

Conditions

Mucopolysaccharidosis III

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2020
• First Posted: December 7, 2020
• Study Start: October 28, 2020
• Primary Completion: April 7, 2022
• Study Completion: April 7, 2022
• Last Updated: May 31, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); US (1); DE (1)