Clinical trial NCT04655911: A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101
Official title: 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)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Mucopolysaccharidosis III-B
Keywords: MPS IIIB
Mucopolysaccharidosis IIIB
Sanfilippo B
Lead sponsor: Abeona Therapeutics, Inc
Sponsor class: INDUSTRY
First posted: 2020-12-07
Enrollment: 1
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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).

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.

Eligibility criteria: 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

Primary outcomes:
measure: Adverse Events
timeFrame: 60 months
Secondary outcomes:
measure: Age Equivalent Compared to Natural History Study Data
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Developmental Quotient Compared to Natural History Study Data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Cognitive Age Equivalent Compared to Natural History Study Data
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: 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
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Adaptive Age Equivalent Compared to Natural History Study Data
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Pediatric Quality of Life lnventory (PedsQL ™)
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Parenting Stress lndex, 4th Edition (PSl-4)
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Parental Global lmpression Scale
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Clinical Global lmpression lmprovement Scale
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: Parent Symptom Scale Questionnaire
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
measure: T-cell responses against the AAV9 capsid
timeFrame: 60 Months
measure: T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product
timeFrame: 60 Months
measure: Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product
timeFrame: 60 Months
measure: Antibody formation (humoral) against the AAV9 capsid
timeFrame: 60 Months
measure: Viral Load
timeFrame: AII study visits (30, 36, 42, 48, and 60 Months)
Other outcomes:
measure: Sanfilippo Behavior Rating Scale
timeFrame: 30, 36, 42, 48, and 60 Months
measure: Children's Sleep Habits Questionnaire (CSHQ)
timeFrame: 30, 36, 42, 48, and 60 Months
measure: Neutralizing antibody against the AAV9 capsid
timeFrame: 60 Months
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Paris
country: France
facility: Armand-Trousseau Hospital
city: Hamburg
country: Germany
facility: University Hospital Hamburg-Eppendorf
Study officials:
References: