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

Mucopolysaccharidosis III-B Clinical Trial

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04655911
• Other study ID #: LTFU-ABO-101
• Secondary ID: 2019-002936-97
• Status: Terminated
• Start date: October 28, 2020
• Est. completion date: April 7, 2022

Study information

• Verified date: May 2022
• Source: Abeona Therapeutics, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: April 7, 2022
• Est. primary completion date: April 7, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• Mucopolysaccharidosis III-B

Intervention

Biological:
• ABO-101
Gene therapy rAAV9.CMV.hNAGLU

Locations

Country	Name	City	State
France	Armand-Trousseau Hospital	Paris
Germany	University Hospital Hamburg-Eppendorf	Hamburg
United States	Nationwide Children's Hospital	Columbus	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Abeona Therapeutics, Inc

Countries where clinical trial is conducted

United States,  France,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	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
Other	Neutralizing antibody against the AAV9 capsid	Long-term immunological responses defined as neutralizing antibody formation against the AAV9 capsid	60 Months
Primary	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	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	T-cell responses against the AAV9 capsid	Long-term immunological responses defined as T-cell responses against the AAV9 capsid	60 Months
Secondary	T-cell responses against the a-N- acetylglucosaminidase (NAGLU) transgene product	Long-term immunological responses defined as T-cell responses against the a-N- acetylglucosaminidase transgene product.	60 Months
Secondary	Antibody formation (humoral) against the a-N- acetylglucosaminidase (NAGLU) transgene product	Long-term immunological responses defined as antibody formation (humoral) against the a-N- acetylglucosaminidase (NAGLU)	60 Months
Secondary	Antibody formation (humoral) against the AAV9 capsid	Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.	60 Months
Secondary	Viral Load	Long-term viral load, in applicable cases.	AII study visits (30, 36, 42, 48, and 60 Months)