Mucopolysaccharidosis III-BNCT04655911
A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101
An observational study of Mucopolysaccharidosis III-B, run by Abeona Therapeutics, Inc at 3 sites in 3 countries.
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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis III-B
- What is being tested?
- ABO-101
- How many people
- 1 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 28 Oct 2020to 7 Apr 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
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)
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).
Read the 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.
- Conditions
- Mucopolysaccharidosis III-B
- Being tested
- ABO-101
- Keywords
- MPS IIIB, Mucopolysaccharidosis IIIB, Sanfilippo B
- Medical subject headings
- Mucopolysaccharidosis III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABO-101Biological
Gene therapy rAAV9.CMV.hNAGLU
| Study group | Role | What they receive |
|---|---|---|
| ABO-101 | Participants from prior interventional trials involving the administration of ABO-101. Receives: Biological: ABO-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: 60 months
Secondary outcomes (15)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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)
Measured: 60 Months
Antibody formation (humoral) against the AAV9 capsid
Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.
Measured: 60 Months
Viral Load
Long-term viral load, in applicable cases.
Measured: AII study visits (30, 36, 42, 48, and 60 Months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2020
- Study start28 Oct 2020
- Primary completion7 Apr 2022
- Study completion7 Apr 2022
Why it stopped: Abeona 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
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis III-BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abeona Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04655911
- Sponsor's protocol ID
- LTFU-ABO-101
- Other IDs
- 2019-002936-97, 2019
- Status last verified
- May 2022
- First submitted
- 17 Nov 2020
- Last updated
- 31 May 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan to share data
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04655911
- Acronym / sponsor ID
- LTFU-ABO-101
- Conditions
- Mucopolysaccharidosis III-B
- Keywords
- MPS IIIB, Mucopolysaccharidosis IIIB, Sanfilippo B
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Abeona 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
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Abeona Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 28 Oct 2020 (actual)
- Primary completion
- 7 Apr 2022 (actual)
- Study completion
- 7 Apr 2022 (actual)
- First posted
- 7 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ABO-101 | – | Biological: ABO-101Participants from prior interventional trials involving the administration of ABO-101. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABO-101Gene therapy rAAV9.CMV.hNAGLU | Biological | ABO-101 |
Primary outcome
Adverse Events
Time frame: 60 months
Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
15 secondary outcomes
Age Equivalent Compared to Natural History Study Data
Time frame: 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
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Cognitive Age Equivalent Compared to Natural History Study Data
Time frame: 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
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Adaptive Age Equivalent Compared to Natural History Study Data
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Pediatric Quality of Life lnventory (PedsQL ™)
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Parenting Stress lndex, 4th Edition (PSl-4)
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Parental Global lmpression Scale
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Clinical Global lmpression lmprovement Scale
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Parent Symptom Scale Questionnaire
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
T-cell responses against the AAV9 capsid
Time frame: 60 Months
T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product
Time frame: 60 Months
Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product
Time frame: 60 Months
Antibody formation (humoral) against the AAV9 capsid
Time frame: 60 Months
Viral Load
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Results and safety
The study was stopped early (reason given: Abeona 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). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2022.
- Individual participant dataSharing planned: No. There is no plan to share data
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis III-B, run by Abeona Therapeutics, Inc at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 31 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, France, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Abeona 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). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: 60 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 May 2022; AzTrials last refreshed its copy on 7 Nov 2026.