A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

2023-510392-66-00 Protocol LTFU-ABO-102 Therapeutic confirmatory (Phase III) Ongoing, recruiting

Start 23 Nov 2020 · Status Ongoing, recruiting · 1 EU/EEA countries · 3 sites · Protocol LTFU-ABO-102

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Ongoing, recruiting
Participants planned 41
Countries 1
Sites 3

Mucopolysaccharidosis type IIIA

To evaluate the long-term safety/tolerability of UX111 in patients with MPS IIIA

Key facts

Sponsor
Ultragenyx Pharmaceutical Inc.
Participant type
Pediatric, Patients
Age range
0-17 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trial duration
23 Nov 2020 → ongoing
Decision date (initial)
2024-02-07
Transition trial
Yes
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
Yes
Funding sources
Ultragenyx Pharmaceutical Inc

External identifiers

EU CT number
2023-510392-66-00
EudraCT number
2019-002979-34
ClinicalTrials.gov
NCT04360265

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Others, Safety

To evaluate the long-term safety/tolerability of UX111 in patients with MPS IIIA

Secondary objectives 1

  1. (No secondary objectives)

Conditions and MedDRA coding

Mucopolysaccharidosis type IIIA

VersionLevelCodeTermSystem organ class
20.1 PT 10056890 Mucopolysaccharidosis III 100000004850

Study design 1 period

#TitleAllocationBlindingRoles blindedArms
1 long-term follow-up (LTFU)
Multicenter, non-interventional, long-term follow-up (LTFU) study in participants from prior interventional trials involving the administration of UX111 (Studies ABT-001 and ABT-003).
 Not Applicable None

Regulatory references

Scientific advice from competent authorities
The Spanish Agency Of Medicines And Medical Devices
Plan to share IPD
No
IPD plan description
N/A
EU CT numberTitleSponsor
2023-510032-37-00 Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Ultragenyx Pharmaceutical Inc.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

  1. Participants that have participated in a prior clinical trial in which they received UX111
  2. 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 2

  1. Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study
  2. Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

  1. Safety: Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs)
  2. Efficacy: Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive raw score

Secondary endpoints 6

  1. Cerebrospinal fluid (CSF) heparan sulfate (HS) (disaccharide) exposure
  2. CSF HS percentage change from baseline in prior trial
  3. BSITD-III Receptive Communication raw score
  4. BSITD-III Expressive Communication raw score
  5. Total cortical volume (cm 3) annualized percentage change from baseline in prior trial
  6. Survival

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

ABO-102

PRD10602523 · Product

Active substance
Rebisufligene Etisparvovec
Other product name
scAAV9.U1a.hSGSH; UX111
Pharmaceutical form
SUSPENSION FOR INJECTION
Route of administration
INTRAVENOUS USE
Max daily dose
0 vector genomes (vg)/mL
Max total dose
0 vector genomes (vg)/mL
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
ULTRAGENYX PHARMACEUTICAL INC.
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/16/176

ABO-102

PRD10602521 · Product

Active substance
Rebisufligene Etisparvovec
Other product name
scAAV9.U1a.hSGSH; UX111
Pharmaceutical form
SUSPENSION FOR INJECTION
Route of administration
INTRAVENOUS USE
Max daily dose
0 vector genomes (vg)/mL
Max total dose
0 vector genomes (vg)/mL
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
ULTRAGENYX PHARMACEUTICAL INC.
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/16/176

ABO-102

PRD10602522 · Product

Active substance
Rebisufligene Etisparvovec
Other product name
scAAV9.U1a.hSGSH; UX111
Pharmaceutical form
SUSPENSION FOR INJECTION
Route of administration
INTRAVENOUS USE
Max daily dose
0 vector genomes (vg)/mL
Max total dose
0 vector genomes (vg)/mL
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
ULTRAGENYX PHARMACEUTICAL INC.
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/16/176

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ultragenyx Pharmaceutical Inc.

14 Total trials 8 Recruiting 6 Ended
Commercial
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Address
60 Leveroni Court Suite 200
City
Novato
Postcode
94949-5746
Country
United States

Scientific contact point

Organisation
Ultragenyx Pharmaceutical Inc.
Contact name
Medical Information

Public contact point

Organisation
Ultragenyx Pharmaceutical Inc.
Contact name
Medical Information

Third parties 15

OrganisationCity, countryDuties
Bioagilytix Labs LLC
ORG-100013030
 Durham, United States Other
Patient Primary
ORL-000005197
 Bracknell, Berkshire, United Kingdom Other
MARKEN Germany GmbH
ORG-100017196
 Hamburg, Germany Other
Pharmapace Inc.
ORG-100048736
 San Diego, United States Data management, E-data capture
Sa Pathology
ORG-100044405
 Adelaide, Australia Laboratory analysis
Marken LLP
ORG-100048834
 Durham, United States Other
Intelerad (Ambra)
ORL-000015185
 Montreal, Canada Other
Fortrea Inc.
ORG-100012602
 Durham, United States Other
Trulab
ORL-000001153
 Durham, United States Other
Quantims, LLC (Igor Nestrasil)
ORL-000015187
 St. Anthony, United States Other
EPL Archives, LLC
ORL-000014844
 Leesburg, United States Other
Lumanity Patient Centered Outcomes LLC
ORG-100044473
 Boston, United States Other
Primevigilance Limited
ORG-100027742
 Guildford, United Kingdom Other
Life Courier (fka Optimize Courier)
ORL-000015186
 Venice, United States Other
DF/Net Research
ORL-000015184
 Seattle, United States Data management

Locations

1 EU/EEA country · 3 investigational sites

By country

CountryMS statusPlanned subjectsSites
Spain Ongoing, recruiting 24 3
Rest of world
Australia, United States
 — 17 —

Investigational sites

Spain

3 sites · Ongoing, recruiting
Complexo Hospitalario Universitario De Santiago
Neurology, Calle Choupana Da S/n, 15706, Santiago De Compostela
Hospital Universitari Vall D Hebron
Pediatrics, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Complexo Hospitalario Universitario De Santiago
Pediatrics, Calle Choupana Da S/n, 15706, Santiago De Compostela

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Spain 2020-11-23 2020-11-23

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 6 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol_2023-510392-66_For Publication 6.0
Recruitment arrangements (for publication) K1_Recruitment arrangements 1
Subject information and informed consent form (for publication) L1_SIS and ICF_Site 002 CHUS_ICF_For Publication 9.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Site 008 VAL_ICF_For Publication 3.0
Synopsis of the protocol (for publication) D1_Protocol synopsis_EN_2023-510392-66 6.0
Synopsis of the protocol (for publication) D1_Protocol synopsis_ES 2023-510392-66 6.0

Application history

4 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-02-01 Spain Acceptable
2024-02-07
 2024-02-07
2 SUBSTANTIAL MODIFICATION SM-1 2024-09-25 Spain Acceptable
2024-11-06
 2024-11-06
3 SUBSTANTIAL MODIFICATION SM-2 2024-11-27 Spain Acceptable
2025-01-22
 2025-01-22
4 SUBSTANTIAL MODIFICATION SM-3 2025-07-23 Spain Acceptable
2025-10-15
 2025-10-20

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