null - FindTrial

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 - not applicable

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-002611-26-ES

Enrollment

308

Registered

2020-02-14

Start date

2020-02-28

Completion date

Unknown

Last updated

2021-08-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal Muscular Atrophy

Interventions

Product Name: AVXS-101 Product Code: AVXS-101 Pharmaceutical Form: Infusion INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC CAS Number: 1922968-73-7 Current Sponsor code: AVXS-101 Concentration unit: Ot

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: > Patients with SMA (with 1, 2 or 3copies of SMN2) who received AVXS-101 gene replacement therapy in an AveXis clinical study > Patient/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule Are the trial subjects under 18? yes Number of subjects for this age range: 308 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 64 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study

Design outcomes

Primary

Measure	Time frame
Main Objective: To collect long-term follow-up safety and efficacy data in patients with spinal muscular atrophy (SMA) Type 1, Type 2 or Type 3 who were treated with AVXS-101 in an AVXS-101 clinical trial, including but not limited to AVXS-101-CL-102 (Phase 1), AVXS-101-CL-302 (Phase 3), AVXS-101-CL-303 (Phase 3), AVXS-101-CL-304 (Phase 3) or AVXS-101-CL-306 (Phase 3).;Secondary Objective: not applicable;Primary end point(s): 1. Number of participants who reach developmental milestones 2. Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score 3. Number of participants who experience a clinically significant change from baseline in pulmonary assessment results 4. Number of participants who experience swallowing dysfunction 5. Number of participants who experience a clinically significant change from baseline in physical examination findings 6. Number of participants who experience a clinically significant change from baseline in vital signs measurements 7. Change from baseline in height measurements 8. Change from baseline in weight measurements 9. Number of participants who experience a clinically significant change from baseline in clinical laboratory assessments 10. Number of participants who experience a clinically significant change from baseline in cardiac assessments 11. Number of participants who experience a clinically significant change from baseline in observational phase questionnaire results 12. Number of participants who experience at least one serious adverse event (SAE) 13. Number of participants who experience at least one adverse event of special interest (AESI);Timepoint(s) of evaluation of this end point: 1. Up to 5 years 2. Up to 2 years 3. Up to 15 years 4. Up to 5 years 5. Up to 5 years 6. Up to 5 years 7. Up to 5 years 8. Up to 5 years 9. Up to 5 years 10. Up to 5 years 11. Year 6 to Year 15 12. Up to 15 years 13. Up to 15 years	—

Measure

Time frame

Main Objective: To collect long-term follow-up safety and efficacy data in patients with spinal muscular atrophy (SMA) Type 1, Type 2 or Type 3 who were treated with AVXS-101 in an AVXS-101 clinical trial, including but not limited to AVXS-101-CL-102 (Phase 1), AVXS-101-CL-302 (Phase 3), AVXS-101-CL-303 (Phase 3), AVXS-101-CL-304 (Phase 3) or AVXS-101-CL-306 (Phase 3).;Secondary Objective: not applicable;Primary end point(s): 1. Number of participants who reach developmental milestones 2. Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score 3. Number of participants who experience a clinically significant change from baseline in pulmonary assessment results 4. Number of participants who experience swallowing dysfunction 5. Number of participants who experience a clinically significant change from baseline in physical examination findings 6. Number of participants who experience a clinically significant change from baseline in vital signs measurements 7. Change from baseline in height measurements 8. Change from baseline in weight measurements 9. Number of participants who experience a clinically significant change from baseline in clinical laboratory assessments 10. Number of participants who experience a clinically significant change from baseline in cardiac assessments 11. Number of participants who experience a clinically significant change from baseline in observational phase questionnaire results 12. Number of participants who experience at least one serious adverse event (SAE) 13. Number of participants who experience at least one adverse event of special interest (AESI);Timepoint(s) of evaluation of this end point: 1. Up to 5 years 2. Up to 2 years 3. Up to 15 years 4. Up to 5 years 5. Up to 5 years 6. Up to 5 years 7. Up to 5 years 8. Up to 5 years 9. Up to 5 years 10. Up to 5 years 11. Year 6 to Year 15 12. Up to 15 years 13. Up to 15 years

—

Secondary

Measure	Time frame
Secondary end point(s): not applicable;Timepoint(s) of evaluation of this end point: not applicable	—

Countries

Australia, Belgium, Canada, France, Italy, Japan, Korea, Republic of, Spain, Taiwan, United Kingdom, United States

Contacts

Public ContactSr. Manager Clin. Trial Operations

AveXis, Inc.

AValluru979@avexis.com001312667 8118

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026