ELIVALDOGENE AUTOTEMCEL for Cerebral adrenoleukodystrophy (CALD)

Inclusion criteria

• {"criterion_text":"- Provision of written informed consent for this study by the subject or subject's parent(s)/ legal guardian(s) and written informed assent by subject, if applicable"}
• {"criterion_text":"- Have received Lenti-D Drug Product in a parent clinical study."}

Exclusion criteria

• {"criterion_text":"- There are no exclusion criteria for this Study."}

Primary endpoints

• {"endpoint_text":"- Major functional disability (MFD)-free survival over time through Year 15 post-drug product infusion. Note: interim analyses (including evaluation of MFD-free survival [i.e., subjects who were alive without an MFD or need for hematopoietic stem cell transplant [HSCT]; also referred to as event-free survival by FDA) will occur once all subjects have completed 5 years and 10 years of post infusion follow-up (see Section 7.6.1).","definition_or_measurement_approach":"MFD-free survival measured over time through Year 15 post-infusion; interim analyses planned once all subjects have completed 5 and 10 years of follow-up. MFD-free survival defined as subjects alive without an MFD or need for HSCT (event-free survival as per FDA)."}
• {"endpoint_text":"- The number of subjects with malignancies through 15 years post-drug product infusion","definition_or_measurement_approach":"Count of subjects who develop malignancies during the 15-year follow-up period after drug product infusion."}
• {"endpoint_text":"- The number of subjects who experience graft versus host disease (GVHD) through 15 years post-drug product infusion","definition_or_measurement_approach":"Count of subjects experiencing GVHD during the 15-year post-infusion follow-up period."}
• {"endpoint_text":"- The number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) through 15 years post drug product infusion","definition_or_measurement_approach":"Count of subjects with immune-related adverse events (including autoimmune disorders, GVHD, opportunistic infections, HIV) through 15 years post-infusion."}
• {"endpoint_text":"- The number of subjects with new or worsening hematologic disorders through 15 years post-drug product infusion","definition_or_measurement_approach":"Count of subjects with new or worsening hematologic disorders during the 15-year follow-up."}
• {"endpoint_text":"- The number of subjects with new or worsening neurologic disorders through 15 years post-drug product infusion","definition_or_measurement_approach":"Count of subjects with new or worsening neurologic disorders through 15 years post-infusion."}

Secondary endpoints

• {"endpoint_text":"- The number of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) through 15 years post-drug product infusion","definition_or_measurement_approach":"Count of subjects undergoing subsequent stem cell transplantation (second HSC infusion) during the 15-year follow-up."}
• {"endpoint_text":"- Change from Baseline (defined in parent study) through Year 15 post drug product infusion in neurologic function score (NFS)","definition_or_measurement_approach":"Change from baseline (baseline defined in the parent study) in Neurologic Function Score (NFS) measured through Year 15 post-infusion."}
• {"endpoint_text":"- The number of subjects without gadolinium enhancement (GdE) on MRI over time through Year 15 post-drug product infusion","definition_or_measurement_approach":"Count of subjects without gadolinium enhancement on MRI over time through Year 15 post-infusion."}

France

Earliest CTIS Part Ii Submission Date

19-07-2024

Latest Decision Or Authorization Date

28-04-2026

Processing Time Days

648

Number Of Sites

1

Number Of Participants

5

Germany

Earliest CTIS Part Ii Submission Date

19-07-2024

Latest Decision Or Authorization Date

29-04-2026

Processing Time Days

649

Number Of Sites

1

Number Of Participants

3

Italy

Earliest CTIS Part Ii Submission Date

19-07-2024

Latest Decision Or Authorization Date

30-04-2026

Processing Time Days

650

Number Of Sites

1

Number Of Participants

1

Netherlands

Earliest CTIS Part Ii Submission Date

19-07-2024

Latest Decision Or Authorization Date

05-05-2026

Processing Time Days

655

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Genetix Biotherapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

PPD Development LP

Responsibilities

Testing for ALD protein expression and vector copy number

Name

PPD International Holdings LLC

Responsibilities

Lab for European sites. Sample prep for ALDP, VCN, RCL, Short-term sample storage

Name

Pharmaceutical Product Development LLC

Responsibilities

Lab for kit supply (sample logistics, sample prep for VCN, ALDP, RCL, short-term sample storage)

Name

Perceptive Informatics Inc.

Responsibilities

Central MRI read

Name

Primevigilance Zagreb d.o.o.

Responsibilities

Pharmacovigilance / safety reporting (role code 8)

Third parties

• {"country":"Switzerland","full_name":"Voisin Consulting CH SARL","duties_or_roles":"12","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"Patient Reimbursement Services for Travel and Stipends","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Perceptive Informatics Inc.","duties_or_roles":"Central MRI read","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Testing for ALD protein expression and vector copy number","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Lab for European sites. Sample prep for ALDP, VCN, RCL, Short-term sample storage","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"ProtaGene CGT GmbH","duties_or_roles":"Specialty lab for ISA","organisation_type":"Pharmaceutical company"}
• {"country":"Croatia","full_name":"Primevigilance Zagreb d.o.o.","duties_or_roles":"8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Genezen Laboratories Inc.","duties_or_roles":"RCL testing","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Lab for kit supply (sample logistics, sample prep for VCN, ALDP, RCL, short-term sample storage)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Multi-Regional Clinical Trials Center Of Brigham And Women's Hospital And Harvard","duties_or_roles":"Bone Marrow NGS","organisation_type":"Pharmaceutical company"}