Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product - LTF-304

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON54368

Enrollment

Registered

2021-04-28

Start date

2022-02-26

Completion date

Unknown

Last updated

2024-09-16

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

Conditions

CALD cerebral adrenoleukodystrophy

Interventions

Not applicable as no investigational medicinal product (IMP) will be administered to the patients.

Eligibility

Age

2 Years to 64 Years

Inclusion criteria

Inclusion criteria: 1.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 2.Have received eli-cel in a parent clinical study 3.Able to comply with study requirements

Exclusion criteria

Exclusion criteria: There are no exclusion criteria for this Study.

Design outcomes

Primary

Measure	Time frame
1. Safety Endpoints include: - Proportion of subjects who experience graft versus host disease (GVHD) - Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) - All drug product-related AEs through 15 years post-drug product infusion - All serious adverse events (SAEs) through 15 years post-drug product infusion (regardless of relatedness to drug product) - Immune-related AEs and new or worsening hematologic or neurologic disorders or malignancies through 15 years post-drug product infusion - Incidence of vector-derived RCL, assessed from archived samples as clinically indicated. - The number of subjects with insertional oncogenesis (myelodysplasia, leukemia, lymphoma, etc.) - The number of subjects with persistent oligoclonality 2. Efficacy Endpoints include: -MFD-free survival	—

Measure

Time frame

1. Safety Endpoints include: - Proportion of subjects who experience graft versus host disease (GVHD) - Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) - All drug product-related AEs through 15 years post-drug product infusion - All serious adverse events (SAEs) through 15 years post-drug product infusion (regardless of relatedness to drug product) - Immune-related AEs and new or worsening hematologic or neurologic disorders or malignancies through 15 years post-drug product infusion - Incidence of vector-derived RCL, assessed from archived samples as clinically indicated. - The number of subjects with insertional oncogenesis (myelodysplasia, leukemia, lymphoma, etc.) - The number of subjects with persistent oligoclonality 2. Efficacy Endpoints include: -MFD-free survival

—

Secondary

Measure	Time frame
Secondary efficacy endpoints include the following: - Overall survival. - Change from Baseline (defined in parent study) in NFS. - Gadolinium enhancement (GdE) status.	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)