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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Genetix Biotherapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

11 Mar 2020 → ongoing

Decision date (initial)

2024-07-29

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-513904-33-00

EudraCT number

2015-002805-13

ClinicalTrials.gov

NCT02698579

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacogenetic, Safety

- Monitor for long-term safety of the Lenti-D Drug Product (also known as elivaldogene autotemcel; hereafter referred to as eli-cel ) administered in parent clinical studies.
- Monitor for long-term efficacy of eli cel administered in parent clinical studies.

Secondary objectives 1

1. Not applicable

Conditions and MedDRA coding

Cerebral Adrenoleukodystrophy (CALD)

Regulatory references

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

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 Lenti-D Drug Product in a parent clinical study.

Exclusion criteria 1

1. There are no exclusion criteria for this Study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 6

1. 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).
2. The number of subjects with malignancies through 15 years post-drug product infusion
3. The number of subjects who experience graft versus host disease (GVHD) through 15 years post-drug product infusion
4. The number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) through 15 years post drug product infusion
5. The number of subjects with new or worsening hematologic disorders through 15 years post-drug product infusion
6. The number of subjects with new or worsening neurologic disorders through 15 years post-drug product infusion

Secondary endpoints 3

1. The number of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) through 15 years post-drug product infusion
2. Change from Baseline (defined in parent study) through Year 15 post drug product infusion in neurologic function score (NFS)
3. The number of subjects without gadolinium enhancement (GdE) on MRI over time through Year 15 post-drug product infusion

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Genetix Biotherapeutics Inc.

Sponsor organisation

Genetix Biotherapeutics Inc.

Address

455 Grand Union Boulevard

City

Somerville

Postcode

02145-1446

Country

United States

Scientific contact point

Organisation

Bluebird Bio Inc.

Contact name

Clinical Trial Information

Public contact point

Organisation

Bluebird Bio Inc.

Contact name

Clinical Trial Information

Third parties 10

Locations

4 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Documents 29 files

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

Application history

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