OTL-200 Long Term Insertional Oncogenesis Study

Status Authorised, recruitment pending · 5 EU/EEA countries · 5 sites · Protocol OTL-200-11

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)

Status Authorised, recruitment pending

Participants planned 35

Countries 5

Sites 5

Metachromatic leukodystrophy

The objective of this study is to monitor the long-term risk of malignancy due to insertional oncogenesis following treatment with OTL-200.

Key facts

Sponsor: Orchard Therapeutics (Europe) Limited
Participant type: Pediatric, Patients
Age range: 0-17 years, 18-64 years
Gender: Male and Female
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Decision date (initial): 2026-06-26
Transition trial: No
Low-intervention: Yes
Rare-disease indication: Yes
Vulnerable population: Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

The objective of this study is to monitor the long-term risk of malignancy due to insertional oncogenesis following treatment with OTL-200.

Conditions and MedDRA coding

Metachromatic leukodystrophy

Study design 1 period

#	Title	Allocation	Blinding	Roles blinded	Arms
1	Not applicable Not applicable	Not Applicable	None

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. The patient with early-onset MLD is enrolled in the LongTERM-MLD OTL-200-10 Study as part of Group 1 (treated with OTL-200 as part of CDP).
2. The patient or, if applicable, the patient’s parent(s)/legal guardian(s), are able and willing to provide written informed consent.

Exclusion criteria 1

There are no exclusion criteria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

Incidence of malignancies due to insertional oncogenesis reported during the study

Secondary endpoints 1

Incidence of clonal abundance ≥30% reported during the study.

Investigational products

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

Test 1

Libmeldy 2-10 x 10^6 cells/mL dispersion for infusion

PRD8611606 · Product

Active substance: Atidarsagene Autotemcel
Substance synonyms: GSK 2696274, OTL-200, AUTOLOGOUS CD34+ CELLS TRANSFECTED WITH LENTIVIRAL VECTOR CONTAINING THE HUMAN ARYLSULFATASE A CDNA, Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene, AUTOLOGOUS CD34+ CELL ENRICHED POPULATION THAT CONTAINS HSPC TRANSDUCED EX VIVO USING A LENTIVIRAL VECTOR ENCODING THE HUMAN ARSA GENE
Pharmaceutical form: DISPERSION FOR INFUSION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 30000000 kg kilogram(s)
Max total dose: 30000000 Kg kilogram(s)
Max treatment duration: 1 Day(s)
Authorisation status: Authorised
ATC code: A16AB21 — -
Marketing authorisation: EU/1/20/1493/001
MA holder: ORCHARD THERAPEUTICS (NETHERLANDS) B.V.
MA country: Iceland
Paediatric formulation: No
Orphan designation: Yes
Orphan designation number: EU/3/07/446
Modified vs. Marketing Authorisation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Orchard Therapeutics (Europe) Limited

Sponsor organisation: Orchard Therapeutics (Europe) Limited
Address: 245 Hammersmith Road
City: London
Postcode: W6 8PW
Country: United Kingdom

Scientific contact point

Organisation: Orchard Therapeutics (Europe) Limited
Contact name: Clinical

Public contact point

Organisation: Orchard Therapeutics (Europe) Limited
Contact name: Clinical

Third parties 2

Organisation	City, country	Duties
ProtaGene CGT GmbH ORG-100041450	Heidelberg, Germany	Laboratory analysis
Premier Research International LLC ORG-100054043	Morrisville, United States	On site monitoring, Code 12, Code 5, Data management

Locations

5 EU/EEA countries · 5 investigational sites