Expanded access program for mepolizumab in subjects with hypereosinophilic syndrome.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Glaxosmithkline Research & Development Limited

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Not possible to specify, Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

29 Mar 2019 → 16 Mar 2026

Decision date (initial)

2024-07-30

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-515247-28-00

EudraCT number

2007-000838-39

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To provide expanded access for subjects that have a clear medical need and in whom the benefit to risk ratio is thought to be appropriate. It must be noted that expanded access is only permitted by regulatory authorities is some but not all countries. GSK does not solicit requests for expanded access from investigators or subjects. An investigator administering mepolizumab through expanded access is responsible for the administration and management of the supply, and for the subject's welfare. Accordingly, the investigator must refer to the Investigator's Brochure (IB) prepared for the ongoing clinical trials for mepolizumab which contain a comprehensive range of information on the drug, and then use his or her clinical judgment as to whether the drug is suitable for the relevant subject.

Conditions and MedDRA coding

Hypereosinophilic Syndrome

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. In accordance with local procedures, written informed consent/assent can be obtained from the subject or legally authorized representative.
2. >/= 12 years of age at the time of signing the informed consent/assent
3. "Meets the diagnostic criteria for HES as defined by: Eosinophilia >1500 cells/mcl for a least 6 months with evidence of symptoms and signs of organ system involvement or dysfunction that can be directly related to eosinophilia (with no evidence of parasitic, allergic or other recognized causes of eosinophilia such as connective tissue disease, malignancy) or Eosinophilia >1500mcl for less than 6 months and meet the other criteria for HES accompanied by clear evidence of eosinophil tissue infiltration and with exclusion of secondary causes of eosinophilia as above"
4. "Subjects meeting all three of the following criteria will be eligible: The indication, HES, is a seriously debilitating or life-threatening disease; There is no satisfactory alternative treatment: documented failure (lack of efficacy or a contra-indication) to at least 3 standard therapies (corticosteroids, cytotoxic agents, immunomodulatory therapy, and Imatinib mesylate) at the appropriate duration and dose or demonstrated clinical benefit from prior treatment with mepolizumab; and There is reason to believe that the benefit:risk ratio for mepolizumab in the indication is positive"

Exclusion criteria 8

1. Subjects without HES but with other conditions associated with eosinophilic pathological processes such as Eosinophilic Granulomatosis with Polyangitis [EGPA], Wegener's Granulomatosis, atopic disorders, parasitic infections, eosinophilic gastroenteropathies.
2. "Female subjects of childbearing potential who are not using a highly effective method of contraception: Consistent and correct use of an acceptable method of birth control for one month prior to the start of the investigational product and until 16 weeks after the last dose."
3. Pregnancy or lactating females
4. Subjects with severe/life-threatening underlying disease unrelated to HES where life expectancy is estimate to be less than 3 months
5. "Subjects with a history of or a current malignancy: Subjects with a history of or current lymphoma Subjects with current malignancy or previous history of malignancy in remission for less than 12 months prior to the first dose. Subjects that had localized carcinoma (i.e., basal or squamous cell) of the skin which was resected for cure will not be excluded."
6. Subject with history of serious allergic reaction (hypersensitivity/anaphylaxis) to anti-IL5 or other antibody therapy or known or suspected hypersensitivity to any component of mepolizumab, leading to treatment discontinuation.
7. Subjects with current drug or alcohol abuse where uncertain compliance with the protocol and/or with the medical management instruction of the investigator may cause safety risk.
8. Subjects who have received treatment with an investigational agent (biologic or non-biologic, excluding mepolizumab) within the past 30 days or 5 drug half-lives whichever is longer, prior to the administration of mepolizumab under this protocol. The term 'investigational' applies to any drug not approved for sale in the country in which it is being used or investigation formulations of marketed products.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. "SAEs Non-serious AEs related to mepolizumab as assessed by the Investigator Mean 28-day SC dose (mg) for the last 3 administrations"

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Glaxosmithkline Research & Development Limited

Sponsor organisation

Glaxosmithkline Research & Development Limited

Address

G S K House, 980 Great West Road 980 Great West Road

City

Brentford

Postcode

TW8 9GS

Country

United Kingdom

Scientific contact point

Organisation

Glaxosmithkline Research & Development Limited

Contact name

EU GSK Clinical Trials Call Center

Public contact point

Organisation

Glaxosmithkline Research & Development Limited

Contact name

EU GSK Clinical Trials Call Center

Third parties 2

Locations

1 EU/EEA country · 1 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 9 files

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

Application history

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