International study for children and adolescents with standard risk ALK-positive anaplastic large cell lymphoma (ALCL) estimating the efficacy of Vinblastine

Overview

Sponsor-declared trial summary

Key facts

Sponsor

GPOH gGmbH

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

5 Feb 2020 → ongoing

Decision date (initial)

2023-03-01

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Deutsche Kinderkrebsstiftung

External identifiers

EU CT number

2022-501454-11-00

EudraCT number

2017-002935-40

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

To show that it is possible to cure at least 75% of patients belonging to the SR group with Vinblastine-monotherapy for 24 months.

Secondary objectives 5

1. To describe overall survival and treatment related mortality of 24 months Vinblastine monotherapy. Overall survival is the time from diagnosis to death of any cause or last follow-up.
2. To identify clinical, pathological and biological factors predictive of progressive disease during / after VBL therapy. The time of progressive disease is the time from diagnosis to progressive disease or a competing event (death from any cause, secondary malignancy) or last follow-up.
3. To estimate the rate of SR patients requiring multi-agent chemotherapy
4. To describe the toxicity of Vinblastine given for 24 months rated with CTCAE v4.03.
5. To describe the response after 3 weeks (between day 17-22), 3 months and 6 months of treatment (including a possible pre-phase) assessed by appropriate imaging methods

Conditions and MedDRA coding

standard risk ALK-positive anaplastic large cell lymphoma (ALCL)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Stratification into the standard risk group (SR) by screening: Newly diagnosed ALK-positive ALCL, Stage I not completely resected, or stage II or stage III, MDD negative
2. Age < 18 years
3. Informed consent of the parents/legal guardians (and assent of the competent child) for study participation and data collection, storage and handling given before study entry
4. Participation in national / study group's reference pathology
5. Follow-up for at least 3 years after enrolment is expected
6. Application of a highly effective contraceptive method (Pearl index <1) in sexually active patients
7. Application of one intrathecal triple therapy with Methotrexate, Cytarabine and Prednisolone (or Hydrocortisone respectively) before start of the protocol treatment

Exclusion criteria 6

1. Progressive disease during a possible clinically indicated pre-phase treatment before inclusion in the study
2. Steroids for more than 2 days or chemotherapy pre-treatment before taking the screening sample for MDD
3. Chemotherapy pre-treatment before start of the study treatment except for: the obligatory initial intrathecal triple therapy with Methotrexate, Cytarabine and Prednisolone (or Hydrocortisone respectively), a possible clinically indicated pre-phase including up to 5 days of steroids combined with up to 3 doses of Vinblastine (and up to 2 doses of Cyclophosphamide)
4. Pregnancy or lactation period
5. Contraindications for the treatment with Vinblastine: hypersensitivity against VBL or other vinca-alkaloids, leukopenia, other than in the context of the ALCL, severe uncontrolled infection
6. Other medical, psychiatric, familial or social condition prohibiting treatment according to the protocol

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Probability of event-free-survival (pEFS) at 3 years, with EFS defined as the time of diagnosis to the first event (progressive disease, nonresponse, secondary malignancy or death due to any cause) or last follow-up.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

GPOH gGmbH

Sponsor organisation

GPOH gGmbH

Address

Holsterhauser Platz 2, Holsterhausen Holsterhausen

City

Essen

Postcode

45147

Country

Germany

Scientific contact point

Organisation

GPOH gGmbH

Contact name

Prof. Dr. Wilhelm Wößmann

Public contact point

Organisation

GPOH gGmbH

Contact name

Prof. Dr. Wilhelm Wößmann

Third parties 2

Locations

8 EU/EEA countries · 106 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 73 files

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

Application history

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