Clinical trial NCT05393024: Patient With MMRR Treated With Belantamab Mafotidine on Monotherapy
Official title: Observational Study in Patient With Multiple Myeloma Relapse/Refractory Treated With Belantamab Mafotidine on Monotherapy Previously Exposed to at Least One Proteasome Inhibitor, Immunomodulatory Agent, and Anti-CD38 Antibody.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Myeloma
Keywords: Mulitple Myeloma
Belantamab Mafoditin
EAP
Lead sponsor: Fondazione EMN Italy Onlus
Sponsor class: OTHER
First posted: 2022-05-26
Enrollment: 78
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Male and female patients with MMRR previously treated with at least one proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Brief summary: This is a retrospective/prospective observational study evaluating the efficacy and safety of Belantamab Mafotidin as a single agent in patients with Multiple Myeloma Relapse/Refractory (MMRR) treated in clinical pratice under compassionate use

Detailed description: Multiple myeloma (MM) is an incurable disease that accounts for 1% of all cancers and 10% of all haematological malignancies, most patients with MM develop resistance to existing therapies at the time of disease recurrence.

Belantamab mafodotin is a new humanized antibody-drug conjugate (IgG1) that is under development for the treatment of MM and has demonstrated a manageable safety profile and positive clinical activity in patients with relapsed or refractory multiple myeloma (MMRR) heavily pretreated.

The objective of this retro-prospective observational study is: to evaluate clinical efficacy as the percentage of patients who have achieved a clinical benefit (minimum or best response), ORR, DoR, PFS, OS; evaluate the safety profile of patients treated with Belantamab Mafodotin as monotherapy in clinical practice.

All patients included in this analysis were treated or are still receiving Belantamab Mafodotin monotherapy under the compassionate use programs (nominal program-NPP and the extended access program-EAP).

Eligibility criteria: Inclusion Criteria:

1. Written informed consent may be obtained from the patient or legally authorized representative according to local regulations (patients who have already died may also be included)
2. Histologically or cytologically confirmed diagnosis of MM as defined according to IMWG criteria of 2016 and:

   1. patient has undergone stem cells transplantation or is considered ineligible for transplantation, and
   2. patient has received at least four therapies
   3. patient is refractory to an anti-CD38 antibody (ex., daratumumab) alone or in combination, and to an IMiD (ex., lenalidomide or pomalidomide), and to a proteasome inhibitor (ex. bortezomib, ixazomib or carfilzomib).
3. Male or female equal and/or upper 18 years (at baseline)
4. Performance Status at baseline by ECOG scale 0-2
5. Adequate organ system functions at baseline
6. Female patients: a female patient is elegible if she is not pregnant or breastfeeding and at least one of the following conditions applies:

   * She is/was not a woman of childbearing potential (WOCBP) OR
   * She is/was using an highly effective contrapcetive method during the treatment period and at least 9 months after the last dose and she agrees not to donate eggs (ova, oocytes) for the purpose of reproduction during this period.
   * Highly sensitive negative serum pregnancy test within 72 hours of therapy (C1D1) and agreed to use effective contraception during the treatment period and for the next 9 months after the last dose of the drug.

   Male patient: male patient were/are elegible if agreed to follow from the first dose until the last dose of treatment to allow clearance of any altered sperm:
   * abstaining from sperm donation PLUS
   * abstaing from heterosexual relationship in accordance with one's preferred and habitual lifestyle (long-term and persistent abstinent) and agreed/accepted to remain abstinent OR
   * agree/agreed to use contraption as described below: agree to use male condom even though they have/had succesfully vasectomy and female partner uses/used an additional highly effective contraceptive method.
7. All toxicities related to previous treatment (defined by National Cancer Institute- Common Toxicity Criteria for Adverse Events (NCI-CTCAE) were Grade 1 or less at t at the time of treatment initiation within compassionate use programs, except alopecia and neuropathy grade 2.

Exclusion Criteria:

* The patients are/were not elegible for compassionate use programs (NPP, EAP)

Primary outcomes:
measure: Best response or minimal response
timeFrame: 1 year
Secondary outcomes:
measure: Overall Response Rate (ORR)
timeFrame: 1 year
measure: Progression Free Survival (PFS)
timeFrame: 1 year
measure: Duration of Response (DoR)
timeFrame: 1 year
measure: Overall Survival
timeFrame: 1 year
Other outcomes:
Locations:
city: Ancona
country: Italy
facility: AOU Ospedali Riuniti Umberto I
city: Bologna
country: Italy
facility: Policlinico Sant'Orsola Malpighi, Aou Di Bologna
city: Brescia
country: Italy
facility: A.O. Spedali Civili di Brescia
city: Cagliari
country: Italy
facility: Ospedale "A. Businco"
city: Catania
country: Italy
facility: AOU Policlinico Vittorio Emanuele
city: Ferrara
country: Italy
facility: A.O.U. Arcispedale Sant'Anna
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Grande Ospedale Maggiore Policlinico
city: Napoli
country: Italy
facility: A.O.U. Federico II
city: Palermo
country: Italy
facility: La Maddalena S.p.a
city: Roma
country: Italy
facility: Policlinico Umberto I - Università La Sapienza
Study officials:
affiliation: Policlinico Umberto I - Università 'Sapienza'
name: Maria Teresa Petrucci, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: A.O.U. Ospedali Riuniti Umberto I - G.M. Lancisi - G. Salesi di Ancona
name: Massimo Offidani, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kumar SK, Lee JH, Lahuerta JJ, Morgan G, Richardson PG, Crowley J, Haessler J, Feather J, Hoering A, Moreau P, LeLeu X, Hulin C, Klein SK, Sonneveld P, Siegel D, Blade J, Goldschmidt H, Jagannath S, Miguel JS, Orlowski R, Palumbo A, Sezer O, Rajkumar SV, Durie BG; International Myeloma Working Group. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012 Jan;26(1):149-57. doi: 10.1038/leu.2011.196. Epub 2011 Jul 29.
citation: Lonial S, Lee HC, Badros A, Trudel S, Nooka AK, Chari A, Abdallah AO, Callander N, Lendvai N, Sborov D, Suvannasankha A, Weisel K, Karlin L, Libby E, Arnulf B, Facon T, Hulin C, Kortum KM, Rodriguez-Otero P, Usmani SZ, Hari P, Baz R, Quach H, Moreau P, Voorhees PM, Gupta I, Hoos A, Zhi E, Baron J, Piontek T, Lewis E, Jewell RC, Dettman EJ, Popat R, Esposti SD, Opalinska J, Richardson P, Cohen AD. Belantamab mafodotin for relapsed or refractory multiple myeloma (DREAMM-2): a two-arm, randomised, open-label, phase 2 study. Lancet Oncol. 2020 Feb;21(2):207-221. doi: 10.1016/S1470-2045(19)30788-0. Epub 2019 Dec 16.