Clinical trial NCT05002777: Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)
Official title: A Multicenter, Open-label, Phase IIb Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (LUMINA 2)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Warm Autoimmune Hemolytic Anemia (wAIHA)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2021-08-12
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks.

There will be a 7-day safety follow-up period after receiving the last dose of study medication either in Part A (for those not eligible for Part B or early terminated) or Part B. In addition, each participant will be asked to attend an EOT-Core Part B visit when the last participant completes 52 weeks in Core Part B. The Extended Part B period will last for up to 253 weeks.


Eligibility criteria: Inclusion Criteria:

* Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
* Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
* Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
* Up-to-date vaccination status as per local guidelines.
* Body mass index (BMI) \>17.5 and \<40 kg/m2
* All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Core Part B

* Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A.
* Completion of Part A treatment period (24 weeks). Extended Part B
* Completion of Core Part B period.

Exclusion Criteria:

* Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
* Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
* Secondary wAIHA from any cause including drugs, lymphoproliferative disorders (low-count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
* Myelodysplastic syndrome.
* Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA.
* HIV infection.
* Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start.
* Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent.

Part B only

* Participants who receive any therapy during Part A known to be active in wAIHA.
* Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Primary outcomes:
measure: Part A: Proportion of participants with overall hemoglobin response
timeFrame: By Week 24 in Part A
measure: Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response
timeFrame: By Week 50 in Part B
Secondary outcomes:
measure: Proportion of participants with durable hemoglobin response
timeFrame: By Week 24 in Part A
measure: Median time from baseline to first hemoglobin response
timeFrame: From Day 1 to Week 24 in Part A
measure: Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received
timeFrame: After Week 1 of treatment to Week 24 in Part A and Week 305 in Part B
measure: Change from baseline in FACIT-Fatigue scale score
timeFrame: Until Week 24 in Part A and Week 305 in Part B
measure: Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs)
timeFrame: Until Week 24 in Part A and Week 305 in Part B
Other outcomes:
Locations:
city: Cerritos
country: United States
facility: The Oncology Institute of Hope & Innovation Site Number : 8400006
state: California
city: Cerritos
country: United States
facility: TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006
state: California
city: Glendale
country: United States
facility: TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006
state: California
city: Long Beach
country: United States
facility: TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006
state: California
city: Los Angeles
country: United States
facility: University of Southern California_Investigational Site Number: 8400009
state: California
city: Santa Ana
country: United States
facility: TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006
state: California
city: Torrance
country: United States
facility: The Lundquist Institute_Investigational Site Number: 8400005
state: California
city: Whittier
country: United States
facility: TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006
state: California
city: Washington
country: United States
facility: Georgetown University Hospital_Investigational Site Number: 8400003
state: District of Columbia
city: Tamarac
country: United States
facility: Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002
state: Florida
city: Boston
country: United States
facility: Massachusetts General Hospital_Investigational Site Number: 8400001
state: Massachusetts
city: Vienna
country: Austria
facility: Hanush-Krankenhaus_Investigational Site Number: 0400001
city: Beijing
country: China
facility: Peking Union Medical College Hospital_Investigational Site Number: 1560002
city: Tianjin
country: China
facility: Institute of hematology&blood diseases hospital_Investigational Site Number: 1560003
city: Odense
country: Denmark
facility: Odense Universitetshospital Hæmatologisk Forskningsenhed_Investigational Site Number: 2080001
city: Essen
country: Germany
facility: Klinik für Hämatologie und Stammzellentransplantation_Investigational Site Number: 2760001
city: Szekesfehervar
country: Hungary
facility: Fejer Varmegyei Szent Gyorgy Egyetemi Oktato Korhaz_Investigational Site Number: 3480001
city: Avellino
country: Italy
facility: Ospedale Giuseppe Moscati_Investigational Site Number: 3800002
city: Meldola
country: Italy
facility: Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori"_Investigational Site Number: 3800003
city: Milan
country: Italy
facility: Ospedale Maggiore Policlinico_Investigational Site Number: 3800001
city: Barakaldo
country: Spain
facility: Hospital Universitario de Cruces_Investigational Site Number: 7240004
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona_Investigational Site Number: 7240001
city: Madrid
country: Spain
facility: Hospital Universitario La Paz_Investigational Site Number: 7240003
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío_Investigational Site Number: 7240002
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust_Investigational Site Number: 8260001
city: London
country: United Kingdom
facility: Barts Health NHS Trust_Investigational Site Number: 8260005
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust_Investigational Site Number: 8260002
Study officials:
References: