Immune ThrombocytopeniaPhase 3LUNA 3NCT04562766
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
A Phase 3 study testing Rilzabrutinib in people with Immune Thrombocytopenia, run by Principia Biopharma, a Sanofi Company at 155 sites in 26 countries.
Trial summary
This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Rilzabrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 232 participants
- Where
- 155 sitesin 26 countries
- Study dates
- 14 Dec 2020to 2 Sep 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female with primary ITP with duration of >6 months in pediatric participants aged 12 to <18 years (pediatric participants aged 10 to <12 years will be enrolled in the EU [EEA countries] only) and duration of >3 months in ages 18 years and above
- Patients who had a response (achievement of platelet count ≥50,000/µL) to IVIg/anti-D or CSs that was not sustained and who have documented intolerance, insufficient response or any contra-indication to any appropriate courses of standard of care ITP therapy
- An average of 2 platelet counts at least 5 days apart of <30,000/µL during the Screening period and no single platelet count >35,000/µL, within 14 days prior to the first dose of study drug.
- Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator.
- Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X 10^9/L, AST/ALT ≤1.5 x upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN [unless the patient has documented Gilbert syndrome], glomerular filtration rate >50 [Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants])
- Hemoglobin >9 g/dL within 1 week prior to Study Day 1
- All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- Patients must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the patient's guardian and agree to the schedule of assessments
Who cannot join
- Patients with secondary ITP
- Pregnant or lactating women
- History (within 5 years of Study Day 1) or current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non melanoma skin cancer
- Transfusion with blood, blood products, plasmapheresis, or use of any other rescue medications with intent to increase platelet count within 14 days before Study Day 1
- Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
- Immunosuppressant drugs other than CSs within 5 times the elimination half-life of the drug or 14 days of Study Day 1, whichever is longer
Show all 15Show fewer
- Treatment with rituximab or splenectomy within the 3 months prior to Study Day 1
- Patients treated with rituximab will have normal B-cell counts prior to enrollment
- Had received any investigational drug within the 30 days before receiving the first dose of study medication, or at least 5 times elimination half-life of the drug (whichever is longer); patient should not be using an investigational device at the time of dosing
- Patients who previously received treatment with Bruton's Tyrosine Kinase (BTK) inhibitors (except rilzabrutinib) within 30 days before the first dose of study drug are not eligible
- Patients who previously received rilzabrutinib at any time are not eligible
- History of solid organ transplant
- Myelodysplastic syndrome
- Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
- Planned surgery in the time frame of the dosing period
Trial locations and contacts
155 sites in 26 countries.
155 sites in 26 countries
United States17 sites
Children's Healthcare of Atlanta_Investigational Site Number 84034
Atlanta, Georgia, 30322
Children's Hospital Colorado_Investigational Site Number 84025
Aurora, Colorado, 80045
Massachusetts General Hospital Site Number : 84038
Boston, Massachusetts, 02114
IMMUNOe International Research Centers_Investigational Site Number 84028
Centennial, Colorado, 80112
Rush University Medical Center_Investigational Site Number 84029
Chicago, Illinois, 60612
University Hospitals Cleveland Medical Center Site Number : 84036
Cleveland, Ohio, 44106
Cleveland Clinic_Investigational Site Number 84026
Cleveland, Ohio, 44195
University of Southern California_Investigational Site Number 84024
Los Angeles, California, 90033
University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033
Louisville, Kentucky, 40202
The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027
Philadelphia, Pennsylvania, 19104
University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035
Salt Lake City, Utah, 84112
UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020
San Francisco, California, 94158
University of Washington Medical Centre Site Number : 84041
Seattle, Washington, 98195
Montefiore Medical Center_Investigational Site Number 84032
The Bronx, New York, 10461
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037
Torrance, California, 90502
ASCLEPES Research Centers_Investigational Site Number 84023
Weeki Wachee, Florida, 34607
The Oncology Institute of Hope and Innovation_Investigational Site Number 84031
Whittier, California, 90602
China12 sites
Investigational Site Number : 15611
Hangzhou, 310018
Investigational Site Number : 15608
Hefei
Qilu Hospital of Shandong University - Investigational Site Number: 15605
Jinan, Shandong, 250012
Second Affiliated Hospital of Kunming Medical University - Investigational Site Number: 15604
Kunming, Yunnan, 650101
Investigational Site Number : 15610
Nanchang, 330006
Shengjing Hospital of China Medical University - Investigational Site Number: 15603
Shenyang, Liaoning, 110022
Investigational Site Number : 15613
Tangshan, 63000
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences - Investigational Site Number: 15602
Tianjin, Tianjin Municipality, 300020
Wuhan Union Hospital of Tongji Medical College of HUST - Investigational Site Number: 15601
Wuhan, Hubei, 430022
Investigational Site Number : 15609
Wuxi, 214023
Shaanxi Provincial People's Hospital - Investigational Site Number: 15607
Xi'an, Shaanxi, 710068
Investigational Site Number : 15614
Zhenjiang, 212001
Japan12 sites
Investigational Site Number : 39208
Bunkyo-ku, Tokyo, 113-8655
Investigational Site Number : 39212
Chiba, 60-0852
Investigational Site Number : 39203
Hiroshima, 730-8619
Investigational Site Number : 39201
Iruma-gun, Saitama, 350-0495
Investigational Site Number : 39205
Kanazawa, Ishikawa-ken, 920-8530
Investigational Site Number : 39210
Meguro-ku, Tokyo, 152-8902
Investigational Site Number : 39207
Sagamihara-shi, Kanagawa, 252-0375
Investigational Site Number : 39206
Saitama-shi, 330-8777
Investigational Site Number : 39204
Setagaya City, Tokyo, 157-0074
Investigational Site Number : 39202
Suita-shi, Osaka, 565-0871
Investigational Site Number : 39209
Sumida-ku, Tokyo, 130-8575
Investigational Site Number : 39214
Tsuchiura-shi, Ibaraki
Spain9 sites
Investigational Site Number : 72412
Barcelona, 08035
Investigational Site Number : 72414
Barcelona, 08035
Investigational Site Number : 72409
Barcelona, 08041
Investigational Site Number : 72416
Burgos
Investigational Site Number : 72410
Madrid, 28007
Investigational Site Number : 72408
Málaga, Málaga, 29010
Investigational Site Number : 72411
Murcia, 30008
Investigational Site Number : 72413
Seville, 41013
Investigational Site Number : 72407
Valencia, Valenciana, Comunidad, 46010
Brazil7 sites
CEMEC Oncologica do Brasil Site Number : 7606
Belém, 66053-000
Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610
Cascavel, Paraná, 85806-300
Hospital De Clinicas De Porto Alegre Site Number : 7605
Porto Alegre, Rio Grande do Sul, 90035 003
HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 7609
Rio de Janeiro, 20211030
Hospital Sao Rafael Instituto D'Or da Bahia Site Number : 7608
Salvador, Estado de Bahia, 41253190
Hospital Santa Marcelina Site Number : 7611
São Paulo, São Paulo, 08270-070
Hospital do Servidor Publico Estadual de Sao Paulo Site Number : 7607
São Paulo, 04039-004
France7 sites
Investigational Site Number : 25014
Angers, 49933
Investigational Site Number : 25011
Créteil, 94000
Investigational Site Number : 25010
Dijon, 21079
Investigational Site Number : 25009
Nantes, 44093
Investigational Site Number : 25008
Paris, 75012
Investigational Site Number : 25012
Paris, 75019
Investigational Site Number : 25007
Pessac, 33600
Russia7 sites
Investigational Site Number : 64307
Moscow, 119049
Investigational Site Number : 64305
Moscow, 125167
Investigational Site Number : 64304
Novosibirsk, 630090
Investigational Site Number : 64301
Pyatigorsk, 357502
Investigational Site Number : 64302
Saint Petersburg, 191024
Investigational Site Number : 64306
Samara, 443099
Investigational Site Number : 64303
Tula, 300053
United Kingdom7 sites
Investigational Site Number : 82604
Harrow, HA1 3UJ
Investigational Site Number : 82607
London, London, City of, W12 0HS
Investigational Site Number : 82606
London, SE5 9PJ
Investigational Site Number : 82609
London, W2 1NY
Investigational Site Number : 82603
Manchester, M13 9WL
Investigational Site Number : 82605
Norfolk, NR31 6LA
Investigational Site Number : 82608
Southampton, SO16 6YD
Australia6 sites
Investigational Site Number : 3609
Adelaide, South Australia, 5000
Investigational Site Number : 3611
Brisbane, Queensland, 4101
Investigational Site Number : 3606
Frankston, Victoria, 3199
Investigational Site Number : 3607
Kogarah, New South Wales, 2217
Investigational Site Number : 3610
Perth, Western Australia, 6000
Investigational Site Number : 3608
Westmead, New South Wales, 2145
Italy6 sites
Investigational Site Number : 38012
Bologna, 40138
Investigational Site Number : 38014
Florence, Tuscany, 50134
Investigational Site Number : 38015
Milan, 20123
Investigational Site Number : 38013
Milan, 20142
Investigational Site Number : 38010
Trieste, 34125
Investigational Site Number : 38011
Vicenza, 36100
Mexico6 sites
Investigational Site Number : 48402
Chihuahua City, 31200
Investigational Site Number : 48405
Delegacion Benito Juarez, 03720
Investigational Site Number : 48404
Durango, 34000
Investigational Site Number : 48406
Mexico City, 06760
Investigational Site Number : 48401
Monterrey, Nuevo León, 64460
Investigational Site Number : 48403
Zapopan, 45030
Poland6 sites
Investigational Site Number : 61615
Gdynia, 81-519
Investigational Site Number : 61617
Piła, Greater Poland Voivodeship, 64-920
Investigational Site Number : 61614
Poznan, 61 696
Investigational Site Number : 61609
Słupsk, Pomeranian Voivodeship, 76-200
Investigational Site Number : 61612
Warsaw, 02-776
Investigational Site Number : 61613
Wroclaw, 50-556
Argentina5 sites
Investigational Site Number : 3206
Capital Federal, Buenos Aires, C1280AEB
Investigational Site Number : 3205
Córdoba, Córdoba Province, X5003DCE
Investigational Site Number : 3209
Corrientes, W3410 FND
Investigational Site Number : 3211
La Plata, Buenos Aires, B1900
Investigational Site Number : 3208
San Juan, 5400
Austria5 sites
Investigational Site Number : 4005
Graz, 8036
Investigational Site Number : 4004
Leoben, 8700
Investigational Site Number : 4001
Linz, A4020
Investigational Site Number : 4003
Steyr, 4400
Investigational Site Number : 4002
Vienna, 1140
Hungary5 sites
Investigational Site Number : 34803
Budapest, 1083
Investigational Site Number : 34805
Debrecen, 4032
Investigational Site Number : 34801
Győr, 9024
Investigational Site Number : 34804
Nyíregyháza, 4405
Investigational Site Number : 34802
Székesfehérvár, 8000
Israel5 sites
Investigational Site Number : 37605
Haifa, 3109601
Investigational Site Number : 37606
Kfar Saba, 4428164
Investigational Site Number : 37607
Tel Aviv, 6423906
Investigational Site Number : 37608
Tel Litwinsky, 52621
Investigational Site Number : 37609
Ẕerifin, 70300
South Korea5 sites
Investigational Site Number : 41004
Busan, Busan, 49241
Investigational Site Number : 41003
Seoul, Seoul-teukbyeolsi, 02841
Investigational Site Number : 41005
Seoul, Seoul-teukbyeolsi, 03080
Investigational Site Number : 41006
Seoul, Seoul-teukbyeolsi, 03722
Investigational Site Number : 41001
Suwon, Gyeonggi-do, 16499
Thailand5 sites
Investigational Site Number : 76405
Bangkok, 10330
Investigational Site Number : 76404
Bangkok, 10400
Investigational Site Number : 76402
Chiang Mai, 50200
Investigational Site Number : 76401
Khon Kaen, 40002
Investigational Site Number : 76403
Songkhla, 90110
Germany4 sites
Investigational Site Number : 27610
Berlin, 10117
Investigational Site Number 27612
Düsseldorf, 40497
Investigational Site Number : 27613
Frankfurt am Main, 60596
Investigational Site Number : 27611
Recklinghausen, 45659
Turkey (Türkiye)4 sites
Investigational Site Number 79208
Ankara, 06620
Investigational Site Number 79210
Istanbul, 34093
Investigational Site Number 79206
Izmir, 35100
Investigational Site Number 79209
Kayseri, 38039
Canada3 sites
Investigational Site Number : 12404
Edmonton, Alberta, T6G 2P4
CHU Sainte-Justine_Investigational site number 12405
Montreal, Quebec, H3T 1C5
Investigational Site Number : 12406
Toronto, Ontario, M5G 1X8
Chile3 sites
Investigational Site Number : 15201
La Serena, Coquimbo Region, 1720430
Investigational Site Number : 15204
Santiago, Reg Metropolitana de Santiago, 7500653
Investigational Site Number : 15202
Viña del Mar, Valparaiso, 322000
Singapore3 sites
Investigational Site Number : 70201
Singapore, 119228
Investigational Site Number : 70202
Singapore, 169608
Investigational Site Number : 70203
Singapore, 308433
Ukraine3 sites
Investigational Site Number : 80408
Dnipropetrovsk, 49102
Investigational Site Number : 80409
Kryvyi Rih, 50025
Investigational Site Number : 80410
Kyiv, 03143
Norway2 sites
Investigational Site Number : 57802
Bergen, 5021
Investigational Site Number : 57801
Grålum, 1714
Netherlands1 site
Erasmus MC_Investigational Site Number 52801
Rotterdam, 3015 GD
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients received rilzabrutinib or placebo 400mg twice daily.
For each patient, the study lasted up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This included Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. Followed by a 4-week post dose follow-up.
Read more
For adult participants, the maximum duration of the long-term extension (LTE) period was 12 months from the date of the last adult participant to enter the LTE.
For pediatric participants, the maximum duration of the LTE period was 12 months from the date of the last pediatric participant to enter the LTE.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Rilzabrutinib, Placebo
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RilzabrutinibDrug
Also called PRN1008
400mg Caplet
PlaceboDrug
Also called PRN1008 Placebo
400mg Caplet
| Study group | Role | What they receive |
|---|---|---|
| Rilzabrutinib | Experimental | Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period Receives: Drug: Rilzabrutinib |
| Placebo | Placebo comparator | Patients receive matching placebo 400mg orally twice daily for up to 24 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom
Durable platelet response per guidance in regions except European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for >=two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, provided that at least 2 non-missing weekly scheduled platelet measurements were at or above 50,000/mcL during the last 6 weeks of the 24-week blinded treatment period.
Measured: Up to 24 weeks
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom
Durable platelet response per guidance in European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
Measured: Up to 24 weeks
Secondary outcomes (13)
DB Period: Number of Weeks With Platelet Count >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy
Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet count >=50,000/mcL or between >=30,000/mcL and <50,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy.
Measured: Up to 24 weeks
DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy
Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet counts >=30,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy.
Measured: Up to 24 weeks
DB Period: Time to First Platelet Count of >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and Doubled From Baseline
Time to first platelet count of >=50,000/mcL or between >=30,000/mcL and <50,000/mcL and doubled from baseline was calculated as: (date of first occurrence of platelet response - date of first study drug intake) + 1.
Measured: Up to 24 weeks
DB Period: Percentage of Participants Who Required Rescue Therapy
Resue therapy included intravenous immunoglobulin (Ig) or high-dose corticosteroids, platelet infusion, or anti-D Ig infusion. Percentage of participants who required rescue therapy are presented.
Measured: Up to 24 weeks
DB Period: Change From Baseline on Item 10 (Physical Fatigue) of the Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) in Adult Participants at Week 13
ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall quality of life(QoL)(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x([possible maximal item score - item score]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in physical fatigue is presented.Baseline:last available value before first dose of DB study drug.
Measured: Baseline (Day 1) and Week 13
DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) Assessment in Adult Participants at Week 25 Per Guidance in European Union and United Kingdom
Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by history (Hx) over previous period. In addition, 2 of these sites, skin and oral, were also assessed by physical examination (PE). These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male and postmenopausal female) at 9 sites (8 for male and postmenopausal female). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement.Baseline:last available value before first dose of DB study drug.
Measured: Baseline (Day 1) and Week 25
DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale Assessment in Adolescent Participants at Week 25 Per Guidance in European Union and United Kingdom
Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by Hx over previous period. In addition, 2 of these sites, skin and oral, were also assessed by PE. These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male) at 9 sites (8 for male). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement. Baseline:last available value before first dose of DB study drug.
Measured: Baseline (Day 1) and Week 25
DB-OL Period: Percentage of Participants With Stability of Response in Adult Participants
Stability of response was defined as the percentage of participants who were able to achieve stable platelet response defined as no 2 scheduled visits, at least 4 weeks apart, with a platelet count <50,000/mcL, without an intervening visit with a platelet count >=50,000/mcL, within a period of 24 weeks following initial achievement of the platelet response (initial platelet response defined as platelet count >=50,000/mcL within 12 weeks of initiation of treatment with rilzabrutinib during the study). This endpoint was assessed from start of DB period through OL period.
Measured: Up to 52 weeks
DB-OL Period: Percentage of Participants With Stability of Response in Adolescent Participants
Measured: Up to 52 weeks
DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and Bleeding Treatment-Emergent Adverse Events >=Grade 2
An AE was any untoward medical occurrence in a participant or clinical investigation participant, administered a study drug and which did not necessarily had a causal relationship with the study drug. An SAE was any AE that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAEs were defined as AEs that developed, worsened or became serious during the TE period. Bleeding TEAEs Grade >=2 (criteria mentioned in statistical analysis plan) are also presented.
Measured: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days (adults) and up to 175 days (adolescent)
DB Period: Plasma Concentrations of Rilzabrutinib
Blood samples were collected at specified timepoints for the analysis of plasma concentration of rilzabrutinib.
Measured: Pre-dose at Weeks 1, 13 and 25 and 2 hours post-dose at Week 1 and 25 (adults); Pre-dose at Weeks 1, 13 and 25, 0.5, 2, 4 and 6 hours post-dose at Weeks 1 and 25 (adolescent)
DB Period: Change From Baseline in the Symptoms, Bother and Activity Domains of the Immune Thrombocytopenia-Patient Assessment Questionnaire in Adult Participants
ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall QoL(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x([possible maximal item score - item score]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in symptoms,bother and activity domains is presented.Baseline:last available value before first dose of DB study drug.
Measured: Baseline (Day 1) and Week 25
DB Period: Change From Baseline in Disease-Specific Quality Of Life as Measured by the Kids' Immune Thrombocytopenia Tools (ITP-KIT) Score in Adolescent Participants
ITP-KIT was a disease-specific instrument and child self-report form designed to be completed by children >=7 years. It comprised of total of 27 items among which 26 items were structured as Likert scales with 5 response options 1: "never", 2: "rarely", 3: "sometimes", 4: "often" and 5: "always". An additional "not applicable" option was available for items 18 to 26, which was scored as 1: the same as "never". Item 27 was a descriptive question answered "yes" or "no" which was not included in the calculation of the summary score. Instrument yielded a summary KIT score which was the summation of the items calculated as: 100 x (1- [{sum of all valid responses - number of valid responses}/{number of valid responses*4}]). Scores were converted to a 0 to 100 with higher scores indicating better disease-specific QoL. Change from baseline of positive value indicated improvement. Baseline was defined as the last available value before first dose of DB study drug.
Measured: Baseline (Day 1) and Week 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2020
- Study start14 Dec 2020
- Primary completion15 Jan 2025
- Study completion2 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 232 participants (actual)
Sponsor and investigators
- Lead sponsor
- Principia Biopharma, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04562766
- Sponsor's protocol ID
- EFC17093
- Other IDs
- PRN1008-018, 2023-509401-71, 2023
- Acronym
- LUNA 3
- Status last verified
- Sep 2026
- First submitted
- 18 Sep 2020
- Last updated
- 7 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Hutter-Kronke ML, Pascual-Izquierdo C, Lucchini E, Audia S, Cordoba M, Krenz H, Yao M, Daak A. Clinical activity and safety of rilzabrutinib in patients with previously treated immune thrombocytopenia (LUNA3): results from the open-label period of a phase 3 trial. Lancet Haematol. 2026 Sep 17:S2352-3026(26)00193-6. doi: 10.1016/S2352-3026(26)00193-6. Online ahead of print.
PubMed 42753771Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Homenda W, Galindo LEM, Basquiera AL, Tan CW, Saydam G, Hutter-Kronke ML, Chai-Adisaksopha C, Gomez-Almaguer D, Tran H, Shin HJ, Dantas da Cunha Junior A, Lazar Z, Izquierdo CP, Kirgner I, Lucchini E, Kuzmina G, Fillitz M, Audia S, Taparia M, Cordoba M, Diab R, Yao M, Gouia I, Lee M, Daak A. Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study. Blood. 2025 Jun 12;145(24):2914-2926. doi: 10.1182/blood.2024027336.
PubMed 40090011Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available.
PubMed 39899371Kuter DJ, Bussel JB, Ghanima W, Cooper N, Gernsheimer T, Lambert MP, Liebman HA, Tarantino MD, Lee M, Guo H, Daak A. Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. Ther Adv Hematol. 2023 Oct 18;14:20406207231205431. doi: 10.1177/20406207231205431. eCollection 2023.
PubMed 37869360