Register a trial

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04562766




Registration number
NCT04562766
Ethics application status
Date submitted
18/09/2020
Date registered
24/09/2020

Titles & IDs
Public title
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Scientific 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)
Secondary ID [1] 0 0
PRN1008-018
Secondary ID [2] 0 0
EFC17093
Universal Trial Number (UTN)
Trial acronym
LUNA 3
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immune Thrombocytopenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Rilzabrutinib
Treatment: Drugs - Placebo

Experimental: Rilzabrutinib - Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period

Placebo comparator: Placebo - Patients receive matching placebo 400mg orally twice daily for up to 24 weeks


Treatment: Drugs: Rilzabrutinib
400mg Caplet

Treatment: Drugs: Placebo
400mg Caplet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Durable platelet response during the last 6 weeks of the 24-week blinded treatment period (not for EU and UK)
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
for EU and UK: Proportion of adult participants able to achieve platelet counts at or above 50,000/µL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy
Timepoint [2] 0 0
24 weeks
Secondary outcome [1] 0 0
Number of weeks with platelet count =50,000/µL OR between =30,000/µL and <50,000/µL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Number of weeks with platelet counts =30,000/µL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Time to first platelet count of =50,000/µL OR between =30,000/µL and <50,000/µL and doubled from baseline
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Proportion of patients requiring rescue therapy during the 24-week blinded treatment period
Timepoint [4] 0 0
24 weeks
Secondary outcome [5] 0 0
Change from baseline on Item 10 of the ITP-Patient Assessment Questionnaire in adult patients (=18 years) at Week 13
Timepoint [5] 0 0
From baseline to Week 13
Secondary outcome [6] 0 0
for EU and UK: Change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25
Timepoint [6] 0 0
At Week 25
Secondary outcome [7] 0 0
Proportion of participants who able to achieve stable platelet response, within a period of 24 weeks following initial achievement of the platelet response
Timepoint [7] 0 0
24 weeks
Secondary outcome [8] 0 0
Frequency and severity of Treatment Emergent Adverse Events
Timepoint [8] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Secondary outcome [9] 0 0
Frequency and severity of bleeding TEAEs
Timepoint [9] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Secondary outcome [10] 0 0
Plasma concentrations of rilzabrutinib
Timepoint [10] 0 0
Until 52 weeks
Secondary outcome [11] 0 0
Change from baseline on the Symptoms, Bother and Activity domains of the ITP Patient Assessment Questionnaire (ITP-PAQ) in adult patients (=18 years)
Timepoint [11] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Secondary outcome [12] 0 0
Change from baseline in disease-specific QoL as measured by the Kids' ITP Tools (ITP-KIT) score in pediatric participants
Timepoint [12] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose

Eligibility
Key inclusion criteria
1. Patients will be 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
2. 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
3. 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.
4. 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])
5. Hemoglobin >9 g/dL within 1 week prior to Study Day 1
6. All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
7. 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
Minimum age
10 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Patients with secondary ITP
2. Pregnant or lactating women
3. 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
4. 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
5. Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
6. 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
7. 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
8. Has 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
9. History of solid organ transplant
10. Myelodysplastic syndrome
11. Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
12. Planned surgery in the time frame of the dosing period

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
20/11/2026
Actual
Sample size
Target
194
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 3607 - Kogarah
Recruitment hospital [2] 0 0
Investigational Site Number : 3608 - Westmead
Recruitment hospital [3] 0 0
Investigational Site Number : 3611 - Brisbane
Recruitment hospital [4] 0 0
Investigational Site Number : 3609 - Adelaide
Recruitment hospital [5] 0 0
Investigational Site Number : 3606 - Frankston
Recruitment hospital [6] 0 0
Investigational Site Number : 3610 - Perth
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4101 - Brisbane
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
3199 - Frankston
Recruitment postcode(s) [6] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Kentucky
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Utah
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
Argentina
State/province [13] 0 0
Buenos Aires
Country [14] 0 0
Argentina
State/province [14] 0 0
Córdoba
Country [15] 0 0
Argentina
State/province [15] 0 0
Corrientes
Country [16] 0 0
Argentina
State/province [16] 0 0
San Juan
Country [17] 0 0
Austria
State/province [17] 0 0
Graz
Country [18] 0 0
Austria
State/province [18] 0 0
Leoben
Country [19] 0 0
Austria
State/province [19] 0 0
Linz
Country [20] 0 0
Austria
State/province [20] 0 0
Steyr
Country [21] 0 0
Austria
State/province [21] 0 0
Wien
Country [22] 0 0
Brazil
State/province [22] 0 0
Bahia
Country [23] 0 0
Brazil
State/province [23] 0 0
Paraná
Country [24] 0 0
Brazil
State/province [24] 0 0
Rio Grande Do Sul
Country [25] 0 0
Brazil
State/province [25] 0 0
São Paulo
Country [26] 0 0
Brazil
State/province [26] 0 0
Belem Do Para
Country [27] 0 0
Brazil
State/province [27] 0 0
Rio de Janeiro
Country [28] 0 0
Canada
State/province [28] 0 0
Alberta
Country [29] 0 0
Canada
State/province [29] 0 0
Ontario
Country [30] 0 0
Canada
State/province [30] 0 0
Quebec
Country [31] 0 0
Chile
State/province [31] 0 0
Coquimbo
Country [32] 0 0
Chile
State/province [32] 0 0
Reg Metropolitana De Santiago
Country [33] 0 0
Chile
State/province [33] 0 0
Valparaíso
Country [34] 0 0
China
State/province [34] 0 0
Hubei
Country [35] 0 0
China
State/province [35] 0 0
Liaoning
Country [36] 0 0
China
State/province [36] 0 0
Shaanxi
Country [37] 0 0
China
State/province [37] 0 0
Shandong
Country [38] 0 0
China
State/province [38] 0 0
Tianjin
Country [39] 0 0
China
State/province [39] 0 0
Yunnan
Country [40] 0 0
China
State/province [40] 0 0
Hangzhou
Country [41] 0 0
China
State/province [41] 0 0
Hefei
Country [42] 0 0
China
State/province [42] 0 0
Nanchang
Country [43] 0 0
China
State/province [43] 0 0
Tangshan
Country [44] 0 0
China
State/province [44] 0 0
Wuxi
Country [45] 0 0
China
State/province [45] 0 0
ZhenJiang
Country [46] 0 0
France
State/province [46] 0 0
Angers
Country [47] 0 0
France
State/province [47] 0 0
Creteil
Country [48] 0 0
France
State/province [48] 0 0
Dijon cedex
Country [49] 0 0
France
State/province [49] 0 0
Nantes Cedex 1
Country [50] 0 0
France
State/province [50] 0 0
Paris
Country [51] 0 0
France
State/province [51] 0 0
Pessac
Country [52] 0 0
Germany
State/province [52] 0 0
Berlin
Country [53] 0 0
Germany
State/province [53] 0 0
Düsseldorf
Country [54] 0 0
Germany
State/province [54] 0 0
Frankfurt am Main
Country [55] 0 0
Germany
State/province [55] 0 0
Recklinghausen
Country [56] 0 0
Hungary
State/province [56] 0 0
Budapest
Country [57] 0 0
Hungary
State/province [57] 0 0
Debrecen
Country [58] 0 0
Hungary
State/province [58] 0 0
Gyor
Country [59] 0 0
Hungary
State/province [59] 0 0
Nyíregyháza
Country [60] 0 0
Hungary
State/province [60] 0 0
Szekesfehervar
Country [61] 0 0
Israel
State/province [61] 0 0
Haifa
Country [62] 0 0
Israel
State/province [62] 0 0
Kfar Saba
Country [63] 0 0
Israel
State/province [63] 0 0
Tel Aviv
Country [64] 0 0
Israel
State/province [64] 0 0
Tel Hashomer
Country [65] 0 0
Israel
State/province [65] 0 0
Zerifin
Country [66] 0 0
Italy
State/province [66] 0 0
Toscana
Country [67] 0 0
Italy
State/province [67] 0 0
Bologna
Country [68] 0 0
Italy
State/province [68] 0 0
Milano
Country [69] 0 0
Italy
State/province [69] 0 0
Trieste
Country [70] 0 0
Italy
State/province [70] 0 0
Vicenza
Country [71] 0 0
Japan
State/province [71] 0 0
Ibaraki
Country [72] 0 0
Japan
State/province [72] 0 0
Ishikawa
Country [73] 0 0
Japan
State/province [73] 0 0
Kanagawa
Country [74] 0 0
Japan
State/province [74] 0 0
Osaka
Country [75] 0 0
Japan
State/province [75] 0 0
Saitama
Country [76] 0 0
Japan
State/province [76] 0 0
Tokyo
Country [77] 0 0
Japan
State/province [77] 0 0
Chiba
Country [78] 0 0
Japan
State/province [78] 0 0
Hiroshima City
Country [79] 0 0
Japan
State/province [79] 0 0
Saitama-shi
Country [80] 0 0
Korea, Republic of
State/province [80] 0 0
Busan-gwangyeoksi
Country [81] 0 0
Korea, Republic of
State/province [81] 0 0
Gyeonggi-do
Country [82] 0 0
Korea, Republic of
State/province [82] 0 0
Seoul-teukbyeolsi
Country [83] 0 0
Mexico
State/province [83] 0 0
Nuevo León
Country [84] 0 0
Mexico
State/province [84] 0 0
Chihuahua
Country [85] 0 0
Mexico
State/province [85] 0 0
Ciudad de México
Country [86] 0 0
Mexico
State/province [86] 0 0
Delegacion Benito Juarez
Country [87] 0 0
Mexico
State/province [87] 0 0
Durango
Country [88] 0 0
Mexico
State/province [88] 0 0
Zapopan
Country [89] 0 0
Netherlands
State/province [89] 0 0
Rotterdam
Country [90] 0 0
Norway
State/province [90] 0 0
Bergen
Country [91] 0 0
Norway
State/province [91] 0 0
Gralum
Country [92] 0 0
Poland
State/province [92] 0 0
Pomorskie
Country [93] 0 0
Poland
State/province [93] 0 0
Wielkopolskie
Country [94] 0 0
Poland
State/province [94] 0 0
Gdynia
Country [95] 0 0
Poland
State/province [95] 0 0
Poznan
Country [96] 0 0
Poland
State/province [96] 0 0
Warszawa
Country [97] 0 0
Poland
State/province [97] 0 0
Wroclaw
Country [98] 0 0
Russian Federation
State/province [98] 0 0
Moscow
Country [99] 0 0
Russian Federation
State/province [99] 0 0
Novosibirsk
Country [100] 0 0
Russian Federation
State/province [100] 0 0
Pyatigorsk
Country [101] 0 0
Russian Federation
State/province [101] 0 0
Samara
Country [102] 0 0
Russian Federation
State/province [102] 0 0
St. Petersburg
Country [103] 0 0
Russian Federation
State/province [103] 0 0
Tula
Country [104] 0 0
Singapore
State/province [104] 0 0
Singapore
Country [105] 0 0
Spain
State/province [105] 0 0
Málaga
Country [106] 0 0
Spain
State/province [106] 0 0
Valenciana, Comunidad
Country [107] 0 0
Spain
State/province [107] 0 0
Barcelona
Country [108] 0 0
Spain
State/province [108] 0 0
Burgos
Country [109] 0 0
Spain
State/province [109] 0 0
Madrid
Country [110] 0 0
Spain
State/province [110] 0 0
Murcia
Country [111] 0 0
Spain
State/province [111] 0 0
Sevilla
Country [112] 0 0
Thailand
State/province [112] 0 0
Bangkok
Country [113] 0 0
Thailand
State/province [113] 0 0
Chiangmai
Country [114] 0 0
Thailand
State/province [114] 0 0
Khon Kaen
Country [115] 0 0
Thailand
State/province [115] 0 0
Songkla
Country [116] 0 0
Turkey
State/province [116] 0 0
Ankara
Country [117] 0 0
Turkey
State/province [117] 0 0
Istanbul
Country [118] 0 0
Turkey
State/province [118] 0 0
Izmir
Country [119] 0 0
Turkey
State/province [119] 0 0
Kayseri
Country [120] 0 0
Ukraine
State/province [120] 0 0
Dnipropetrovsk
Country [121] 0 0
Ukraine
State/province [121] 0 0
Kryvyi Rih City
Country [122] 0 0
Ukraine
State/province [122] 0 0
Kyiv
Country [123] 0 0
United Kingdom
State/province [123] 0 0
London, City Of
Country [124] 0 0
United Kingdom
State/province [124] 0 0
Harrow
Country [125] 0 0
United Kingdom
State/province [125] 0 0
London
Country [126] 0 0
United Kingdom
State/province [126] 0 0
Manchester
Country [127] 0 0
United Kingdom
State/province [127] 0 0
Norfolk
Country [128] 0 0
United Kingdom
State/province [128] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Principia Biopharma, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free number for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04562766