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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)
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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)
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Secondary ID [1]
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PRN1008-018
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Secondary ID [2]
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EFC17093
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Universal Trial Number (UTN)
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Trial acronym
LUNA 3
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Immune Thrombocytopenia
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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Other blood disorders
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Inflammatory and Immune System
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0
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Durable platelet response during the last 6 weeks of the 24-week blinded treatment period (not for EU and UK)
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Assessment method [1]
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Durable platelet response is defined as a proportion of participants able to achieve platelet counts at or above 50,000/µL 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 are at or above 50,000/µL.
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Timepoint [1]
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24 weeks
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Primary outcome [2]
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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
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Assessment method [2]
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Timepoint [2]
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24 weeks
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Secondary outcome [1]
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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
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Assessment method [1]
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Timepoint [1]
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24 weeks
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Secondary outcome [2]
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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
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Assessment method [2]
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Timepoint [2]
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24 weeks
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Secondary outcome [3]
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Time to first platelet count of =50,000/µL OR between =30,000/µL and <50,000/µL and doubled from baseline
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Assessment method [3]
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Timepoint [3]
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24 weeks
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Secondary outcome [4]
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Proportion of patients requiring rescue therapy during the 24-week blinded treatment period
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Assessment method [4]
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Timepoint [4]
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24 weeks
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Secondary outcome [5]
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Change from baseline on Item 10 of the ITP-Patient Assessment Questionnaire in adult patients (=18 years) at Week 13
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Assessment method [5]
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Timepoint [5]
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From baseline to Week 13
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Secondary outcome [6]
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for EU and UK: Change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25
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Assessment method [6]
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Timepoint [6]
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At Week 25
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Secondary outcome [7]
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Proportion of participants who able to achieve stable platelet response, within a period of 24 weeks following initial achievement of the platelet response
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Assessment method [7]
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Stable platelet response is defined as no 2 scheduled visits, at least 4 weeks apart, with a platelet count less than 50,000/µL, without an intervening visit with a platelet count =50,000/µL. Initial platelet response defined as platelet count =50,000/µL within 12 weeks of initiation of treatment with rilzabrutinib during the study.
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Timepoint [7]
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24 weeks
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Secondary outcome [8]
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Frequency and severity of Treatment Emergent Adverse Events
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Assessment method [8]
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Including physical examination, ECG, clinical laboratory test results, vital signs and laboratory tests (serum chemistry, hematology, except for platelet counts included in the primary efficacy endpoint)
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Timepoint [8]
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52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
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Secondary outcome [9]
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Frequency and severity of bleeding TEAEs
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Assessment method [9]
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Timepoint [9]
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52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
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Secondary outcome [10]
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Plasma concentrations of rilzabrutinib
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Assessment method [10]
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Timepoint [10]
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Until 52 weeks
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Secondary outcome [11]
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Change from baseline on the Symptoms, Bother and Activity domains of the ITP Patient Assessment Questionnaire (ITP-PAQ) in adult patients (=18 years)
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Assessment method [11]
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The ITP Patient Assessment Questionnaire™ (ITP-PAQ™) is a disease-specific instrument that was designed to measure the Quality of Life (QoL) of adult patients with immune thrombocytopenia. The items employ a 4-week recall with responses recorded on 4-, 5- or 7-point Likert scales. All item scores are transformed to a 0 to 100 continuum where higher scores represent better QoL and are weighted equally to derive the scale scores.
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Timepoint [11]
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52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
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Secondary outcome [12]
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Change from baseline in disease-specific QoL as measured by the Kids' ITP Tools (ITP-KIT) score in pediatric participants
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Assessment method [12]
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The ITP-KIT include a battery of three disease-specific instruments, a child self-report form designed to be completed by children =7 years, a parent proxy report form for children \<7 and a parent impact form. Respondents record their disease experience based on a 1-week recall. The instrument yields a total score which is the summation of the items converted to a 0 to 100 score with higher scores indicating better disease-specific QoL.
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Timepoint [12]
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52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
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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
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Minimum age
10
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/12/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
20/11/2026
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Actual
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Sample size
Target
194
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
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Recruitment hospital [1]
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Investigational Site Number : 3607 - Kogarah
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Recruitment hospital [2]
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Investigational Site Number : 3608 - Westmead
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Recruitment hospital [3]
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Investigational Site Number : 3611 - Brisbane
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Recruitment hospital [4]
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Investigational Site Number : 3609 - Adelaide
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Recruitment hospital [5]
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Investigational Site Number : 3606 - Frankston
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Recruitment hospital [6]
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Investigational Site Number : 3610 - Perth
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Recruitment postcode(s) [1]
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2217 - Kogarah
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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4101 - Brisbane
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Recruitment postcode(s) [4]
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5000 - Adelaide
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Recruitment postcode(s) [5]
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3199 - Frankston
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Recruitment postcode(s) [6]
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6000 - Perth
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Recruitment outside Australia
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United States of America
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California
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Colorado
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Florida
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Georgia
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Illinois
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Kentucky
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Massachusetts
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Utah
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Washington
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Argentina
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Poland
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Wielkopolskie
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Gdynia
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Russian Federation
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Moscow
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Russian Federation
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Novosibirsk
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Russian Federation
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Pyatigorsk
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Russian Federation
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Samara
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Russian Federation
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St. Petersburg
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Russian Federation
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Tula
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Singapore
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Singapore
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Málaga
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Valenciana, Comunidad
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Barcelona
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Madrid
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Murcia
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Sevilla
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Chiangmai
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Thailand
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Khon Kaen
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Thailand
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Songkla
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Turkey
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Ankara
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Istanbul
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Izmir
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Turkey
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Kayseri
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Ukraine
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Dnipropetrovsk
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Ukraine
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Kryvyi Rih City
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Ukraine
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Kyiv
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United Kingdom
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London, City Of
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United Kingdom
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Harrow
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United Kingdom
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London
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United Kingdom
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Manchester
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United Kingdom
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Norfolk
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United Kingdom
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Principia Biopharma, a Sanofi Company
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Ethics approval
Ethics application status
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Summary
Brief summary
This is 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 will receive rilzabrutinib or placebo 400mg twice daily. For each patient, the study will last up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This includes 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. For adult participants, the maximum duration of the long-term extension (LTE) period will be 12 months from the date of the last adult participant to enter the LTE. For pediatric participants, the maximum duration of the LTE period will be 12 months from the date of the last pediatric participant to enter the LTE.
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Trial website
https://clinicaltrials.gov/study/NCT04562766
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Contact person for public queries
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Trial Transparency email recommended (Toll free number for US & Canada)
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800-633-1610
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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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
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
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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