MedPath

Myeloma XIV: Frailty-adjusted Therapy in Transplant Non-Eligible Patients With Newly Diagnosed Multiple Myeloma

Phase 3
Recruiting
Conditions
Multiple Myeloma
Registration Number
NCT03720041
Lead Sponsor
University of Leeds
Brief Summary

Trial Title:

FiTNEss (UK-MRA Myeloma XIV) - Frailty-adjusted therapy in Transplant Non-Eligible patients with newly diagnosed Multiple Myeloma

Overview:

A phase III, multi-centre, randomised controlled trial to compare standard (reactive) and frailty-adjusted (adaptive) induction therapy delivery with the novel triplet ixazomib, lenalidomide and dexamethasone (IRD), and to compare maintenance lenalidomide (R) to lenalidomide plus ixazomib (R+I) in patients with newly diagnosed multiple myeloma not suitable for a stem cell transplant.

All participants receive induction treatment with ixazomib, lenalidomide and dexamethasone and are randomised on a 1:1 basis at trial entry to the use of frailty score-adjusted up-front dose reductions vs. standard up-front dosing followed by toxicity dependent reactive dose-modifications during therapy. Following 12 cycles of induction treatment participants alive and progression-free undergo a second randomisation on a 1:1 basis to maintenance treatment with lenalidomide plus placebo versus lenalidomide plus ixazomib. Participants and their treating physicians will be blinded to maintenance allocation.

Participant population:

* Newly diagnosed as having Multiple Myeloma (MM) according to the updated IMWG diagnostic criteria 2014 (see Appendix 1 for criteria)

* Not eligible for stem cell transplant

* Aged at least 18 years

* Able to provide written informed consent

Number of participants:

740 participants will be entered into the trial at Randomisation 1 (R1), with 478 participants at Randomisation 2 (R2).

Objectives:

The primary objectives of this study are to determine:

* Early treatment cessation (within 60 days of randomisation) for standard versus frailty-adjusted up-front dosing

* Progression-free survival (PFS, from maintenance randomisation) for lenalidomide + placebo (R) versus lenalidomide + ixazomib (R+I)

The secondary objectives of this study are to assess progression-free survival (PFS) for standard versus frailty-adjusted up-front dosing reductions, time to progression, time to 2nd PFS event (PFS2), overall survival (OS), survival after progression, deaths within 12 months of R1, overall response rate (ORR), attainment of ≥VGPR, attainment of MRD negativity, duration of response, time to improved response, time to next treatment, treatment compliance and total amount of therapy delivered, toxicity \& safety including the incidence of SPMs, Quality of Life (QoL), cost effectiveness of standard versus frailty-adjusted up-front dosing of IRD and cost-effectiveness of R + I versus R.

Exploratory objectives are prospective validation of a novel frailty risk score (UK-MRA Myeloma Risk Profile - MRP), usefulness of Karnofsky Performance Status (PS), and association of molecular subgroups with response, PFS and OS.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
740
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
Randomisation 1: Number of participants with early treatment cessationWithin 60 days of Randomisation 1

Early treatment cessation is defined as a binary endpoint. Participants will be defined to have experienced an event if they die, progress, or are withdrawn from treatment (by a treating clinician) or withdraw consent for trial treatment, within 60 days of Randomisation 1.

Randomisation 2: Progression-free survival (PFS-R2)The time from the date of Randomisation 2 to the date of first documented evidence of disease progression or death from any cause, up to 120 months

PFS-R2 is defined as the time from Randomisation 2 to the time of first documented evidence of disease progression or death from any cause. Individuals who are lost to follow-up or progression-free at the time of analysis will be censored at their last known date to be alive and progression-free. Disease progression is defined according to the IMWG Uniform Response Criteria for Multiple Myeloma.

Secondary Outcome Measures
NameTimeMethod
Deaths within 12 months of Randomisation 1 (R1)Within 12 months of Randomisation 1

Deaths within 12 months of Randomisation 1 is defined as a binary endpoint. Participants will be defined to have experienced an event if they die within 12 months of Randomisation 1.

Progression-free survival (PFS-R1)The time from the date of Randomisation 1 to the date of first documented evidence of disease progression or death from any cause, up to 120 months

PFS-R1 is defined as the time from R1 to the time of first documented evidence of disease progression or death from any cause. Individuals who are lost to follow-up or progression-free at the time of analysis will be censored at their last known date to be alive and progression-free. Disease progression is defined according to the IMWG Uniform Response Criteria for Multiple Myeloma.

Overall response rate (ORR)From the date of Randomisation 1 to the end of 12 cycles of induction treatment (each induction cycle is 28 days)

Overall response rate is defined as a categorical outcome consisting of whether a participant had Stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), Minimal Response (MR), Stable Disease (SD) or Progressive Disease (PD) at the end of induction according to the IMWG Uniform Response Criteria for Multiple Myeloma.

Duration of response (DoR)The time from the date of the first observation of response ≥ Partial Response following Randomisation 1, to the date of first documented evidence of disease progression or death confirmed related to progression, up to 120 months

Duration of response is defined as the time from the first observation of response ≥ Partial Response (PR), following Randomisation 1, to the time of first documented evidence of disease progression or death confirmed related to progression. Individuals who are lost to follow-up, or still alive and progression-free at the time of analysis will be censored at their last known date to be alive and progression-free. Individuals whose cause of death is unrelated to disease progression will be censored at their date of death.

Overall survival (OS)The time from the date of randomisation to the date of death from any cause, up to 120 months

Overall survival is defined separately for each randomisation. In each case it is the time from randomisation to the time of death from any cause. Individuals who are lost to follow-up or still alive at the time of analysis will be censored at their last known date to be alive.

Survival after progressionThe date of first documented evidence of disease progression to the date of death from any cause, up to 120 months

Survival after progression is defined from the date of first documented evidence of disease progression to the date of death from any cause. Individuals who are lost to follow-up or alive at the time of analysis will be censored at their last known date to be alive following their first documented evidence of disease progression.

Time to disease progressionThe time from the date of randomisation to the date of first documented evidence of disease progression, up to 120 months

Time to disease progression is defined for both Randomisation 1 and Randomisation 2 as the time from randomisation to the date of first documented evidence of disease progression. Participants who die prior to documentation of disease progression will be censored in the analysis at their date of death. Participants not reaching disease progression at the time of analysis will be censored at the last date known to be progression-free. Participants dying from causes not primarily due to progression will also be censored.

Progression-free survival two (PFS2)The time from the date of randomisation to the date of the second documented disease progression, up to 120 months

For both Randomisation 1 and Randomisation 2, progression-free survival two is defined as the time from randomisation to the time of the second documented disease progression. Individuals who are lost to follow-up or second progression-free at the time of analysis will be censored at their last known date to be alive and second progression-free.

Attainment of Minimal Residual Disease (MRD) negativityFrom the date of Randomisation 1 to the end of 12 cycles of induction treatment (each induction cycle is 28 days); and 12 months after the date of Randomisation 2

Attainment of Minimal Residual Disease (MRD) negativity is defined as a binary endpoint. MRD negativity will be determined at the end of induction therapy and 12 months post Randomisation 2 according to the IMWG MRD criteria.

Treatment compliance and total amount of therapy deliveredNumber of induction and maintenance cycles a participant received (each cycle of induction or maintenance is 28 days), until disease progression, up to 120 months

In the first instance treatment compliance is defined as a binary outcome i.e. did the participant receive 12 cycles of induction treatment. The total amount of therapy delivered will be first defined as the number of induction and maintenance cycles which the participant received. This may be extended to consider the percentage of protocol dose delivered. For each treatment (ixazomib, lenalidomide, dexamethasone) this will be defined as the total dose received in a cycle compared to the total dose the participant should have received in the cycle without modifications, averaged across all cycles of treatment.

EORTC QLQ-C30_questionnaireRandomisation 1; after cycles 2, 4, 6 and 12 of induction treatment (each induction cycle is 28 days); after cycles 6 and 12 of maintenance treatment (each maintenance cycle is 28 days)

European Organization for the Research \& Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) is used to measure patient-assessed quality of life (QoL) at R1, after cycles 2, 4, 6 \& 12 of induction, and after cycles 6 and 12 of maintenance (all cycles are 28 days).

The QLQ-C30 comprises multi-item scales \& single-item measures: 5 functional scales (physical, role, cognitive, emotional, social), 3 symptom scales (fatigue, pain, nausea \& vomiting), a global health status/QoL scale, \& 6 single items assessing additional symptoms (dyspnoea, loss of appetite, insomnia, constipation \& diarrhoea). All scales \& single-item measures range in score from 0 to 100. A high scale score represents a higher response level: a high score for a functional scale represents a high/healthy level of functioning, a high score for the global health status/QoL represents a high quality of life, but a high score for a symptom scale/item represents a high level of symptomatology/problems.

Attainment of ≥VGPRFrom the date of Randomisation 1 to the end of 12 cycles of induction treatment (each induction cycle is 28 days)

Attainment of ≥VGPR is defined as a binary outcome of whether a participant had ≥VGPR (VGPR, CR, sCR) or \<VGPR (PR, MR, SD, PD) at the end of induction, according to the IMWG Uniform Response Criteria for Multiple Myeloma.

Time to next treatmentThe time from the date of Randomisation 1 to the start date of the next line of treatment or death from any cause, up to 120 months

Time to next treatment is defined as the time from Randomisation 1 to the start date of the next line of treatment or death from any cause.

Time to improved responseThe time from the date of Randomisation 2 to the date the response category is first improved, up to 120 months

Time to improved response is defined as the time from the date of Randomisation 2 to the date the response category is first improved based on the Modified International Uniform Response Criteria for Multiple Myeloma. Subjects without any improvement of the baseline status at Randomisation 2 will be censored at the last date of response assessment.

EORTC QLQ-MY20_questionnaireRandomisation 1; after cycles 2, 4, 6 and 12 of induction treatment (each induction cycle is 28 days); after cycles 6 and 12 of maintenance treatment (each maintenance cycle is 28 days)

The European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire Multiple Myeloma Module (EORTC QLQ-MY20) is used to measure patient-assessed quality of life (QoL) at Randomisation 1, after cycles 2, 4, 6 and 12 of induction treatment (each induction cycle is 28 days), and after cycles 6 and 12 of maintenance treatment (each maintenance cycle is 28 days).

The EORTC QLQ-MY20 comprises 20 questions that address four myeloma-specific QoL domains: Disease Symptoms, Side Effects of Treatment, Future Perspectives, and Body Image. Disease Symptoms, Side Effects of Treatment, and Future Perspectives are all multi-item scales, and Body Image is a single-item scale. Domain scores are averaged and transformed linearly to a score ranging from 0-100. A high score for Disease Symptoms and Side Effects of Treatment represents a high level of symptomatology or problems, whereas a high score for Future Perspective and Body Image represents better outcomes.

Incidence of treatment-emergent adverse events (Toxicity and safety, including incidence of second malignancies)Baseline, end of each induction cycle (each induction cycle is 28 days), end of each maintenance cycle (each maintenance cycle is 28 days), until disease progression, up to 120 months

Toxicity \& safety, including incidence of second malignancies Toxicity and safety will be reported based on the adverse events, as graded by CTCAE V5 and determined by routine clinical assessments at each centre. The number of SAEs will be reported according to MedDRA System Organ Class. All second primary malignancies will be reported based on information collected on the Case Report Form.

EQ-5D-3L_questionnaireRandomisation 1; after cycles 2, 4, 6 and 12 of induction treatment (each induction cycle is 28 days); after cycles 6 and 12 of maintenance treatment (each maintenance cycle is 28 days)

The EuroQol 5 Dimension 3 Level questionnaire (EQ-5D-3L) will be used to measure participant-assessed quality of life at Randomisation 1, after cycles 2, 4, 6 and 12 of induction treatment (each induction cycle is 28 days), and after cycles 6 and 12 of maintenance treatment (each maintenance cycle is 28 days).

The EQ-5D-3L descriptive system comprises the following five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: no problems, some problems, and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions. This decision results into a 1-digit number that expresses the level selected for that dimension. The digits for the five dimensions can be combined into a 5-digit number that describes the patient's health state.

Trial Locations

Locations (109)

North Devon District Hospital

🇬🇧

Barnstaple, United Kingdom

Queen Elizabeth Hospital

🇬🇧

Birmingham, United Kingdom

Wrightington Hosptial

🇬🇧

Appley Bridge, United Kingdom

Furness General Hospital

🇬🇧

Barrow In Furness, United Kingdom

Dorset County Hospital

🇬🇧

Dorchester, United Kingdom

Colchester General Hospital

🇬🇧

Colchester, United Kingdom

Ninewells Hospital

🇬🇧

Dundee, United Kingdom

Royal United Hospital

🇬🇧

Bath, United Kingdom

Basingstoke and North Hampshire Hospital

🇬🇧

Basingstoke, United Kingdom

Pilgrim Hospital

🇬🇧

Boston, United Kingdom

Royal Bolton Hospital

🇬🇧

Bolton, United Kingdom

St Richard's Hospital

🇬🇧

Chichester, United Kingdom

Russells Hall Hospital

🇬🇧

Dudley, United Kingdom

Medway Maritime Hospital

🇬🇧

Gillingham, United Kingdom

Royal Surrey County Hospital

🇬🇧

Guildford, United Kingdom

Withybush General Hospital

🇬🇧

Haverfordwest, United Kingdom

Salford Royal Hospital

🇬🇧

Salford, United Kingdom

Scarborough General Hospital

🇬🇧

Scarborough, United Kingdom

Scunthorpe General Hospital

🇬🇧

Scunthorpe, United Kingdom

Sunderland Royal Hospital

🇬🇧

Sunderland, United Kingdom

Good Hope Hospital

🇬🇧

Sutton Coldfield, United Kingdom

St George's Hospital

🇬🇧

Tooting, United Kingdom

Singleton Hospital

🇬🇧

Swansea, United Kingdom

Tunbridge Wells Hospital

🇬🇧

Tunbridge Wells, United Kingdom

Aberdeen Royal Infirmary

🇬🇧

Aberdeen, United Kingdom

Nevill Hall Hospital

🇬🇧

Abergavenny, United Kingdom

Ysbyty Gwynedd

🇬🇧

Bangor, United Kingdom

Belfast City Hospital

🇬🇧

Belfast, United Kingdom

Birmingham Heartlands Hospital

🇬🇧

Birmingham, United Kingdom

Royal Blackburn Hospital

🇬🇧

Blackburn, United Kingdom

Blackpool Victoria Hospital

🇬🇧

Blackpool, United Kingdom

Bradford Royal Infirmary

🇬🇧

Bradford, United Kingdom

Royal Bournemouth Hospital

🇬🇧

Bournemouth, United Kingdom

Bristol Haematology and Oncology Centre

🇬🇧

Bristol, United Kingdom

Southmead Hospital

🇬🇧

Bristol, United Kingdom

Queen's Hospital

🇬🇧

Romford, United Kingdom

Kent and Canterbury Hospital

🇬🇧

Canterbury, United Kingdom

Chelmsford & Essex Hospital

🇬🇧

Chelmsford, United Kingdom

Cheltenham General Hospital

🇬🇧

Cheltenham, United Kingdom

Countess of Chester Hospital

🇬🇧

Chester, United Kingdom

University Hospital Coventry

🇬🇧

Coventry, United Kingdom

Croydon University Hospital

🇬🇧

Croydon, United Kingdom

Royal Derby Hospital

🇬🇧

Derby, United Kingdom

Western General Hospital

🇬🇧

Edinburgh, United Kingdom

Royal Devon & Exeter Hospital

🇬🇧

Exeter, United Kingdom

Gloucestershire Royal Hospital

🇬🇧

Gloucester, United Kingdom

Grantham and District Hospital

🇬🇧

Grantham, United Kingdom

Diana Princess of Wales Hospital

🇬🇧

Grimsby, United Kingdom

Calderdale Royal Hospital

🇬🇧

Halifax, United Kingdom

Harrogate District Hospital

🇬🇧

Harrogate, United Kingdom

Hereford County Hospital

🇬🇧

Hereford, United Kingdom

Huddersfield Royal Infirmary

🇬🇧

Huddersfield, United Kingdom

Raigmore Hospital

🇬🇧

Inverness, United Kingdom

Ipswich Hospital

🇬🇧

Ipswich, United Kingdom

Airedale Hospital

🇬🇧

Keighley, United Kingdom

Westmorland General Hospital

🇬🇧

Kendal, United Kingdom

Kettering General Hospital

🇬🇧

Kettering, United Kingdom

Kidderminster Hospital & Treatment Centre

🇬🇧

Kidderminster, United Kingdom

Victoria Hospital

🇬🇧

Kirkcaldy, United Kingdom

Royal Lancaster Infirmary

🇬🇧

Lancaster, United Kingdom

St James's University Hospital

🇬🇧

Leeds, United Kingdom

Leicester Royal Infirmary

🇬🇧

Leicester, United Kingdom

Lincoln County Hospital

🇬🇧

Lincoln, United Kingdom

Aintree University Hospital

🇬🇧

Liverpool, United Kingdom

Royal Liverpool Hospital

🇬🇧

Liverpool, United Kingdom

King's College Hospital

🇬🇧

London, United Kingdom

Queen Elizabeth Hospital Greenwich

🇬🇧

London, United Kingdom

St Bartholomew's Hospital

🇬🇧

London, United Kingdom

University College Hospital

🇬🇧

London, United Kingdom

University Hospital Lewisham

🇬🇧

London, United Kingdom

Manchester Royal Infirmary

🇬🇧

Manchester, United Kingdom

Maidstone Hospital

🇬🇧

Maidstone, United Kingdom

James Cook University Hospital

🇬🇧

Middlesbrough, United Kingdom

Freeman Hospital

🇬🇧

Newcastle, United Kingdom

Royal Gwent Hospital

🇬🇧

Newport, United Kingdom

North Tyneside General Hospital

🇬🇧

North Shields, United Kingdom

Nottingham City Hospital

🇬🇧

Nottingham, United Kingdom

Royal Oldham Hospital

🇬🇧

Oldham, United Kingdom

Princess Royal University Hospital

🇬🇧

Orpington, United Kingdom

Peterborough City Hospital

🇬🇧

Peterborough, United Kingdom

Derriford Hospital

🇬🇧

Plymouth, United Kingdom

Whiston Hospital

🇬🇧

Prescot, United Kingdom

Royal Preston Hospital

🇬🇧

Preston, United Kingdom

Royal Berkshire Hospital

🇬🇧

Reading, United Kingdom

Alexandra Hospital

🇬🇧

Redditch, United Kingdom

Glan Clwyd Hospital

🇬🇧

Rhyl, United Kingdom

Salisbury District Hospital

🇬🇧

Salisbury, United Kingdom

Royal Hallamshire Hospital

🇬🇧

Sheffield, United Kingdom

Royal Shrewsbury Hospital

🇬🇧

Shrewsbury, United Kingdom

Southampton General Hospital

🇬🇧

Southampton, United Kingdom

St Helens Hospital

🇬🇧

St Helens, United Kingdom

Stafford County Hospital

🇬🇧

Stafford, United Kingdom

Stepping Hill Hospital

🇬🇧

Stockport, United Kingdom

Royal Stoke University Hospital

🇬🇧

Stoke-on-Trent, United Kingdom

Torbay District General Hospital

🇬🇧

Torquay, United Kingdom

Royal Cornwall Hospital

🇬🇧

Truro, United Kingdom

Hillingdon Hospital

🇬🇧

Uxbridge, United Kingdom

Pinderfields General Hospital

🇬🇧

Wakefield, United Kingdom

Warwick Hospital

🇬🇧

Warwick, United Kingdom

Sandwell General Hospital

🇬🇧

West Bromwich, United Kingdom

Royal Albert Edward Infirmary

🇬🇧

Wigan, United Kingdom

Royal Hampshire County Hospital

🇬🇧

Winchester, United Kingdom

New Cross Hospital

🇬🇧

Wolverhampton, United Kingdom

Worcestershire Royal Hospital

🇬🇧

Worcester, United Kingdom

Worthing Hospital

🇬🇧

Worthing, United Kingdom

Wrexham Maelor Hospital

🇬🇧

Wrexham, United Kingdom

York Hospital

🇬🇧

York, United Kingdom

Guy's Hospital

🇬🇧

London, United Kingdom

Castle Hill Hospital

🇬🇧

Hull, United Kingdom

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