Chronic Kidney Disease + Hyperkalaemia +/- Heart Failure Clinical Trial
— APPETIZEOfficial title:
Non-Interventional, Exploratory, Phase IV, Single-Blind, Cross-Sectional, Randomised, Cross-over Study Evaluating Patient Palatability and Preference of 3 Potassium Binders in Patients With Chronic Kidney Disease & Hyperkalaemia (APPETIZE).
| Verified date | November 2023 |
| Source | AstraZeneca |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
This non-interventional, Phase IV, exploratory, cross-over, randomised, single-blind, active comparator-controlled study has been designed to measure the palatability and preference of Lokelma® versus Veltassa® versus S/CPS in patients with dialysis and non-dialysis chronic kidney disease (CKD) and hyperkalaemia (HK). The sponsor hypothesizes that palatability, in terms of taste, texture, smell, and mouthfeel, will score higher (better) for Lokelma when compared with Veltassa and S/CPS.
| Status | Completed |
| Enrollment | 147 |
| Est. completion date | January 12, 2022 |
| Est. primary completion date | January 12, 2022 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 130 Years |
| Eligibility | Inclusion Criteria: 1. Participants must be adults aged =18 years, at the time of signing the informed consent. 2. Participants should have CKD defined by having an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2 (calculated using CKD-EPI equation) measured twice at least 90 days apart. (The eGFR should be measured when the participant is considered to be in a steady state without recent changes in volume status, medications that could impact the result (eg, nonsteroidal anti-inflammatory drugs, aminoglycosides, co-trimoxazole), or changes in dietary protein intake.) 3. Prevalent HK with serum K+ >5 mmol/L. 4. Male and/or female 5. Capable of giving signed informed consent as described in Appendix A which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Informed consent must be obtained prior to any study-specific procedures performed. Exclusion Criteria: 1. Screening serum K+ value which, in the opinion of the investigator, requires immediate medical intervention (ie, cannot wait until after tasting procedures). 2. As judged by the investigator, any evidence of any condition which in the investigator's opinion makes it undesirable for the participant to participate in the study. 3. Known history of drug or alcohol abuse within 6 months of screening. 4. History of QT prolongation associated with other medications that required discontinuation of that medication, including congenital long QT syndrome. 5. Symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia. Participants with atrial fibrillation controlled by medication are permitted. 6. Have a life expectancy of <6 months. 7. 12-lead ECG with reported QTcF >550 msec at screening. 8. Are current smoker. 9. Have mouth ulcers/mouth infection, respiratory infection, nasal congestion, or other condition, medication, or procedure which may interfere with sense of smell or taste, in opinion of the investigator. 10. Participants currently prescribed a K+ binder at time of screening/enrolment. 11. Participants unable to hold other oral medications from 3 hours prior to the start of tasting through 3 hours after the end of tasting. 12. Current participation or participation within the previous 28 days in another clinical study with an investigational product administered. 13. Participants with a known hypersensitivity to Lokelma, Veltassa, or S/CPS or any of the excipients of the NIMPs. 14. Involvement in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study site). 15. Judgment by the investigator that the participant should not participate in the study if the participant is unlikely to comply with study procedures, restrictions and requirements. 16. Previous enrolment or randomisation in the present study. 17. For women only - currently pregnant (confirmed with positive pregnancy test) or breast-feeding. 18. Participants unable to read the local language and therefore unable to complete the questionnaires. |
| Country | Name | City | State |
|---|---|---|---|
| Canada | Research Site | Greenfield Park | Quebec |
| Canada | Research Site | Montreal | Quebec |
| Canada | Research Site | Quebec | |
| Canada | Research Site | Toronto | Ontario |
| France | Research Site | Amiens | |
| France | Research Site | Boulogne Billancourt Cedex | |
| France | Research Site | Nice | |
| Italy | Research Site | Genova | |
| Italy | Research Site | Parma | |
| Italy | Research Site | Pavia | |
| Spain | Research Site | Barcelona | |
| Spain | Research Site | Córdoba | |
| Spain | Research Site | La Coruña | |
| Spain | Research Site | Madrid | |
| Sweden | Research Site | Eskilstuna | |
| Sweden | Research Site | Stockholm | |
| United States | Research Site | Temple Terrace | Florida |
| United States | Research Site | West Palm Beach | Florida |
| Lead Sponsor | Collaborator |
|---|---|
| AstraZeneca | Calyx, ERT: Clinical Trial Technology Solutions, Labcorp Corporation of America Holdings, Inc, Medidata Solutions |
United States, Canada, France, Italy, Spain, Sweden,
Laureati P, Xu Y, Trevisan M, Schalin L, Mariani I, Bellocco R, Sood MM, Barany P, Sjolander A, Evans M, Carrero JJ. Initiation of sodium polystyrene sulphonate and the risk of gastrointestinal adverse events in advanced chronic kidney disease: a nationwide study. Nephrol Dial Transplant. 2020 Sep 1;35(9):1518-1526. doi: 10.1093/ndt/gfz150. — View Citation
Noel JA, Bota SE, Petrcich W, Garg AX, Carrero JJ, Harel Z, Tangri N, Clark EG, Komenda P, Sood MM. Risk of Hospitalization for Serious Adverse Gastrointestinal Events Associated With Sodium Polystyrene Sulfonate Use in Patients of Advanced Age. JAMA Intern Med. 2019 Aug 1;179(8):1025-1033. doi: 10.1001/jamainternmed.2019.0631. Erratum In: JAMA Intern Med. 2020 Feb 24;: — View Citation
Thomsen RW, Nicolaisen SK, Hasvold P, Sanchez RG, Pedersen L, Adelborg K, Egstrup K, Egfjord M, Sorensen HT. Elevated potassium levels in patients with chronic kidney disease: occurrence, risk factors and clinical outcomes-a Danish population-based cohort study. Nephrol Dial Transplant. 2018 Sep 1;33(9):1610-1620. doi: 10.1093/ndt/gfx312. — View Citation
Zann V, McDermott J, Jacobs JW, Davidson JP, Lin F, Korner P, Blanks RC, Rosenbaum DP. Palatability and physical properties of potassium-binding resin RDX7675: comparison with sodium polystyrene sulfonate. Drug Des Devel Ther. 2017 Sep 6;11:2663-2673. doi: 10.2147/DDDT.S143461. eCollection 2017. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | See primary and secondary endpoints | Overall palatability scores (0-40) between Lokelma and Veltassa, and between Lokelma and S/CPS will be described and compared for all primary and secondary endpoints for: North America, per individual EU countries (where data permit), and over all countries/regions | Tasting visit (day 1) | |
| Other | See primary and secondary endpoints | Patient preference between Lokelma and Veltassa, and between Lokelma and S/CPS will be described and compared for all primary and secondary endpoints for: North America, per individual EU countries (where data permit), and over all countries/regions. | Tasting visit (day 1) | |
| Other | Categories or combinations based on Appeal of each attribute (taste, texture, smell, and mouthfeel) | Appeal test score (1-9) for each attribute (taste, texture, smell, and mouthfeel) will be measured using AdSAM questionnaires using published research and analysed using AdSAM methodology and compare between Lokelma and Veltassa, and between Lokelma and S/CPS for the US, Canada, EU, North America, per individual EU countries (where data permit), and over all countries/regions. | Tasting visit (day 1) | |
| Other | Categories or combinations based on Engagement of each attribute (taste, texture, smell, and mouthfeel) | Engagement test score (1-9) for each attribute (taste, texture, smell, and mouthfeel) will be measured using AdSAM questionnaires team using published research and analysed using AdSAM methodology and compare between Lokelma and Veltassa, and between Lokelma and S/CPS for the US, Canada, EU, North America, per individual EU countries (where data permit), and over all countries/regions | Tasting visit (day 1) | |
| Other | Categories or combinations based on Empowerment of each attribute (taste, texture, smell, and mouthfeel) | Empowerment test score (1-9) for each attribute (taste, texture, smell, and mouthfeel) will be measured using AdSAM questionnaires team using published research and analysed using AdSAM methodology and compare between Lokelma and Veltassa, and between Lokelma and S/CPS for the US, Canada, EU, North America, per individual EU countries (where data permit), and over all countries/regions | Tasting visit (day 1) | |
| Other | Scoring (0-10) of taste, texture, smell, and mouthfeel | Scores of individual palatability attributes (taste, texture, smell, and mouthfeel) will be described individually by countries/regions | Tasting visit (day 1) | |
| Other | Determining specific feelings regarding taste, texture, smell, and mouthfeel using the AdSAM emotional response model | Emotional response of individual palatability attributes (taste, texture, smell, and mouthfeel) will be described individually by countries/regions | Tasting visit (day 1) | |
| Primary | Difference in scores (0-40) for overall palatability of NIMPs | To compare patient-reported overall palatability (composite of taste, texture, smell, and mouthfeel) between Lokelma and Veltassa, and between Lokelma and S/CPS in the United States (US) | Tasting visit (day 1) | |
| Secondary | Difference in scores (0-40) for overall palatability of NIMPs | To compare patient-reported overall palatability (composite of taste, texture, smell, and mouthfeel) between Lokelma and Veltassa, and between Lokelma and S/CPS in Canada | Tasting visit (day 1) | |
| Secondary | Difference in scores (0-40) for overall palatability of NIMPs | To compare patient-reported overall palatability (composite of taste, texture, smell, and mouthfeel) between Lokelma and Veltassa, and between Lokelma and S/CPS in the European Union (EU) | Tasting visit (day 1) | |
| Secondary | Difference in scores for feelings of Appeal (4-36), Engagement (4-36), and Empowerment (4-36) regarding taste overall palatability of NIMPs using the AdSAM emotional response tool | To compare patient-reported emotional response to overall palatability (composite of taste, texture, smell, and mouthfeel) taste between Lokelma and Veltassa, and between Lokelma and S/CPS in the US | Tasting visit (day 1) | |
| Secondary | Difference in scores for feelings of appeal (4-36), engagement (4-36), and empowerment (4-36) regarding overall palatability of NIMPs using the AdSAM emotional response tool | To compare patient-reported emotional response to overall palatability (composite of taste, texture, smell, and mouthfeel) between Lokelma and Veltassa, and between Lokelma and S/CPS in Canada | Tasting visit (day 1) | |
| Secondary | Difference in scores for feelings of appeal (4-36), engagement (4-36), and empowerment (4-36) regarding overall palatability of NIMPs using the AdSAM emotional response tool | To compare patient-reported emotional response to overall palatability (composite of taste, texture, smell, and mouthfeel) between Lokelma and Veltassa, and ,between Lokelma and S/CPS in the EU | Tasting visit (day 1) | |
| Secondary | Scoring of palatability (0-40), Appeal of palatability (4-36), Engagement of palatability (4-36), Empowerment of palatability (4-36), Overall composite emotional strength indicator scores (0-1200), Feelings towards palatability | To describe patient-reported preference for overall palatability (composite of taste, texture, smell, and mouthfeel) (scoring and non-verbal emotional response for how each NIMP made patients feel ofto each NIMP) of 3 currently marketed K+ binders ((of Lokelma, Veltassa, and S/CPS) in the US, Canada, and EU, respectively) | Tasting Visit (day 1) | |
| Secondary | Scoring (0-10), Appeal (9-1), Engagement (9-1), Empowerment (1-9), Overall emotional strength indicator score (0-300), and Feelings towards willingness to take a K+ binder | To describe and compare, based on the overall palatability experience, scoring and emotional response for how willing patients would be to take each K+ binder to help manage their serum potassium (likelihood of adherence) in the US, Canada, and EU | Tasting Visit (day 1) | |
| Secondary | Overall preference ranking of NIMPs (1, 2, or 3) of Lokelma, Veltassa, and S/CPS • Use of Comparative AdSAM Emotional Strength Index and Emotional TempIndicator scores to derive overall preference | To describe patient-reported preference by ranking the NIMPs, and derived preference based on the emotional strength indexindicator scores in the US, Canada, and EU | Tasting visit (day 1) |