Patiromer for Pediatric Hyperkalemia

Recruiting at 54 trial locations

Overseen By EMERALD-2 Clinical Study Team

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Vifor Pharma, Inc.

Must not be taking: Digoxin, Bronchodilators, Theophylline, others

Disqualifiers: Preterm birth, Renal conditions, Severe GI issues, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 4 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called patiromer (also known as Veltassa or Patiromer sorbitex calcium) to evaluate its safety and effectiveness for children under 12 with hyperkalemia (high potassium levels in the blood). Researchers aim to understand how the drug affects potassium levels and how well children tolerate it. This study suits children who regularly experience high potassium levels and can take medication through regular feeding or feeding tubes. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that if you are taking certain medications like RAAS inhibitors, beta blockers, fludrocortisone, or diuretics, you must be on a stable dose for at least 14 days before starting the trial. If you are taking other medications like digoxin or bronchodilators, those doses also need to be stable for 14 days before the trial and should not change during the trial.

Is there any evidence suggesting that patiromer is likely to be safe for children under 12 years of age?

Research has shown that patiromer is safe for children aged 2 to 18 with high potassium levels. In studies, 23 children took patiromer without experiencing serious side effects. The FDA has approved this treatment for children 12 and older, indicating its general safety. While more information is needed for younger children, current evidence is encouraging. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hyperkalemia?

Researchers are excited about patiromer for treating pediatric hyperkalemia because it offers a novel approach compared to standard treatments like sodium polystyrene sulfonate. Unlike traditional options, patiromer works by binding potassium in the gut, which can help lower potassium levels more effectively. Additionally, patiromer has the potential to be better tolerated with fewer gastrointestinal side effects, making it a more appealing option for young children. This targeted action and improved safety profile are why researchers are hopeful about its potential benefits for kids with high potassium levels.

What evidence suggests that patiromer might be an effective treatment for pediatric hyperkalemia?

Research shows that patiromer effectively treats high potassium levels in adults and older children. It attaches to potassium in the gut, lowering blood potassium levels. Studies have found it safe and successful in reducing potassium levels in patients. This treatment is approved for use in individuals over 12 years old with high potassium. The current trial examines patiromer's effectiveness in younger children, with ongoing research to determine if it works similarly across all ages. ^{2 4 6 7 8}

Who Is on the Research Team?

Julian Platon, MD, PhD

Principal Investigator

Vifor Pharma, Inc.

Are You a Good Fit for This Trial?

This trial is for children under 6 with hyperkalemia, a condition of high potassium levels in the blood. They must be on stable doses of certain medications if used and able to take food and medicine regularly, including through feeding tubes. Infants born preterm or those with severe kidney issues, hypersensitivity to patiromer, active cancer, significant GI conditions affecting drug transit, or recent investigational drug use are excluded.

Inclusion Criteria

Parent(s) or legally authorised representative(s) or another appropriate person delegated by the legally authorised representatives must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day; accurately and reliably dispense investigational product as directed

I am under 12 years old and will not turn 12 during the first 28 days of the trial.

My child is under 12 and has high potassium levels.

Exclusion Criteria

My baby was not born before 37 weeks of pregnancy.

Use of any investigational product for an unapproved indication within 30 days prior to screening or within 5 half-lives, whichever is longer

I have kidney issues like dialysis, narrowed arteries, recent injury, or past insufficiency but not chronic disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive patiromer for a 4-week pharmacodynamic/dose-ranging period

4 weeks

Visits on Day 3, Day 7, Day 14, and Day 28

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Participants are monitored for long-term safety and effectiveness

Up to 52 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Patiromer

Trial Overview The study tests the effects and safety of patiromer in treating hyperkalemia in young children. It aims to understand how well the drug works (pharmacodynamics), its tolerability, and any side effects it may cause during treatment.

How Is the Trial Designed?

Group I: Patiromer Experimental Treatment 1 Intervention

4-week pharmacodynamic /dose-ranging period Cohort 1: 6 to less than (\<)12 years of age Cohort 2: 2 to \<6 years of age Cohort 3: 0 to \<2 years of age; In Cohort 3, a minimum of 3 study participants will be assessed in the subgroup of 0 to \<6 months and another 3 study participants in the subgroup 6 to \<24 months of age.

Patiromer is already approved in United States, European Union for the following indications:

Approved in United States as Veltassa for:

• Hyperkalemia in adults and geriatric patients

Approved in European Union as Veltassa for:

• Hyperkalemia

Who Is Running the Clinical Trial?

Vifor Pharma, Inc.

Lead Sponsor

Trials

6

Recruited

1,600+

Published Research Related to This Trial

Patiromer is a cation-exchange polymer approved for treating hyperkalaemia in adults, offering a safer alternative to traditional cation-exchange resins, which can cause serious side effects like gastrointestinal necrosis.

Unlike other treatments, patiromer can be used in a broader range of patients, not just those with severe kidney issues or on dialysis, making it a more versatile option in managing high potassium levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

▼Patiromer for the management of hyperkalaemia. [2018]

Pre-treatment with patiromer effectively reduces potassium levels in infant formulas, with a more significant decrease observed at higher doses, indicating its potential as a treatment for hyperkalemia in infants.

Calcium levels increased in the formulas after patiromer treatment, primarily due to the ion exchange process, which suggests that patiromer could be a safe and effective option for managing potassium levels in infants with chronic kidney disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of infant formula with patiromer dose dependently decreases potassium concentration. Paloian, NJ., Bowman, B., Bartosh, SM.[2020]

Patiromer is an effective treatment for hyperkalaemia, significantly reducing serum potassium levels in patients with chronic kidney disease and allowing them to continue using RAAS inhibitors for up to 52 weeks.

The medication is generally well tolerated, with a low risk of causing low potassium levels (hypokalaemia), although mild to moderate gastrointestinal disorders and low magnesium levels (hypomagnesaemia) were the most common side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patiromer: A Review in Hyperkalaemia. Blair, HA.[2022]

Patiromer significantly reduced the risk of recurrent hyperkalemia by 48% within 8 weeks in patients with chronic kidney disease (CKD) on RAASi treatment, with a number needed to treat (NNT) of 2.1 to prevent one hyperkalemic event.

The use of patiromer not only improves patient outcomes by reducing hospitalizations and mortality associated with hyperkalemia but also leads to cost savings of approximately CHF 303 per patient over 8 weeks in the Swiss healthcare system.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cost-Effectiveness of Treating Patients with Chronic Kidney Disease and Prior Hyperkalemia with Renin-Angiotensin-Aldosterone System Inhibitor and Patiromer: A Swiss Public Healthcare Perspective. Fischer, B., Serra, A., Telser, H.[2022]

Patiromer significantly reduced serum potassium levels in patients with chronic kidney disease and hyperkalaemia, maintaining these reductions effectively over 8 weeks compared to placebo, allowing more patients to continue their RAASi therapy.

The treatment was generally well tolerated, with no serious adverse events reported, although mild to moderate constipation and low magnesium levels were common side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patiromer: A Review in Hyperkalaemia. Kim, ES., Deeks, ED.[2018]

Patiromer, approved by the FDA in 2015, is the first new treatment for hyperkalemia in 50 years, effectively lowering serum potassium levels in patients with varying degrees of hyperkalemia, including those with chronic kidney disease and heart failure.

The drug works by exchanging calcium for potassium in the colon, and while it has some gastrointestinal side effects, these are generally manageable and do not lead to significant treatment discontinuation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patiromer: a significant advance in the management of hyperkalemia. Paton, DM.[2017]

Citations

1. fda.gov fda.gov/media/175678/download

Application 205739 S-038|Veltassa|Page 1 of 50 mechanism of action, Veltassa is expected to be effective in treating hyperkalemia in pediatric patients. ... (Patiromer Sorbitex Calcium) for Oral Suspension in ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9292454/

Clinical Efficacy, Safety, Tolerability, and Real-World Data ... Patiromer has been shown to be safe and effective in patients with hyperkalemia, but there are still many questions to be answered and different populations ...

3. veltassa.com veltassa.com/pi

HIGHLIGHTS OF PRESCRIBING INFORMATION These ... The safety and effectiveness of Veltassa for lowering serum potassium levels have been established in pediatric patients ages 12 years and older. Use of ...

4. cda-amc.ca cda-amc.ca/sites/default/files/cdr/complete/SR0665-combined.pdf

Patiromer (Veltassa) The objective of this report was to perform a systematic review of the beneficial and harmful effects of patiromer (as patiromer sorbitex calcium: 8.4 g, 16.8 g ...

5. tga.gov.au tga.gov.au/sites/default/files/auspar-patiromer-sorbitex-calcium-190911.pdf

Australian Public Assessment Report for patiromer sorbitex ... Veltassa is indicated for the treatment of hyperkalemia. Patiromer sorbitex calcium was approved by the European Medicines Agency (EMA) in. July ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2023/205739s038lbl.pdf

VELTASSA (patiromer) - accessdata.fda.gov Veltassa is a potassium binder indicated for the treatment of hyperkalemia in adults and pediatric patients ages 12 years and older. --------------------------- ...

7. ema.europa.eu ema.europa.eu/en/documents/rmp/veltassa-epar-risk-management-plan_en.pdf

eu risk management plan for veltassa® (patiromer) - EMA Safety data is available for 23 paediatric study participants, aged 6 to less than 18 years. (Study RLY5016-206p (EMERALD)). The exposure for ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT03087058

NCT03087058 | Pharmacodynamic & Safety of Patiromer ... Another purpose of the study is to evaluate the safety and tolerability of patiromer in children 2 - < 18 years of age with CKD and hyperkalemia. Detailed ...