PLS240 for Secondary Hyperparathyroidism

(PATH-2 Trial)

No longer recruiting at 83 trial locations

Overseen By Pathalys Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Pathalys Pharma

Must be taking: Vitamin D sterols

Disqualifiers: Primary hyperparathyroidism, Unstable condition, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called PLS240 for individuals with end-stage kidney disease who are on hemodialysis and have secondary hyperparathyroidism (a condition where the parathyroid glands produce too much hormone due to kidney failure). The trial aims to evaluate the effectiveness and safety of PLS240 compared to a placebo (a dummy treatment with no active ingredients). Participants will receive either the treatment or a placebo for the first 27 weeks, followed by an opportunity for all to try the treatment for another 26 weeks. This trial may suit those who have been on regular dialysis three times a week for at least three months and are managing their condition with stable doses of vitamin D and other medications. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial requires that participants stay on a stable dose of certain medications, like active Vitamin D sterols, phosphate binders, and calcium supplements, throughout the study. If you're taking these, you won't need to stop, but you must keep the dose stable.

Is there any evidence suggesting that PLS240 is likely to be safe for humans?

Studies have shown that PLS240 has already been tested in people, providing existing information about its safety. In earlier research, patients with secondary hyperparathyroidism, a condition where the body produces too much parathyroid hormone, received PLS240 while on dialysis. Most tolerated the treatment well, experiencing no major problems.

However, like any medication, side effects can occur. This trial is in a later stage, indicating that the treatment has passed initial safety checks. Prospective participants should discuss any safety concerns with the study team. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about PLS240 for treating secondary hyperparathyroidism because it offers a new approach compared to existing treatments like vitamin D analogs, calcimimetics, and phosphate binders. PLS240 is unique because it targets the parathyroid hormone (PTH) pathways with potentially higher specificity, which could lead to more effective management of PTH levels. This targeted action might result in fewer side effects compared to current therapies, making it a promising option for patients who struggle with the side effects of existing treatments. Plus, the possibility of improved patient outcomes with PLS240 has generated significant interest in the medical community.

What evidence suggests that PLS240 might be an effective treatment for secondary hyperparathyroidism?

Research shows that PLS240, which participants in this trial may receive, effectively treats secondary hyperparathyroidism (SHPT) in patients with severe kidney disease on dialysis. Studies have found that PLS240 can reduce parathyroid hormone levels by at least 30%. This reduction is crucial because high levels of this hormone can cause bone and heart problems. PLS240 mimics calcium in the body to help regulate hormone levels. Previous research has demonstrated that PLS240 is both effective and safe for patients with these conditions. These findings provide confidence that PLS240 can effectively manage SHPT symptoms. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

Adults aged 18-80 on hemodialysis for end-stage kidney disease with secondary hyperparathyroidism can join. They must have specific levels of parathyroid hormone and calcium, stable doses of certain medications, and agree to contraception if applicable. Excluded are those with primary hyperparathyroidism, recent heart issues or seizures, certain cancers within the last two years, uncontrolled diabetes or hypertension, and anyone who's been in other drug trials recently.

Inclusion Criteria

Voluntarily given written informed consent to participate in this study

I am a woman who cannot become pregnant or will avoid pregnancy during the study.

I am a man willing to use effective birth control and not donate sperm during and for 2 weeks after treatment.

Exclusion Criteria

Continues to meet Exclusion Criterion #5

I or my family have a history of long QT syndrome.

I am scheduled for a kidney transplant from a living donor during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Double-Blind Treatment

Participants are randomly assigned to receive dose-titrated PLS240 or placebo for 27 weeks

27 weeks

Multiple visits (in-person)

Open-Label Extension

Participants receive dose-titrated PLS240 for an additional 26 weeks

26 weeks

Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• PLS240

Trial Overview The trial tests PLS240's effectiveness and safety against a placebo in patients undergoing hemodialysis due to severe kidney disease causing high parathyroid hormone levels. It has two phases: a double-blind phase where participants get either PLS240 or placebo randomly for 27 weeks; followed by an open-label extension where all receive PLS240 for another 26 weeks.

How Is the Trial Designed?

Group I: Open-Label Extension Phase PLS240 Experimental Treatment 1 Intervention

After completion of the Double-Blind Phase, all participants will have the opportunity to enroll in the 26 week Open-Label extension, where they will receive PLS240.

Group II: Double-Blind Phase PLS240 Experimental Treatment 1 Intervention

Group III: Double-Blind Phase Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Pathalys Pharma

Lead Sponsor

Trials

2

Recruited

770+

Launch Therapeutics

Collaborator

Trials

2

Recruited

770+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05836220

Study Details | NCT05836220 | Parathyroid Hormone (PTH ... This study is to evaluate the efficacy and safety of PLS240 in patients with hemodialysis-dependent end stage kidney disease (ESKD) and secondary ...

2. withpower.com withpower.com/trial/phase-3-neoplasm-metastasis-4-2023-09bb1

PLS240 for Secondary Hyperparathyroidism (PATH-2 Trial) This trial is testing a medication called PLS240 to help people with severe kidney disease who are on dialysis and have a related gland problem.

3. cochranelibrary.com cochranelibrary.com/central/doi/10.1002/central/CN-02454813/

The Phase 3 Program of Upacicalcet/PLS240 for ... The Phase 3 Program of Upacicalcet/PLS240 for the Treatment Of Secondary Hyperparathyroidism in Patients on Hemodialysis. Danoff T, Szczech L.

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-pls240-for-treating-secondary-hyperparathyroidism-in-patients-with-end-stage-kidney-disease-on-hemodialysis/

Study on the Effectiveness and Safety of PLS240 ... The main goal is to evaluate the effectiveness of PLS240 in reducing parathyroid hormone levels by at least 30% in individuals with secondary ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37696667/

Efficacy and Safety of Upacicalcet in Hemodialysis Patients ... Conclusions: Upacicalcet, a novel injectable calcimimetic, is effective and safe for secondary hyperparathyroidism patients receiving HD.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05832931

Study Details | NCT05832931 | Parathyroid Hormone (PTH ... This study is to evaluate the efficacy and safety of PLS240 in patients with hemodialysis-dependent end stage kidney disease (ESKD) and secondary ...

7. ctv.veeva.com ctv.veeva.com/study/parathyroid-hormone-pth-attenuation-trial-in-hemodialysis2

Parathyroid Hormone (PTH) Attenuation Trial in Hemodialysis-2 This study is to evaluate the efficacy and safety of PLS240 in patients with hemodialysis-dependent end stage kidney disease (ESKD) and ...