TransCon PTH for Hypoparathyroidism

(PaTHway Trial)

No longer recruiting at 26 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Ascendis Pharma Bone Diseases A/S

Must be taking: Calcitriol, Alfacalcidol, Calcium supplements

Must not be taking: Loop diuretics, Digoxin, Lithium, Corticosteroids

Disqualifiers: Diabetes, Liver disease, Cancer, 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: Unknown

Duration: 26 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called TransCon PTH to determine its effectiveness for people with hypoparathyroidism, a condition where the body produces too little parathyroid hormone, resulting in low calcium levels. Participants will receive either the TransCon PTH treatment (a hormone replacement therapy) or a placebo (a substance with no active drug) for the first 26 weeks. Afterward, all participants will receive the actual treatment to evaluate its long-term effects. This trial may suit individuals who have had hypoparathyroidism for over six months and require regular calcium and vitamin D supplements. Participants should be comfortable with daily injections using a pre-filled pen. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

Do I need to stop taking my current medications to join the trial?

The trial does not specify that you must stop taking your current medications, but certain medications are not allowed, such as loop diuretics, phosphate binders, and some osteoporosis therapies. It's best to discuss your specific medications with the trial team to see if they are permitted.

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

Research has shown that TransCon PTH is generally safe for people. In a 52-week study, adults with hypoparathyroidism tolerated TransCon PTH well, with most side effects being mild or moderate and no new safety concerns identified. Another study over 3 years confirmed that the treatment did not cause any new safety issues, indicating that participants did not experience serious problems over time from taking TransCon PTH. Overall, these findings suggest the treatment is safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

TransCon PTH is unique because it offers a new approach to treating hypoparathyroidism by delivering a long-acting form of parathyroid hormone through a once-daily subcutaneous injection. Unlike traditional treatments that often rely on calcium and active vitamin D supplements to manage symptoms, TransCon PTH directly addresses the hormone deficiency at the root of the condition. Researchers are excited about this treatment because it has the potential to improve calcium balance and reduce the need for multiple daily medications, potentially simplifying the management of this chronic condition for patients.

What evidence suggests that TransCon PTH might be an effective treatment for hypoparathyroidism?

This trial will compare TransCon PTH with a placebo for treating hypoparathyroidism. Studies have shown that TransCon PTH can effectively address this condition. In a previous study, patients taking TransCon PTH reported significant improvements in symptoms and overall health. After 52 weeks, the treatment maintained its effectiveness, demonstrating lasting benefits. Patients experienced fewer disease-related symptoms, suggesting an improved quality of daily life. Real-world data further supports that TransCon PTH leads to positive outcomes for those with hypoparathyroidism. Overall, these findings indicate that TransCon PTH is a promising treatment option. ^{1 2 3 5 6}

Who Is on the Research Team?

Aimee D Shu, MD

Principal Investigator

Ascendis Pharma A/S Medical Monitor/Medical Expert

Are You a Good Fit for This Trial?

Adults over 18 with hypoparathyroidism who need certain doses of supplements and have stable thyroid-related treatments can join. They must be able to self-inject medication, not have conditions affecting calcium metabolism other than hypoparathyroidism, and agree to contraception if necessary. Excluded are those with PTH-resistance, recent high-risk cancer, seizures, or severe heart issues.

Inclusion Criteria

25(OH) vitamin D levels of 20-80 ng/mL (49-200 nmol/L) and

BMI 17- 40 kg/m2 at Screening

Able and willing to provide written and signed informed consent in accordance with GCP

Exclusion Criteria

Pregnant or lactating women

I haven't had serious heart issues like heart failure or a heart attack in the last 6 months.

My thyroid cancer is high risk and I need treatment to lower my TSH below 0.2.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive TransCon PTH or placebo for 26 weeks, with doses titrated to optimal levels

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive TransCon PTH with dose adjustments based on individual needs

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• TransCon PTH

Trial Overview The trial tests TransCon PTH against a placebo in adults with hypoparathyroidism. For the first half-year, participants won't know which they're getting as they adjust their dose for optimal effect. Afterward, all get TransCon PTH in a long-term study where doses are tailored individually.

How Is the Trial Designed?

Group I: TransCon PTH Experimental Treatment 1 Intervention

TransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose

Group II: Placebo Placebo Group 1 Intervention

Placebo for TransCon PTH delivered once daily by subcutaneous injection

Who Is Running the Clinical Trial?

Ascendis Pharma Bone Diseases A/S

Lead Sponsor

Trials

2

Recruited

80+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39376010/

52-Week Results From the Phase 3 PaTHway Trial Conclusion: At week 52 of the PaTHway trial, TransCon PTH showed sustained efficacy, safety, and tolerability in adults with hypoparathyroidism.

2. academic.oup.com academic.oup.com/jcem/article/110/4/951/7815470

Efficacy and Safety of TransCon PTH in Adults With ... TransCon PTH treatment was associated with sustained improvements in clinical outcomes as well as disease-related symptoms and the impact of hypoparathyroidism ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S1530891X25010031

Early U.S. Real-World Treatment Patterns and Outcomes in ... To examine the impact of palopegteriparatide (YORVIPATH; TransCon PTH) on real-world clinical outcomes in adult patients with hypoparathyroidism ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40846304/

Early U.S. Real-World Treatment Patterns and Outcomes in ... Objective: To examine the impact of palopegteriparatide (YORVIPATH; TransCon PTH) on real-world clinical outcomes in adult patients with ...

5. investors.ascendispharma.com investors.ascendispharma.com/news-releases/news-release-details/new-3-year-phase-3-data-confirmed-sustained-response-transconr

New 3-Year Phase 3 Data Confirmed Sustained Response ... Patients in the trial reported continued improvements from baseline in their hypoparathyroidism-related symptoms and health-related quality of ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04701203

NCT04701203 | A Trial Investigating the Safety, Tolerability ... A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Daily in Adults With Hypoparathyroidism (PaTHway). ClinicalTrials ...