TransCon PTH for Hypoparathyroidism

(PaTHway Trial)

Recruiting at 24 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

84 Participants Needed

Trial Summary

What is the purpose of this trial?

During the first 26 weeks of the trial, participants will be randomly assigned to one of two groups: one group will receive TransCon PTH and one group will receive placebo. All subjects will start with study drug at a dose of 18 mcg/day and will be individually and progressively titrated to an optimal dose in dose increments of 3 mcg/day. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs. This is a global trial that will be conducted in the United States, Canada, Germany, Denmark, Norway, Italy, and Hungary.

Research Team

Aimee D Shu, MD

Principal Investigator

Ascendis Pharma A/S Medical Monitor/Medical Expert

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+