TransCon PTH for Hypoparathyroidism

(PaTH Forward Trial)

Recruiting at 14 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ascendis Pharma A/S

Must be taking: Active vitamin D, Calcium

Must not be taking: Loop diuretics, Corticosteroids, Bisphosphonates, others

Disqualifiers: Diabetes, Cardiac disease, Malignancy, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

59 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests TransCon PTH, an injectable medication, on patients who might benefit from PTH treatment. It helps regulate calcium levels in the body, which is important for bone health and other functions. TransCon PTH is designed to overcome limitations of existing treatments.

Research Team

Aimee Shu, MD

Principal Investigator

Ascendis Pharma A/S Medical Monitor/Medical Expert

Eligibility Criteria

Adults over 18 with chronic hypoparathyroidism (HP) for at least half a year, taking specific vitamin D and calcium supplements, can join this trial. They must be able to self-inject medication and not have certain genetic mutations or recent use of related drugs. Pregnant women, those with severe heart or kidney issues, drug/alcohol dependence, or other conditions affecting calcium metabolism are excluded.

Inclusion Criteria

My kidney function is good.

Your body mass index (BMI) is between 17 and 40, which is a measure of your weight in relation to your height.

25(OH) vitamin D levels of 30-70 ng/mL (75-175 pmol/mL) and

Exclusion Criteria

I haven't taken drugs affecting bone metabolism like calcitonin or fluoride in the last 12 weeks.

My genetic test shows a mutation in the CaSR gene.

I don't have any health issues that could stop me from finishing the trial or that would make the trial unsafe for me.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants are randomly assigned to receive either TransCon PTH or placebo for four weeks, administered as a subcutaneous injection using a pre-filled injection pen

4 weeks

Open-label extension

Participants receive open-label TransCon PTH with dose adjustments based on individual needs for up to 262 weeks

262 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Placebo for TransCon PTH
• TransCon PTH

Trial Overview The trial is testing TransCon PTH against a placebo in adults with HP. Initially, participants are randomly assigned to receive either the drug or placebo via injection without knowing which one they're getting. After four weeks, all move on to receive TransCon PTH tailored to their needs in an extension study.

Participant Groups

Group I: TransCon PTH 21 mcg Experimental Treatment 1 Intervention

TransCon PTH 21 mcg delivered once daily by subcutaneous injection

Group II: TransCon PTH 18 mcg Experimental Treatment 1 Intervention

TransCon PTH 18 mcg delivered once daily by subcutaneous injection

Group III: TransCon PTH 15 mcg Experimental Treatment 1 Intervention

TransCon PTH 15 mcg delivered once daily by subcutaneous injection

Group IV: Open-Label Extension Period Experimental Treatment 1 Intervention

Subjects who complete the four-week blinded period are assigned to open-label treatment with TransCon PTH for up to 262 weeks, with up to an initial 14 weeks of TransCon PTH titration and standard of care optimization, followed by approximately 248 weeks of individualized dosing.

Group V: Placebo Placebo Group 1 Intervention

Placebo mimicking 15, 18, or 21 mcg of TransCon PTH delivered once daily by subcutaneous injection

Who Is Running the Clinical Trial?

Ascendis Pharma A/S

Lead Sponsor

Trials

15

Recruited

1,600+