Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

PaTH Forward: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel Group Trial with an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults with Hypoparathyroidism

The 'PaTH Forward' study is testing a new daily injection medicine, TransCon PTH, for adults living with hypoparathyroidism. This condition means the body doesn't produce enough parathyroid hormone, affecting calcium levels. The study wants to find out if TransCon PTH can help patients keep their calcium levels within a healthy range, making them feel better. Researchers are checking how safe the medicine is, if people can take it without too many problems, and if it helps reduce the need for strong calcium and vitamin D supplements that many people with hypoparathyroidism currently rely on. It's a phase 2 study, meaning it's an early-stage trial trying to get more information about the drug's effects.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Ascendis Pharma Bone Diseases A/S

Enrolment target

Start

30 Jul 2024

Hypoparathyroidism (HP) in Adults

What is this study about?

This study, called 'PaTH Forward', is investigating a new treatment named TransCon PTH for adults who have hypoparathyroidism. Hypoparathyroidism is a condition where your body doesn't make enough parathyroid hormone (PTH). This hormone is really important for keeping the right amount of calcium in your blood. When you don't have enough PTH, your calcium levels can drop too low, which can cause many different symptoms and affect your day-to-day life.

The main goal of this study is to see if taking TransCon PTH daily as an injection can help people with hypoparathyroidism keep their blood calcium levels stable and normal. For many people with this condition, managing calcium levels often involves taking large doses of vitamin D and calcium supplements. The study hopes that TransCon PTH could potentially reduce the need for these supplements, simplifying treatment and potentially reducing side effects associated with high doses of vitamin D and calcium.

The researchers will closely monitor participants' calcium levels and how much vitamin D and calcium supplements they need. They will be looking to see if, after four weeks of treatment, participants' calcium levels are normal, if the amount of calcium passed in their urine is normal or significantly reduced, and if they can stop taking active vitamin D supplements or reduce their calcium supplements to a lower, safer level (specifically, 1000 mg or less per day).

Key takeaways

This study evaluates a new treatment, TransCon PTH, for adults with hypoparathyroidism.
It aims to normalise calcium levels and potentially reduce the need for other medications.
Participation involves daily injections, regular clinic visits, and blood/urine tests.
You might receive the active medicine or a placebo during the first part of the study.
The study assesses safety, how well people tolerate the medicine, and its effectiveness.
You can withdraw from the study at any time without affecting your normal care.

Who may be eligible?

To be considered for this study, you must be an adult with hypoparathyroidism. This means you are 18 years old or older, and there's no upper age limit, so people of all adult ages can be considered. The study is open to both men and women.

Beyond these basic requirements, the research team will have more specific medical criteria to ensure the study is safe and appropriate for you. For example, they'll need to check your current health, medical history, and how you are currently managing your hypoparathyroidism. They will also need to review any other medications you are taking.

It's important to remember that meeting the basic age and condition requirements doesn't automatically mean you'll be able to join. The medical team will conduct thorough checks to make sure the study is a good fit for you and that you meet all the detailed health requirements.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult (18 years or older)?
Have you been diagnosed with hypoparathyroidism?
Are you able to attend regular clinic appointments?
Are you comfortable with daily injections?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first go through a screening process to make sure it's safe and suitable for you. If you qualify, you will receive the study medicine, TransCon PTH, as a daily injection under your skin, or a placebo (a dummy medicine with no active ingredients). This part of the study is 'double-blind', meaning neither you nor your study doctor will know whether you are receiving the active medicine or the placebo, to keep the results fair and unbiased. Your treatment will last for at least four weeks.

Throughout the study, you would have regular visits to the clinic for medical check-ups, blood tests (to measure calcium and other levels), and urine tests. These appointments are important for monitoring your health, checking how well the treatment is working, and adjusting your other medications if needed, like your current vitamin D and calcium supplements. After the initial four-week period, there's often an 'open-label extension' phase where all participants receive the active TransCon PTH medicine, and follow-up will continue for a longer period to understand the long-term effects. The total duration of your involvement, including all visits and follow-up, would be discussed in detail by the study team.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as gaining access to a new treatment for hypoparathyroidism that is not yet widely available. This treatment might help you better manage your calcium levels and potentially reduce your need for other supplements. However, like all medicines, TransCon PTH may have side effects, which could range from mild to more serious; the research team will explain all known possibilities. You might also receive the placebo and not experience any direct benefits from the study medicine at first. There will also be some inconvenience from clinic visits and tests. It's very important to remember that participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
Denmark
—
Unverified
Germany
—
Unverified
Norway
—
Unverified
Italy

Common questions

What is hypoparathyroidism?

Hypoparathyroidism is a condition where your body doesn't produce enough parathyroid hormone (PTH), which leads to low calcium levels in your blood.

What is TransCon PTH?

TransCon PTH is a new medicine being tested in this study, designed to replace the missing parathyroid hormone in people with hypoparathyroidism.

What does 'Phase 2' mean?

'Phase 2' means this is an early stage of research where doctors are mainly looking at how safe the new medicine is, how well people tolerate it, and if it shows promise in treating the condition.

Will I definitely get the new medicine?

Not necessarily at first. In the main part of the study, some participants will receive the active medicine, while others will receive a dummy medicine (placebo). Neither you nor the doctors will know which you are getting.

Can I stop participating if I want to?

Yes, absolutely. Your participation is completely voluntary, and you can choose to leave the study at any time, for any reason, without affecting your medical care.