Clinical Trials for Hypoparathyroidism

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by Mary Chapman |

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There currently is no cure for hypoparathyroidism, and moreover, few therapy options are available — particularly for patients for whom the standard treatment of calcium and active vitamin D is not very effective in controlling the disease. Participating in clinical trials can help researchers develop new and better treatments.

What are clinical trials?

Trials are part of clinical research and are at the heart of all medical advances. The goal of clinical trials is to determine whether a device, procedure, intervention, or pharmaceutical therapy is effective and safe. These studies also may test new ways of using existing treatments or assess other aspects of care. Some simply record daily life with a disease over time; such observational studies can provide important data for researchers and clinicians.

Depending on the goal and phase of the trial, both people with the disease and healthy volunteers may be able to enroll. The trials are controlled to ensure they are carried out as intended. The investigators monitor all participants to identify any issue or potential risk as soon as possible. All trials are regulated by law and require government approval before they can begin.

Is it an experiment?

The short answer is yes. For trials testing a particular intervention, the scientists designing the trial aim to gather enough scientific evidence — that which is clinically meaningful and reproducible — to support an application to a regulatory agency for approval or authorization of that which they are testing. In the U.S., investigators submit all such applications to the Food and Drug Administration (FDA) for approval.

It is important to remember that a new treatment being tested may have serious side effects that cannot be foreseen.

What are the different types of clinical trials?

There are several different types of clinical trials, with varying aims. Prevention trials look for better ways to keep a disease from occurring in individuals who have never had it, or to prevent the disorder from returning. Screening trials test new ways to detect health conditions, and diagnostic studies compare tests or procedures for diagnosing a specific disease. Meanwhile, treatment trials test new therapies or treatment approaches, and behavioral studies assess ways to promote beneficial behavioral changes. Supportive care investigations, also called quality of life trials, explore and measure ways to improve patients’ comfort and their quality of life.

In trials that compare a new product or therapy with an existing one, scientists may assign some participants to receive a placebo — an inactive product that resembles the test product. In randomized trials, treatments are assigned to participants by chance rather than by choice. This is done to avoid any bias in assigning volunteers to one treatment or another. Blinded or masked studies are designed to prevent research team members or participants from influencing study results. By contrast, in an open-label trial, researchers and patients both know which treatment is being administered.

What are the different phases of clinical trials?

Experimental medications typically undergo three trial “phases.” In the first phase, a relativity small number of participants are drawn from the general population to test the proposed treatment for safety, tolerability, and other properties.

Phase 2 involves testing the medication in the intended patient population. Tests here are for safety and early evidence of effectiveness, and generally include a small number of patients.

A Phase 3 trial aims to show scientifically that the medicine is of benefit for its target population. This phase usually requires a statistically valid and representative patient group to minimize scientific bias. Thus, these studies usually include a great number of participants.

Phase 4 studies also may be conducted after a therapy has been approved to assess its long-term safety and effectiveness.

Who can enroll in clinical trials?

Clinical studies have standards, called eligibility criteria, outlining who can participate. These are based on characteristics such as age, gender, disorder type and stage, the patient’s previous treatment history, and the presence of other medical conditions, among other criteria.

What are the potential benefits?

By enrolling yourself or your child in a clinical trial, you can:

  • gain access to new treatments before they are widely available.
  • receive regular and careful medical attention from a research team that includes physicians and other health professionals.
  • help others by contributing to knowledge about new treatments or procedures.

Questions to ask

If you are considering registering in a trial, you should feel free to ask any questions or broach any issues concerning the trial at any time. Make a list of your questions or concerns to be sure all of them are addressed.

Some general questions you might want to ask include:

  • What is the purpose of the study?
  • Who will fund the study?
  • How long will the study last?
  • Who will inform me of the study results?
  • What are the possible benefits and risks?
  • What kinds of therapies, procedures, or tests will I (or my child) undergo?
  • Who will be in charge of my (child’s) care?

Hypoparathyroidism clinical trials

A number of hypoparathyroidism clinical trials are taking place at universities and medical centers globally. Each study summary provides a list of where it’s taking place and whether it is recruiting participants. To participate in a study, it’s often necessary to contact the study coordinator of the participating institution.

You can find a list of dozens of hypoparathyroidism studies on the U.S. National Library of Medicine website.

One currently recruiting observational trial in France (NCT04126941) is studying the efficacy and safety of Forteo (teriparatide), a medicine that helps build new bone, in children with hypoparathyroidism. A separate, unrelated study recently found that lower levels of PTH in patients may lead to decreased bone material strength and increased fracture risk.

Meanwhile, a U.S. multicenter, randomized, double-blind, placebo-controlled clinical trial (NCT03324880) is evaluating whether adding an investigational therapy called recombinant human parathyroid hormone (rhPTH) to calcium and vitamin D tablets is more effective than the standard vitamin supplement therapy.

A clinical study (NCT03878953) in Japan aims to gauge the safety and efficacy of repeated dosing of rhPTH in participants with chronic hypoparathyroidism for a 26-week period. And, an observational disease and treatment registry (NCT01922440) is recruiting participants with chronic hypoparathyroidism to assess the safety and effectiveness of rhPTH in routine clinical practice, and to observe the clinical disease course.

The Hypoparathyroidism Association also provides information about current research and clinical trials for the disease.


Last updated: Sept. 21, 2020


Hypoparathyroidism News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.