What side effects are associated with this type of intervention?

Common treatments for hypoparathyroidism, particularly Parathyroid Hormone (PTH) replacement therapies like TransCon PTH, often include side effects such as hypercalcemia (high calcium levels), hypocalcemia (low calcium levels), and injection site reactions. Other potential side effects may include headaches, nausea, and muscle cramps. Long-term use of PTH therapy can also lead to concerns about bone health, such as osteosarcoma, although this is rare. It is important for patients to be closely monitored by their healthcare provider to manage these risks effectively.

What prior FDA approvals exist?

The FDA has approved parathyroid hormone (PTH) therapies for the treatment of hypoparathyroidism, a condition characterized by insufficient production of parathyroid hormone. One notable FDA-approved drug is Natpara (parathyroid hormone), which is used as a hormone replacement therapy to manage hypocalcemia in patients with hypoparathyroidism. Natpara is administered via subcutaneous injection and aims to regulate calcium levels in the blood. Similar to TransCon PTH, these treatments focus on replacing the deficient hormone to maintain calcium and phosphate balance in the body.

What other types of research exist?

As of now, there are no specific novel alternatives to TransCon PTH mentioned in the provided context for hypoparathyroidism treatment in 2024. Patients should consult with their healthcare providers to explore the latest clinical trials and emerging therapies in parathyroid hormone replacement.

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Hormone Therapy

TransCon PTH for Hypoparathyroidism (PaTH Forward Trial)

Phase 2

Waitlist Available

Research Sponsored by Ascendis Pharma A/S

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

eGFR >30 mL/min/1.73m2 during Screening

If ≤25 years of age, radiological evidence of epiphyseal closure based on x-ray of non-dominant wrist and hand

Must not have

Use of other drugs known to influence calcium and bone metabolism, such as calcitonin, fluoride tablets (> 0.5 mg/day), strontium, or cinacalcet hydrochloride within 12 weeks prior to Visit 1

Known activating mutation in the calcium-sensing receptor (CaSR) gene

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 4

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects of TransCon PTH may include reactions at the injection site such as redness or pain, headaches, nausea, muscle cramps or spasms due to changes in calcium levels. Long-term effects are being studied since this is part of what the trial aims to discover.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function is good.

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My growth plates in my wrist and hand have fully developed, confirmed by an x-ray.

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I can give myself daily injections or have someone who can do it for me.

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I take at least 400 mg of calcium citrate or carbonate twice a day.

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I am taking a specific dose of active vitamin D.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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My genetic test shows a mutation in the CaSR gene.

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My body does not respond well to PTH, and I have high PTH levels with low calcium.

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I have used bisphosphonates or denosumab in the last 2 years.

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I am not using specific medications like loop diuretics or methotrexate.

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I do not have any conditions that affect my calcium or hormone levels.

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I haven't had serious heart issues in the last 6 months.

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I am not pregnant or breastfeeding and willing to use contraception during the trial.

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I have not used PTH-like drugs in the last 5 weeks.

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I have not been in a drug study within the last 8 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 4

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 4

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 4 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Efficacy - Primary Endpoint During the Blinded Period

Secondary study objectives

Efficacy - Key Secondary Endpoint During the Blinded Period

Trial Design

5Treatment groups

Experimental Treatment

Placebo Group

Group I: TransCon PTH 21 mcgExperimental Treatment1 Intervention

TransCon PTH 21 mcg delivered once daily by subcutaneous injection

Group II: TransCon PTH 18 mcgExperimental Treatment1 Intervention

TransCon PTH 18 mcg delivered once daily by subcutaneous injection

Group III: TransCon PTH 15 mcgExperimental Treatment1 Intervention

TransCon PTH 15 mcg delivered once daily by subcutaneous injection

Group IV: Open-Label Extension PeriodExperimental Treatment1 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: PlaceboPlacebo Group1 Intervention

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

0 / 15Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Parathyroid Hormone Replacement Therapy, such as TransCon PTH, works by providing a synthetic form of parathyroid hormone (PTH) to patients with Hypoparathyroidism. This hormone is crucial for regulating calcium and phosphate levels in the blood. By mimicking the natural hormone, these treatments help maintain normal calcium levels, reduce symptoms like muscle cramps and tingling, and prevent long-term complications such as kidney stones and cardiovascular issues. This is particularly important for Hypoparathyroidism patients who lack sufficient endogenous PTH, as it directly addresses the underlying hormone deficiency and helps restore metabolic balance.