MBX 2109 for Hypoparathyroidism

No longer recruiting at 32 trial locations

Overseen By Salomon Azoulay, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: MBX Biosciences

Must be taking: Active vitamin D, Calcium

Must not be taking: Diuretics, Bisphosphonates

Disqualifiers: Pseudohypoparathyroidism, Cardiovascular disease, others

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

Compensation: Varies

Number of Visits: 15

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called MBX 2109 to assess its safety and tolerability for individuals with hypoparathyroidism, a condition characterized by insufficient production of parathyroid hormone, resulting in low calcium levels. Participants will receive varying doses of MBX 2109 or a placebo, administered as a weekly injection, to compare effects. Suitable candidates for this trial have had hypoparathyroidism for at least six months and currently take specific vitamin D and calcium supplements. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant advancements in treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that certain medications like loop diuretics, thiazide diuretics, and others should not be used within 4 weeks before the screening. If you're on chronic systemic corticosteroids, the dose should be stable for 4 weeks before the screening.

Will I have to stop taking my current medications?

The trial does not specify that you need to stop taking your current medications, but certain drugs like loop diuretics, thiazide diuretics, and others affecting calcium and bone metabolism should not be used within specific timeframes before the trial. If you're on chronic systemic corticosteroids or thyroid medication, the dose should be stable for at least 4 weeks before the trial.

Is there any evidence suggesting that MBX 2109 is likely to be safe for humans?

Research shows that MBX 2109 is generally safe for patients. In earlier studies, no unexpected safety issues arose, meaning the treatment didn't cause surprising side effects.

The treatment involves a weekly injection and has been tested at various doses. Safety results indicate that patients tolerated it well at all dose levels. This suggests that MBX 2109 could be a safe option for people with hypoparathyroidism, a condition where the body doesn't produce enough parathyroid hormone.

In summary, MBX 2109 demonstrated a good safety record in earlier research, with patients managing the treatment without major problems. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hypoparathyroidism?

Unlike the standard treatments for hypoparathyroidism, which typically involve oral calcium and active vitamin D supplements, MBX 2109 offers a novel approach by using a once-weekly subcutaneous injection. This method could provide more stable calcium levels by directly addressing parathyroid hormone deficiencies, potentially reducing the need for frequent dosing and minimizing fluctuations in calcium levels. Researchers are excited about MBX 2109 because it may offer a more convenient and effective way to manage hypoparathyroidism, improving patients' quality of life by potentially reducing the burden of daily medication management.

What evidence suggests that MBX 2109 might be an effective treatment for hypoparathyroidism?

Research has shown that MBX 2109 could be promising for treating hypoparathyroidism. In earlier studies, 63% of patients responded positively to MBX 2109 after 12 weeks, experiencing significant benefits. This trial will test different starting doses of MBX 2109, administered once weekly by subcutaneous injection, to evaluate its effectiveness and safety. The treatment replaces a hormone that helps maintain balanced calcium levels in the body. Previous studies reported that it was generally well-tolerated, with no major safety issues. Its long-lasting effects and steady presence in the body make it a strong option for people with this condition. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with hypoparathyroidism. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be required to have a confirmed diagnosis of the condition being studied.

Inclusion Criteria

I have been taking specific vitamin D and calcium doses for over 4 weeks.

At the Screening visit or at completion of the Optimization Period, specific serum analytes must be within defined ranges

Has an estimated glomerular filtration rate >60 mL/min/1.73 m2 at the Screening visit

Exclusion Criteria

I have a condition that affects my body's calcium or PTH levels.

Has a known allergy or sensitivity to PTH or any of the excipients used in MBX 2109

Has any other reason that would prevent the patient from completing participation or following the study schedule

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MBX 2109 or placebo once weekly by subcutaneous injection to evaluate safety, pharmacokinetics, and efficacy

12 weeks

15 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• MBX 2109

Trial Overview

The study tests MBX 2109's safety and how well it works compared to a placebo in people with hypoparathyroidism. It's given once weekly, and the trial likely involves regular monitoring of participants' responses to treatment.

How Is the Trial Designed?

Group I: Starting dose of 800 µg once-weekly by subcutaneous injection Experimental Treatment 1 Intervention

Group II: Starting dose of 600 µg once-weekly by subcutaneous injection Experimental Treatment 1 Intervention

Group III: Starting dose of 400 µg once-weekly by subcutaneous injection Experimental Treatment 1 Intervention

Group IV: Placebo - once-weekly by subcutaneous injection Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

MBX Biosciences

Lead Sponsor

Trials

5

Recruited

290+

Published Research Related to This Trial

Hypoparathyroidism is relatively common among hospitalized patients in Italy, with a prevalence of 5.3 cases per 100,000 inhabitants per year, and the majority of cases (72.2%) occur in women.

There has been a significant decrease in hospitalization rates for hypoparathyroidism, particularly for postsurgical cases, over the 8-year study period from 2006 to 2013.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Epidemiology of Hypoparathyroidism in Italy: An 8-Year Register-Based Study. Cipriani, C., Pepe, J., Biamonte, F., et al.[2018]

Hypoparathyroidism, often caused by thyroid surgery, leads to low calcium and high phosphorus levels, and current treatments focus on calcium and vitamin D supplementation to maintain serum calcium in the low-normal range.

Recombinant human PTH 1-84 is now available as a hormone replacement therapy for patients who do not respond well to standard treatments, marking a significant advancement in managing this rare endocrine disorder.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review of Hypoparathyroidism. Abate, EG., Clarke, BL.[2019]

After 8 years of treatment with recombinant human parathyroid hormone (1-84), patients with hypoparathyroidism experienced significant reductions in their need for supplemental calcium (57%) and active vitamin D (76%), indicating improved management of their condition.

The therapy was associated with stable renal function and increased bone mineral density (BMD) at the lumbar spine and total hip, demonstrating both safety and efficacy over the long term, with minimal occurrences of hypercalcemia or hypocalcemia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapy of Hypoparathyroidism With rhPTH(1-84): A Prospective, 8-Year Investigation of Efficacy and Safety. Tay, YD., Tabacco, G., Cusano, NE., et al.[2020]

Patients with hypoparathyroidism, despite receiving standard treatment with calcium and vitamin D, report a significantly reduced quality of life compared to norm-based populations, indicating that the absence of parathyroid hormone may directly affect their well-being.

A systematic review of five studies found that hypoparathyroidism patients experience various physical, mental, and emotional symptoms, highlighting the need for further research to better understand and quantify these impacts on quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Quality of life in patients with hypoparathyroidism receiving standard treatment: a systematic review. Büttner, M., Musholt, TJ., Singer, S.[2018]

A consensus was reached among Delphi panels in the UK, Sweden, and Portugal on key characteristics that define chronic hypoparathyroidism as inadequately controlled, focusing on patient characteristics, comorbidities, biochemistry, and symptoms affecting quality of life.

Important factors identified include compliance issues, chronic kidney disease stages 4 and 5, and specific biochemical parameters like serum calcium and urinary calcium, which should be further evaluated for their relevance in clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Defining the Characteristics of Chronic Hypoparathyroidism Not Adequately Controlled on Conventional Therapy: Consensus Findings of Three European Delphi Panels. Iqbal, K., Dass, N., Gip, C., et al.[2020]

Citations

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MBX 2109, A Once-Weekly Parathyroid Hormone ...

Efficacy and safety of parathyroid hormone replacement with TransCon PTH in hypoparathyroidism: 26-week results from the phase 3 PaTHway trial. J Bone Miner ...

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This study is to investigate the safety, pharmacokinetics, and efficacy of MBX 2109 administered once weekly to patients with hypoparathyroidism.

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MBX Biosciences Announces Once-Weekly Canvuparatide ...

MBX Biosciences Announces Once-Weekly Canvuparatide Achieved Primary Endpoint in Phase 2 Trial with 63% Responder Rate at 12 Weeks; 79% ...

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The sustained-action PTH prodrug MBX 2109 was well tolerated with no unexpected, off-target safety issues. The long half-life and flat exposure profile of MBX ...

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clinicaltrialsarena.com/news/mbx-biosciences-hypoparathyroidism-trial/

MBX Biosciences dosing subjects in hypoparathyroidism ...

MBX Biosciences has dosed the first subject in a Phase II trial of MBX 2109, a potential treatment for chronic hypoparathyroidism (HP).

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MBX 2109, A Once-Weekly Parathyroid Hormone ...

Conclusion: The sustained-action PTH prodrug MBX 2109 was well tolerated with no unexpected, off-target safety issues. The long half-life and ...

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NCT06531941 | Extension Study to Evaluate the Long- ...

This study is to investigate the long-term safety and tolerability of MBX 2109 administered once weekly to patients with hypoparathyroidism.

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Exploring the Potential of Weekly Parathyroid Hormone ...

Safety results indicated that MBX 2109 was well tolerated across all dose ... MBX 2109 shows potential as a long-acting PTH replacement therapy for ...