MBX 2109 for Hypoparathyroidism

Recruiting at 25 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

64 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this study is to investigate the safety and tolerability of MBX 2109 administered once weekly to patients with hypoparathyroidism.

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.

How is the drug MBX 2109 different from other treatments for hypoparathyroidism?

MBX 2109 is unique because it may offer a new approach to treating hypoparathyroidism, which traditionally relies on calcium and vitamin D supplements that can lead to complications. Unlike these supplements, MBX 2109 could potentially address the underlying hormone deficiency, similar to recombinant human PTH 1-84, which is a hormone replacement therapy for patients not well controlled on standard treatments. ^{1 2 3 4 5}

What makes the drug MBX 2109 unique for treating hypoparathyroidism?

MBX 2109 is unique because it potentially offers a new approach to treating hypoparathyroidism, which traditionally relies on calcium and vitamin D supplements that can lead to complications. Unlike these supplements, MBX 2109 may provide a more direct method of managing the condition by addressing the underlying hormone deficiency. ^{1 2 3 4 5}

Eligibility Criteria

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

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

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Therapy of Hypoparathyroidism With rhPTH(1-84): A Prospective, 8-Year Investigation of Efficacy and Safety. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

The Epidemiology of Hypoparathyroidism in Italy: An 8-Year Register-Based Study. [2018]

3. United States pubmed.ncbi.nlm.nih.gov

Quality of life in patients with hypoparathyroidism receiving standard treatment: a systematic review. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Defining the Characteristics of Chronic Hypoparathyroidism Not Adequately Controlled on Conventional Therapy: Consensus Findings of Three European Delphi Panels. [2020]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Review of Hypoparathyroidism. [2019]