MBX 2109 for Hypoparathyroidism

Recruiting at 20 trial locations

Overseen By Kristi Schneider, RN, ANP-BC

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: MBX Biosciences

Disqualifiers: Others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

48 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the long-term safety and tolerability of MBX 2109 in patients with hypoparathyroidism who completed the 12-week treatment period in the Phase 2 study, MBX-2H1002.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

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 used for patients not well controlled on standard therapy. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults over 18 with hypoparathyroidism (post-surgical, idiopathic, or autoimmune) diagnosed for at least half a year. Participants must have finished a previous 12-week Phase 2 study of MBX-2109 and be able to self-administer the drug after proper demonstration.

Inclusion Criteria

I have been diagnosed with a specific type of low parathyroid hormone for over 6 months.

Must have completed the Week 12 Visit in MBX-2H1002 Study

I am 18 years old or older.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MBX 2109 once weekly to evaluate long-term safety and tolerability

104 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• MBX 2109

Trial Overview The trial tests the long-term safety and tolerability of MBX-2109 in patients with hypoparathyroidism. It involves weekly subcutaneous injections ranging from 200 to 1600 µg, following up on an earlier phase study.

Participant Groups

Group I: 400 µg once-weekly by subcutaneous injection Experimental Treatment 1 Intervention

Group II: 200-1600 µg once-weekly by subcutaneous injection Experimental Treatment 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]

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]

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]

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. Switzerland pubmed.ncbi.nlm.nih.gov

Review of Hypoparathyroidism. [2019]

5. 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]