Parathyroid Transplant for Hypoparathyroidism

(PATH Trial)

Overseen By Brenessa Lindeman, MD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: University of Alabama at Birmingham

Must not be taking: Immunosuppressants

Disqualifiers: Active malignancy, Uncontrolled diabetes, Severe osteoporosis, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment option for people with hypoparathyroidism, a condition where the body produces insufficient parathyroid hormone, leading to low calcium levels. The trial tests parathyroid allotransplantation, a procedure that transplants healthy parathyroid tissue to better manage calcium levels. It targets those who have tried other treatments without success and continue to struggle with low calcium affecting their daily life. Ideal participants have had hypoparathyroidism for at least a year, often due to neck surgery, and still experience issues despite treatment. As an unphased trial, this study provides a unique opportunity to explore a novel treatment approach for managing hypoparathyroidism.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, since the trial involves a transplant, you may need to take immunosuppressive drugs, which could affect your current medication regimen.

What prior data suggests that parathyroid allotransplantation is safe for patients with hypoparathyroidism?

Research has shown that parathyroid allotransplantation might be a safe treatment for people with hypoparathyroidism. One review suggested this method could be safe and effective for those with permanent hypoparathyroidism. Another source reported a success rate of 46% for patients who received this treatment, indicating that nearly half of the patients benefited.

While these studies provide some insight, parathyroid allotransplantation remains a new treatment option. Only a few centers worldwide have successfully offered this therapy. Therefore, anyone considering joining a trial should discuss the possible risks and benefits with their healthcare provider. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for hypoparathyroidism, such as calcium and vitamin D supplements, parathyroid allotransplantation offers a potentially transformative approach by transplanting healthy parathyroid tissue from a donor. This method could restore the body's natural ability to regulate calcium levels, potentially reducing or eliminating the need for lifelong medication. Researchers are excited about this treatment because it targets the root cause of the condition, offering a more permanent solution compared to the current management strategies.

What evidence suggests that parathyroid allotransplantation might be an effective treatment for hypoparathyroidism?

Research has shown that parathyroid allotransplantation has about a 46% success rate in improving parathyroid function. Nearly half of the patients who receive this treatment experience enhanced parathyroid activity, crucial for managing calcium levels in the body. As a relatively new treatment, only a few centers worldwide have successfully performed it. Although promising, the current success rate suggests that further research is needed to enhance its effectiveness. Potential participants should consider these findings when deciding to join a trial for this treatment. ^{1 6 7 8 9}

Who Is on the Research Team?

Brenessa Lindeman, MD, MEHP

Principal Investigator

University of Alabama at Birmingham

Are You a Good Fit for This Trial?

This trial is for adults aged 18-80 with hypoparathyroidism caused by neck surgery, congenital issues, or treatment failure. They must have had the condition for at least a year and live near Birmingham. Participants need to speak English and commit to the trial's process. Exclusions include active cancer (except skin), severe heart/liver/kidney/brain diseases, uncontrolled diabetes, clotting disorders, psychiatric illness, osteoporosis, allergies to immunosuppressants, non-adherence to medical therapies.

Inclusion Criteria

I have low calcium due to a neck surgery that affected my parathyroid glands.

I have low calcium due to underdeveloped parathyroid glands.

I have low calcium due to hypoparathyroidism for over a year.

Exclusion Criteria

You are allergic or highly sensitive to certain medications that can weaken the immune system.

I rely on a nursing home or long-term care for my daily needs.

I quit smoking more than 3 months ago.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Virtual consults and in-person visits at MD discretion

Pre-graft function

Daily phone check-ins with labs every other day and clinic visits twice weekly with ultrasound of graft

2 weeks

Post-graft function

Weekly labs and clinic visits for 3 months, followed by monthly visits for 1 year

15 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Parathyroid Allotransplantation

Trial Overview The UAB PATH trial is testing parathyroid allotransplantation as a new treatment option for patients with medically refractory hypocalcemia due to hypoparathyroidism. This innovative procedure aims to provide relief where other treatments have failed.

Parathyroid Allotransplantation is already approved in United States, European Union for the following indications:

Approved in United States as Parathyroid Allotransplantation for:

• Severe, refractory hypocalcemia due to hypoparathyroidism

Approved in European Union as Parathyroid Allotransplantation for:

• Permanent hypoparathyroidism

Who Is Running the Clinical Trial?

University of Alabama at Birmingham

Lead Sponsor

Trials

1,677

Recruited

2,458,000+

Published Research Related to This Trial

Parathyroid allotransplantation shows a mean graft survival rate of 47% at 6 months and 41% at 12 months in patients with post-thyroidectomy hypoparathyroidism, based on a review of 9 studies involving 146 patients.

While some evidence suggests that parathyroid hormone (PTH) and serum calcium levels remain elevated post-transplantation, the long-term benefits and risks of this treatment remain unclear due to significant variability among the studies, highlighting the need for further controlled clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parathyroid allotransplantation to treat post-thyroidectomy hypoparathyroidism: A review of case studies. Parameswaran, R., Samuel, M., Satish, RL., et al.[2021]

A new parathyroid allotransplant technique was developed, showing a 70% success rate in restoring parathyroid function in 10 patients with permanent hypoparathyroidism after a mean follow-up of 12 months.

The procedure is cost-effective, requires minimal staff and equipment, and involves only short-term use of immunosuppressants, with no major complications reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parathyroid Allotransplant With a New Technique: A Prospective Clinical Trial. Aysan, E., Altug, B., Ercan, C., et al.[2017]

Parathyroid glands harvested using a two-stage procurement method and stored for up to 48 hours in cold University of Wisconsin media maintained their viability and functional integrity, showing no signs of hypoxic stress or apoptosis.

The glands demonstrated preserved calcium-sensing capacity and successfully engrafted in vivo after transplantation into mice, indicating that this method is effective for ensuring the success of parathyroid allotransplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of Multi-stage Procurement on the Viability and Function of Human Donor Parathyroid Glands. Kelly, YM., Ward, C., Zhang, R., et al.[2022]

A 47-year-old woman with permanent hypoparathyroidism successfully underwent parathyroid allotransplantation, resulting in the cessation of her long-term intravenous calcium therapy after 3.5 years of treatment.

The transplantation was safe, with no complications reported, and led to stable serum calcium levels without the need for IV calcium, highlighting its efficacy as a cost-effective treatment option for patients with persistent hypoparathyroidism.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Discharging a Patient Treated With Parathyroid Allotransplantation After Having Been Hospitalized for 3.5 Years With Permanent Hypoparathyroidism: A Case Report. Aysan, E., Yucesan, E., Idiz, UO., et al.[2020]

Transplanting human adenomatous parathyroid cells into rabbits resulted in a significant increase in parathyroid hormone levels, indicating that these cells can function effectively after transplantation.

No signs of malignant transformation were observed in the transplanted parathyroid cells, suggesting that this method could be a safe alternative for treating permanent hypoparathyroidism in patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Xenotransplantation of human cryopreserved parathyroid tissue isolated from parathyroid adenomas to normocalcemic rabbits. Ayşan, E., Düzköylü, Y., Can, İ., et al.[2020]

In a study of 85 patients who underwent allotransplantation of cultured parathyroid cells for surgical hypoparathyroidism, 55.1% retained endocrine function for more than 2 months, suggesting that this method can be a viable treatment option for permanent hypoparathyroidism.

The study demonstrated that the survival of the parathyroid cell allografts averaged 6.35 months, and interestingly, the success of the grafts did not depend on the initial viability or secretory activity of the cultured cells used for transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Allotransplantation of cultured parathyroid progenitor cells without immunosuppression: clinical results. Nawrot, I., Woźniewicz, B., Tołłoczko, T., et al.[2021]

In a study of 57 patients who underwent total parathyroidectomy plus autotransplantation, those with hypoparathyroidism required significantly more calcium carbonate and vitamin D3 supplements than those without hypoparathyroidism, indicating a higher need for these nutrients post-surgery.

Despite the increased supplement requirements, hypoparathyroid patients did not experience any adverse side effects, and overall symptoms improved significantly within three months, with bone mineral density also increasing after one year.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hypoparathyroidism after total parathyroidectomy plus subcutaneous autotransplantation for secondary hyperparathyroidism--any side effects? Chou, FF., Chi, SY., Hsieh, KC.[2021]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8953572/

Parathyroid Allotransplantation: A Systematic Review - PMC With only the studies eligible for our assessment, the pooled success rate for parathyroid allotransplantation emerged to be 46% (13/28 ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0748798325004354

Effectiveness of parathyroid autotransplantation during ... PAT is effectively restores and maintains parathyroid function following total thyroidectomy. Functional recovery begins 1 week post-PAT and is complete in 1 ...

3. gs.amegroups.org gs.amegroups.org/article/view/17084/html

Short and long-term impact of parathyroid autotransplantation ... Parathyroid autotransplantation has been demonstrated to be effective, clinically and biochemically, with a functional graft survival rate of 93% (9). However, ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35323218/

Parathyroid Allotransplantation: A Systematic Review With only the studies eligible for our assessment, the pooled success rate for parathyroid allotransplantation emerged to be 46% (13/28 ...

5. facs.org facs.org/for-medical-professionals/news-publications/news-and-articles/acs-brief/february-4-2025-issue/parathyroid-transplantation-does-not-prevent-hypoparathyroidism-after-thyroid-surgery/

Parathyroid Transplantation Does Not Prevent ... The authors concluded that parathyroid transplantation did not alter outcomes in patients with low postoperative parathyroid hormone levels.

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0002961021004050

Parathyroid allotransplantation for the treatment of ... Parathyroid allotransplantation presents a potentially safe and effective therapeutic option for the definitive treatment of permanent hypoparathyroidism.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5756758/

Short and long-term impact of parathyroid autotransplantation ... Parathyroid autotransplantation has been demonstrated to be effective, clinically and biochemically, with a functional graft survival rate of 93% (9). However, ...

8. jes-online.org jes-online.org/pdf/10.16956/jes.2025.25.1.1

Advancements and Clinical Strategies in Parathyroid ... Parathyroid transplantation holds significant promise as a treatment for hypoparathyroidism. Further research and clinical trials are warranted ...

9. gs.amegroups.org gs.amegroups.org/article/view/15246/html

Parathyroid transplantation in thyroid surgery - Barczyński This paper is aimed to provide a review of current status of various parathyroid transplantation techniques in thyroid surgery.