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TransCon PTH for Hypoparathyroidism (PaTHway Trial)

Phase 3
Waitlist Available
Research Sponsored by Ascendis Pharma Bone Diseases A/S
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
If ≤25 years of age, radiological evidence of epiphyseal closure based on X-ray of nondominant wrist and hand
eGFR ≥30 mL/min/1.73 m2 during Screening
Must not have
High risk thyroid cancer within 2 years, requiring suppression of TSH <0.2 mIU/mL
Disease processes that adversely affect gastrointestinal absorption, including but not limited to short bowel syndrome, significant small bowel resection, gastric bypass, tropical sprue, active celiac disease, active ulcerative colitis, active Crohn's disease, gastroparesis and AIRE gene mutations with malabsorption
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 26 weeks
Awards & highlights
Pivotal Trial

Summary

This trial is testing a new drug called TransCon PTH to see if it is better than placebo at treating a disease called hypoparathyroidism. The trial will last for 26 weeks, and then participants will be able to continue taking TransCon PTH as part of a long-term extension study.

Who is the study for?
Adults over 18 with hypoparathyroidism who need certain doses of supplements and have stable thyroid-related treatments can join. They must be able to self-inject medication, not have conditions affecting calcium metabolism other than hypoparathyroidism, and agree to contraception if necessary. Excluded are those with PTH-resistance, recent high-risk cancer, seizures, or severe heart issues.
What is being tested?
The trial tests TransCon PTH against a placebo in adults with hypoparathyroidism. For the first half-year, participants won't know which they're getting as they adjust their dose for optimal effect. Afterward, all get TransCon PTH in a long-term study where doses are tailored individually.
What are the potential side effects?
While specific side effects aren't listed here, common reactions may include irritation at the injection site, potential allergic reactions to ingredients in the medication mix (like metacresol), and changes related to calcium levels due to treatment adjustments.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My growth plates in my wrist and hand have fully developed, as confirmed by an X-ray.
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My kidney function is adequate.
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I've been taking specific doses of calcitriol, alfacalcidol, or calcium for over 12 weeks.
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I can give myself daily injections or have someone who can.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My thyroid cancer is high risk and I need treatment to lower my TSH below 0.2.
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I do not have conditions that affect my body's ability to absorb nutrients.
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I have not had a stroke in the last 5 years.
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I am not taking specific medications like loop diuretics, phosphate binders (except calcium), digoxin, lithium, methotrexate, high-dose biotin, or systemic corticosteroids (unless it's for replacement therapy).
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I haven't used any osteoporosis treatments affecting calcium or bone in the last 2 years.
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My female partner might become pregnant and we are not planning to use birth control during the trial.
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I have a condition where my body doesn't respond to PTH hormone, leading to low calcium.
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I am allergic to PTH or ingredients in its medication.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~26 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 26 weeks 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
Secondary study objectives
Change from baseline in HPES Impact - Daily Life Domain score
Change from baseline in HPES Impact - Physical Functioning Domain score
Change from baseline in HPES Symptom - Cognitive Domain score
+2 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: TransCon PTHExperimental Treatment1 Intervention
TransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection
Group II: PlaceboPlacebo Group1 Intervention
Placebo for TransCon PTH delivered once daily by subcutaneous injection

Find a Location

Who is running the clinical trial?

Ascendis Pharma Bone Diseases A/SLead Sponsor
1 Previous Clinical Trials
1 Trials studying Hypoparathyroidism
Aimee D Shu, MDStudy DirectorAscendis Pharma A/S North American Medical Monitor/Medical Expert
3 Previous Clinical Trials
606 Total Patients Enrolled
Michael Beckert, MDStudy DirectorAscendis Pharma A/S European Medical Monitor/Medical Expert
7 Previous Clinical Trials
657 Total Patients Enrolled
1 Trials studying Hypoparathyroidism
59 Patients Enrolled for Hypoparathyroidism

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT04701203 — Phase 3
Hypoparathyroidism Research Study Groups: TransCon PTH, Placebo
Hypoparathyroidism Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT04701203 — Phase 3
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT04701203 — Phase 3
~17 spots leftby Dec 2025
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