HypoparathyroidismPhase 3PaTHwayNCT04701203
A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Daily in Adults With Hypoparathyroidism
A Phase 3 study testing TransCon PTH in people with Hypoparathyroidism and Endocrine System Diseases, run by Ascendis Pharma Bone Diseases A/S at 21 sites in 7 countries.
Trial summary
During the first 26 weeks of the trial, participants were randomly assigned to one of two groups: one group received TransCon PTH and one group received placebo. All participants started with study drug at a dose of 18 mcg/day and were individually and progressively titrated to an optimal dose in dose increments of 3… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypoparathyroidism, Endocrine System Diseases, Parathyroid Diseases
- What is being tested?
- TransCon PTH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 84 participants
- Where
- 21 sitesin 7 countries
- Study dates
- 16 Feb 2021to 21 Jan 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females, ≥18 years of age
- Subjects with postsurgical chronic HP, or auto-immune, genetic, or idiopathic HP for at least 26 weeks. Diagnosis of HP is established based on historic hypocalcemia in the setting of inappropriately low serum PTH levels
- Requirement for doses of SoC (e.g., calcitriol, alfacalcidol, calcium supplements) at or above a minimum threshold:
- For countries other than Japan: requirement for a dose of calcitriol ≥0.5 μg/day, or alfacalcidol ≥1.0 μg/day and (elemental) calcium ≥800 mg/day (e.g., calcium citrate, calcium carbonate etc.) for at least 12 weeks prior to Screening. In addition, the dose of calcitriol, or alfacalcidol, or calcium should be stable for at least 5 weeks prior to Screening
- For Japan: requirement for a dose of calcitriol ≥1.0 μg/day, or alfacalcidol ≥2.0 μg/day for at least 12 weeks prior to Screening. In addition, the dose of calcitriol or alfacalcidol should be stable for at least 5 weeks prior to Screening. In Japan only (due to local practice and dietary patterns), there is no requirement to exceed a minimum dose of calcium supplements
- Optimization of supplements prior to randomization to achieve the target serum levels of:
Show all 17Show fewer
- 25(OH) vitamin D levels of 20-80 ng/mL (49-200 nmol/L) and
- Magnesium level in the normal range, or just below the normal range and
- Albumin-adjusted or ionized sCa level in the normal range, or just below the normal range
- The subject demonstrates a 24-hour uCa excretion of ≥125 mg/24h (on a sample collected within 52 weeks prior to Screening or during the Screening Period)
- BMI 17- 40 kg/m2 at Screening
- If ≤25 years of age, radiological evidence of epiphyseal closure based on X-ray of nondominant wrist and hand
- Thyroid-stimulating hormone (TSH) within normal laboratory limits within the 6 weeks prior to Visit 1; if on suppressive therapy for a history of thyroid cancer, TSH level must be ≥0.2 mIU/mL
- If treated with thyroid hormone replacement therapy, the dose must have been stable for at least 5 weeks prior to Screening
- eGFR ≥30 mL/min/1.73 m2 during Screening
- Able to perform daily subcutaneous self-injections of study drug (or have a designee to perform injections) via a pre-filled injection pen
- Able and willing to provide written and signed informed consent in accordance with GCP
Who cannot join
- Impaired responsiveness to PTH (pseudohypoparathyroidism) which is characterized as PTH-resistance, with elevated PTH levels in the setting of hypocalcemia
- Any disease that might affect calcium metabolism or calcium-phosphate homeostasis or PTH levels other than HP, such as active hyperthyroidism; Paget disease of bone; severe hypomagnesemia; type 1 diabetes mellitus or poorly controlled type 2 diabetes mellitus (HbA1C >9%, documented HbA1C result drawn within 12 weeks prior to Screening is acceptable); severe and chronic liver, or renal disease; Cushing syndrome; multiple myeloma; active pancreatitis; malnutrition; rickets; recent prolonged immobility; active malignancy (other than low-risk well differentiated thyroid cancer or basal cell skin cancer); active hyperparathyroidism; parathyroid carcinoma within 5 years prior to Screening; acromegaly; or multiple endocrine neoplasia types 1 and 2
- High risk thyroid cancer within 2 years, requiring suppression of TSH <0.2 mIU/mL
- Use of loop diuretics, phosphate binders (other than calcium supplements), digoxin, lithium, methotrexate, biotin >30 μg/day, or systemic corticosteroids (other than as replacement therapy)
- Use of thiazide diuretic within 4 weeks prior to the 24-hour urine collection scheduled to occur within 1 week prior to Visit 1
- Use of PTH-like drugs (whether commercially available or through participation in an investigational trial), including PTH(1-84), PTH(1-34), or other N-terminal fragments or analogs of PTH or PTH-related protein, within 4 weeks prior to Screening
Show all 22Show fewer
- Use of other drugs known to influence calcium and bone metabolism, such as calcitonin, fluoride tablets (>0.5 mg/day), strontium, or cinacalcet hydrochloride, within 12 weeks prior to Screening
- Use of osteoporosis therapies known to influence calcium and bone metabolism, i.e., bisphosphonate (oral or intravenous [IV]), denosumab, raloxifene, or romosozumab therapies within 2 years prior to Screening
- Non-hypocalcemic seizure disorder with a history of a seizure within 26 weeks prior to Screening
- Increased risk for osteosarcoma, such as those with Paget's disease of bone or unexplained elevations of alkaline phosphatase, hereditary disorders predisposing to osteosarcoma, or with a prior history of substantial external beam or implant radiation therapy involving the skeleton
- Pregnant or lactating women
- Male who has a female partner who intends to become pregnant or is of childbearing potential and is unwilling to use adequate contraceptive methods during the trial
- Diagnosed drug or alcohol dependence within 3 years prior to Screening
- 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
- Chronic or severe cardiac disease within 26 weeks prior to Screening including but not limited to congestive heart failure, myocardial infarction, severe or uncontrolled arrhythmias, bradycardia (resting heart rate <48 beats/minute, unless chronic and asymptomatic), symptomatic hypotension or systolic BP <80 mm Hg or diastolic <40 mm Hg or poorly controlled hypertension (systolic BP >165 mm Hg or diastolic >95 mm Hg). In the absence of a prior history of hypertension, an isolated BP >165/95 in the setting of white coat hypertension/anxiety may not be exclusionary and a measurement can be repeated prior to randomization
- Cerebrovascular accident within 5 years prior to Screening
- Within 26 weeks prior to Screening: acute colic due to nephrolithiasis, or acute gout. Subjects with asymptomatic renal stones are permitted
- Participation in any other interventional trial in which receipt of investigational drug or device occurred within 8 weeks (or within 5.5 times the half-life of the investigational drug (whichever comes first) prior to Screening
- Any disease or condition that, in the opinion of the investigator, may require treatment or make the subject unlikely to fully complete the trial, or any condition that presents undue risk from the investigational product or procedures, including treated malignancies that are likely to recur within the approximate 3.5-year duration of the trial
- Known allergy or sensitivity to PTH or any of the excipients [metacresol, mannitol, succinic acid, NaOH/(HCl)]
- Likely to be non-compliant with respect to trial conduct
- Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule
Trial locations and contacts
21 sites in 7 countries.
21 sites in 7 countries
United States9 sites
Ascendis Pharma Investigational Site
Austin, Texas, 78731
Ascendis Pharma Investigational Site
Chicago, Illinois, 60637
Ascendis Pharma Investigational Site
Fort Worth, Texas, 76132
Ascendis Pharma Investigational Site
Greenville, North Carolina, 27834
Ascendis Pharma Investigational Site
New York, New York, 10032
Ascendis Pharma Investigational Site
Reno, Nevada, 89511
Ascendis Pharma Investigational Site
Rochester, Minnesota, 55905
Ascendis Pharma Investigational Site
San Francisco, California, 94143
Ascendis Pharma Investigational Site
Spokane, Washington, 99204
Canada3 sites
Ascendis Pharma Investigational Site
Halifax, Nova Scotia, B3H 2Y9
Ascendis Pharma Investigational Site
Oakville, Ontario, L6M 1M1
Ascendis Pharma Investigational Site
Québec, Quebec, G1V 4G2
Italy3 sites
Ascendis Pharma Investigational Site
Bologna, Emilia-Romagna, 40138
Ascendis Pharma Investigational Site
Pisa, Piacenza, 56126
Ascendis Pharma Investigational Site
Rome, Lazio, 00128
Denmark2 sites
Ascendis Pharma Investigational Site
Aarhus, Central Jutland, 8200
Ascendis Pharma Investigational Site
Copenhagen, Capital Region, 2100
Hungary2 sites
Ascendis Pharma Investigational Site
Budapest, 1083
Ascendis Pharma Investigational Site
Szeged, Csongrád megye, 6720
About this study
Official title: PaTHway TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism
During the first 26 weeks of the trial, participants were randomly assigned to one of two groups: one group received TransCon PTH and one group received placebo. All participants started with study drug at a dose of 18 mcg/day and were individually and progressively titrated to an optimal dose in dose increments of 3 mcg/day. TransCon PTH or placebo were administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors knew who had been assigned to each group. After the 26 weeks, participants continued in the trial as part of a long-term extension study. During the extension, all participants received TransCon PTH, with the dose adjusted to their individual needs. This was a global trial that was conducted in the United States, Canada, Germany, Denmark, Norway, Italy, and Hungary.
- Being tested
- TransCon PTH, Placebo
- Keywords
- Hypoparathyroidism, Parathyroid Hormone, TransCon PTH, PTH(1-34), Prodrug, Sustained Release, Parathyroid Hormone Replacement Therapy, Palopegteriparatide
- Medical subject headings
- Hypoparathyroidism, Endocrine System Diseases, Parathyroid Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TransCon PTHCombination product
TransCon PTH drug product is supplied as a solution with a concentration of 0.3 mg PTH(1-34)/mL in a single-patient-use prefilled pen intended for subcutaneous injection.
PlaceboCombination product
Placebo is supplied as a solution containing the formulation buffer for TransCon PTH in a single-patient-use prefilled pen intended for subcutaneous injection.
| Study group | Role | What they receive |
|---|---|---|
| TransCon PTH | Experimental | TransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose Receives: Combination Product: TransCon PTH |
| Placebo | Placebo comparator | Placebo for TransCon PTH delivered once daily by subcutaneous injection Receives: Combination Product: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy - Primary Endpoint During the Blinded Period
The primary endpoint was a multi-component endpoint that included the percentage of participants who met the following criteria at 26 weeks of blinded treatment: 1) albumin-adjusted serum calcium measured within 4 weeks prior to and on Week 26 visit within the normal range (8.3 to 10.6 mg/dL), and 2) independence from active vitamin D within 4 weeks prior to Week 26 visit (i.e., all daily standing dose of active vitamin D equal to zero AND use of PRN ≤7 days during the 4 weeks), and 3) independence from therapeutic doses of calcium within 4 weeks prior to Week 26 visit (i.e., average daily standing dose of elemental calcium ≤600 mg AND use of PRN doses on ≤7 days during the 4 weeks), and 4) no increase in prescribed study drug within 4 weeks prior to Week 26 visit.
Measured: 26 weeks
Secondary outcomes (5)
Change From Baseline to Week 26 in HPES Symptom - Physical Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Symptom - Physical Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in hypoparathyroidism disease related physical symptoms.
Measured: 26 weeks
Change From Baseline to Week 26 in HPES Symptom - Cognitive Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Symptom - Cognitive Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in hypoparathyroidism disease related cognitive symptoms.
Measured: 26 weeks
Change From Baseline to Week 26 in HPES Impact - Physical Functioning Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Impact - Physical Functioning Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in physical functioning health-related quality of life.
Measured: 26 weeks
Change From Baseline to Week 26 in HPES Impact - Daily Life Domain Score
Change from baseline in Hypoparathyroidism Patient Experience Scale (HPES) Impact - Daily Life Domain score, a disease-specific patient reported outcome, at 26 weeks of treatment. The measure uses a scale of 0-100 and values represent the change in scores from baseline. A decrease in HPES score denotes an improvement in daily health-related quality of life.
Measured: 26 weeks
Change From Baseline to Week 26 in SF-36 Physical Functioning Subscale Score
Change from baseline in the 36-item Short Form Survey (SF-36) Physical Functioning subscale score, a generic health survey, at 26 weeks of treatment. The Physical Functioning subscale uses a range of 19-57.6 and values represent the change in scores from baseline. An increase in SF-36 score denotes an improvement in physical functioning health-related quality of life.
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2021
- Study start16 Feb 2021
- Primary completion12 Jan 2022
- Study completion21 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for HypoparathyroidismStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-blind, placebo controlled, parallel group with participants randomized into two treatment groups (3:1): TransCon PTH at a starting dose of 18 mcg/day and titrated to an optimal dose, and placebo for TransCon PTH
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma Bone Diseases A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, MD, Study director, Ascendis Pharma A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04701203
- Sponsor's protocol ID
- TCP-304
- Other IDs
- 2020-003380-26
- Acronym
- PaTHway
- Status last verified
- Jun 2026
- First submitted
- 6 Jan 2021
- Last updated
- 7 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Brod M, Pfeiffer KM, Beck JF, Smith A. Measuring treatment impacts on symptoms in adults with hypoparathyroidism: findings from the PaTHway trial. J Patient Rep Outcomes. 2024 Aug 13;8(1):94. doi: 10.1186/s41687-024-00757-1.
PubMed 39136801Rejnmark L, Gosmanova EO, Khan AA, Makita N, Imanishi Y, Takeuchi Y, Sprague S, Shoback DM, Kohlmeier L, Rubin MR, Palermo A, Schwarz P, Gagnon C, Tsourdi E, Zhao C, Makara MA, Ominsky MS, Lai B, Ukena J, Sibley CT, Shu AD. Palopegteriparatide Treatment Improves Renal Function in Adults with Chronic Hypoparathyroidism: 1-Year Results from the Phase 3 PaTHway Trial. Adv Ther. 2024 Jun;41(6):2500-2518. doi: 10.1007/s12325-024-02843-8. Epub 2024 Apr 30.
PubMed 38691316
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PaTHway TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism
- ClinicalTrials.gov ID
- NCT04701203
- Acronym / sponsor ID
- PaTHway / TCP-304
- Keywords
- Hypoparathyroidism, Parathyroid Hormone, TransCon PTH, PTH(1-34), Prodrug, Sustained Release, Parathyroid Hormone Replacement Therapy, Palopegteriparatide
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- Ascendis Pharma Bone Diseases A/SIndustry (pharma, biotech, devices)
- Study start
- 16 Feb 2021 (actual)
- Primary completion
- 12 Jan 2022 (actual)
- Study completion
- 21 Jan 2025 (actual)
- First posted
- 8 Jan 2021 (actual)
- Results
- Posted 28 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
During the first 26 weeks of the trial, participants were randomly assigned to one of two groups: one group received TransCon PTH and one group received placebo. All participants started with study drug at a dose of 18 mcg/day and were individually and progressively titrated to an optimal dose in dose increments of 3 mcg/day. TransCon PTH or placebo were administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors knew who had been assigned to each group. After the 26 weeks, participants continued in the trial as part of a long-term extension study. During the extension, all participants received TransCon PTH, with the dose adjusted to their individual needs. This was a global trial that was conducted in the United States, Canada, Germany, Denmark, Norway, Italy, and Hungary.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Double-blind, placebo controlled, parallel group with participants randomized into two treatment groups (3:1): TransCon PTH at a starting dose of 18 mcg/day and titrated to an optimal dose, and placebo for TransCon PTH
| Arm | Type | Receives |
|---|---|---|
| TransCon PTH | Experimental | Combination Product: TransCon PTHTransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose |
| Placebo | Placebo comparator | Combination Product: PlaceboPlacebo for TransCon PTH delivered once daily by subcutaneous injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Males and females, ≥18 years of age
- Subjects with postsurgical chronic HP, or auto-immune, genetic, or idiopathic HP for at least 26 weeks. Diagnosis of HP is established based on historic hypocalcemia in the setting of inappropriately low serum PTH levels
- Requirement for doses of SoC (e.g., calcitriol, alfacalcidol, calcium supplements) at or above a minimum threshold:
- Optimization of supplements prior to randomization to achieve the target serum levels of:
- The subject demonstrates a 24-hour uCa excretion of ≥125 mg/24h (on a sample collected within 52 weeks prior to Screening or during the Screening Period)
Key exclusion criteria (22 listed)
- Impaired responsiveness to PTH (pseudohypoparathyroidism) which is characterized as PTH-resistance, with elevated PTH levels in the setting of hypocalcemia
- Any disease that might affect calcium metabolism or calcium-phosphate homeostasis or PTH levels other than HP, such as active hyperthyroidism; Paget disease of bone; severe hypomagnesemia; type 1 diabetes mellitus or poorly controlled type 2 diabetes mellitus (HbA1C >9%, documented HbA1C result drawn within 12 weeks prior to Screening is acceptable); severe and chronic liver, or renal disease; Cushing syndrome; multiple myeloma; active pancreatitis; malnutrition; rickets; recent prolonged immobility; active malignancy (other than low-risk well differentiated thyroid cancer or basal cell skin cancer); active hyperparathyroidism; parathyroid carcinoma within 5 years prior to Screening; acromegaly; or multiple endocrine neoplasia types 1 and 2
- High risk thyroid cancer within 2 years, requiring suppression of TSH <0.2 mIU/mL
- Use of loop diuretics, phosphate binders (other than calcium supplements), digoxin, lithium, methotrexate, biotin >30 μg/day, or systemic corticosteroids (other than as replacement therapy)
- Use of thiazide diuretic within 4 weeks prior to the 24-hour urine collection scheduled to occur within 1 week prior to Visit 1
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TransCon PTHTransCon PTH drug product is supplied as a solution with a concentration of 0.3 mg PTH(1-34)/mL in a single-patient-use prefilled pen intended for subcutaneous injection. | Combination product | TransCon PTH |
| PlaceboPlacebo is supplied as a solution containing the formulation buffer for TransCon PTH in a single-patient-use prefilled pen intended for subcutaneous injection. | Combination product | Placebo |
Primary outcome
Efficacy - Primary Endpoint During the Blinded Period
Time frame: 26 weeks
The primary endpoint was a multi-component endpoint that included the percentage of participants who met the following criteria at 26 weeks of blinded treatment: 1) albumin-adjusted serum calcium measured within 4 weeks prior to and on Week 26 visit within the normal range (8.3 to 10.6 mg/dL), and 2) independence from active vitamin D within 4 weeks prior to Week 26 visit (i.e., all daily standing dose of active vitamin D equal to zero AND use of PRN ≤7 days during the 4 weeks), and 3) independence from therapeutic doses of calcium within 4 weeks prior to Week 26 visit (i.e., average daily standing dose of elemental calcium ≤600 mg AND use of PRN doses on ≤7 days during the 4 weeks), and 4) no increase in prescribed study drug within 4 weeks prior to Week 26 visit.
5 secondary outcomes
Change From Baseline to Week 26 in HPES Symptom - Physical Domain Score
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Symptom - Cognitive Domain Score
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Impact - Physical Functioning Domain Score
Time frame: 26 weeks
Change From Baseline to Week 26 in HPES Impact - Daily Life Domain Score
Time frame: 26 weeks
Change From Baseline to Week 26 in SF-36 Physical Functioning Subscale Score
Time frame: 26 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39136801Brod M, Pfeiffer KM, Beck JF, Smith A. Measuring treatment impacts on symptoms in adults with hypoparathyroidism: findings from the PaTHway trial. J Patient Rep Outcomes. 2024 Aug 13;8(1):94. doi: 10.1186/s41687-024-00757-1.
- Derived from this study: PubMed 38691316Rejnmark L, Gosmanova EO, Khan AA, Makita N, Imanishi Y, Takeuchi Y, Sprague S, Shoback DM, Kohlmeier L, Rubin MR, Palermo A, Schwarz P, Gagnon C, Tsourdi E, Zhao C, Makara MA, Ominsky MS, Lai B, Ukena J, Sibley CT, Shu AD. Palopegteriparatide Treatment Improves Renal Function in Adults with Chronic Hypoparathyroidism: 1-Year Results from the Phase 3 PaTHway Trial. Adv Ther. 2024 Jun;41(6):2500-2518. doi: 10.1007/s12325-024-02843-8. Epub 2024 Apr 30.
Related trials for Hypoparathyroidism
Recruiting
NIRAF-Guided Parathyroid Identification During Thyroidectomy
Hypocalcemia, Hypoparathyroidism
RecruitingPhase 3
A Phase 3 Randomized Clinical Trial to Investigate the Safety and Efficacy of Palopegteriparatide at Doses Greater Than 30 μg/Day in Adult Participants With Hypoparathyroidism
Hypoparathyroidism, Endocrine System Diseases
RecruitingPhase 3
Efficacy, Safety, and Tolerability of Encaleret in Participants With Chronic Hypoparathyroidism
Hypoparathyroidism, Chronic Hypoparathyroidism
RecruitingPhase 3
A Study to Investigate Canvuparatide Compared With Placebo in Adult Patients With Hypoparathyroidism
Hypoparathyroidism
RecruitingObservational study
The ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) Study is the Largest U.S.-Based Prospective Study Demonstrating a Circulating Fetal Cell (CFC) Based Approach to Non-invasive Fetal Risk Assessment
Pregnant Individuals, Aneuploidy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director, MD study director
Ascendis Pharma A/S
21 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing TransCon PTH in people with Hypoparathyroidism and Endocrine System Diseases, run by Ascendis Pharma Bone Diseases A/S at 21 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Canada, Italy, Denmark, Hungary and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy - Primary Endpoint During the Blinded Period (time frame: 26 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04701203, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026.