Evidence map›Paper›PMID 39376010›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2025

Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial.

Bart L Clarke, Aliya A Khan, Mishaela R Rubin, Peter Schwarz, Tamara Vokes, Dolores M Shoback, Claudia Gagnon, Andrea Palermo, Lisa G Abbott, Lorenz C Hofbauer and 10 more

Erratum issuedAbstract readClinical Trial, Phase IIIRandomized Controlled TrialMulticenter Study
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 21 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

21 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Guideline
  2. Trial
  3. Trial
  4. Trial
  5. Article
  6. Article
  7. Article
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  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Article
  17. Advances in Parathyroid Hormone-based medicines.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2025
    Review
  18. Article
  19. Article
  20. Increased risk of vertebral fractures and reduced risk of femur fractures in patients with chronic hypoparathyroidism: a nationwide cohort study in Sweden.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2025
    Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

20 authors.

Bart L ClarkeEndocrinology, Mayo Clinic E18-A, Rochester, MN 55905, USA.
Aliya A KhanEndocrinology, Metabolism, and Geriatrics, McMaster University, Hamilton, ON L8S 4L8, Canada.
Mishaela R RubinEndocrinology, Columbia University, New York, NY 10032, USA.
Peter SchwarzInternal Medicine and Endocrinology, Rigshospitalet, 2100 Copenhagen, Denmark.
Tamara VokesEndocrinology, Diabetes, and Metabolism, University of Chicago, Chicago, IL 60637, USA.
Dolores M ShobackEndocrinology, VA Medical Center, University of California, San Francisco, San Francisco, CA 94143, USA.ORCID 0000-0003-2936-4468
Claudia GagnonEndocrinology and Nephrology, CHU de Québec-Université Laval Research Centre, Quebec City, QC G1V 4G2, Canada.ORCID 0000-0001-9623-962X
Andrea PalermoUnit of Metabolic Bone and Thyroid Disorders, Fondazione Policlinico Campus Bio-medico, 00128 Rome, Italy.ORCID 0000-0002-1143-4926
Lisa G AbbottEndocrinology, Northern Nevada Endocrinology, Reno, NV 89511, USA.
Lorenz C HofbauerEndocrinology, Diabetes, and Metabolic Bone Diseases, Technische Universität Dresden Medical Center, 01307 Dresden, Germany.
Lynn KohlmeierEndocrinology and Spokane Osteoporosis, Arthritis Northwest Research Center, Spokane, WA 99223, USA.
Filomena CetaniEndocrine Unit, University Hospital of Pisa, 56126 Pisa, Italy.ORCID 0000-0003-2558-9547
Susanne PihlClinical Pharmacology and Bioanalysis, Ascendis Pharma A/S, 2900 Hellerup, Denmark.
Xuebei AnBiostatistics, Ascendis Pharma Inc, Palo Alto, CA 94304, USA.
Alden R SmithGlobal Health Economics Outcomes Research, Ascendis Pharma Inc, Palo Alto, CA 94304, USA.
Bryant LaiEndocrine Medical Sciences, Ascendis Pharma Inc, Palo Alto, CA 94304, USA.
Jenny UkenaEndocrine Medical Sciences, Ascendis Pharma Inc, Palo Alto, CA 94304, USA.
Christopher T SibleyEndocrine Medical Sciences, Ascendis Pharma Inc, Palo Alto, CA 94304, USA.
Aimee D ShuEndocrine Medical Sciences, Ascendis Pharma Inc, Palo Alto, CA 94304, USA.ORCID 0000-0003-1801-5289
Lars RejnmarkClinical Medicine and Endocrinology, Aarhus University Hospital, 8200 Aarhus N, Denmark.ORCID 0000-0002-2152-4247

Funding

Ascendis Pharma Bone Diseases A/S, Hellerup, Denmark
6 · The paper itself

Abstract

contextConventional therapy for hypoparathyroidism aims to alleviate symptoms of hypocalcemia but does not address insufficient parathyroid hormone (PTH) levels.

objectiveAssess the long-term efficacy and safety of TransCon PTH (palopegteriparatide) for hypoparathyroidism.

designPhase 3 trial with a 26-week, double-blind, placebo-controlled period followed by a 156-week, open-label extension (OLE).

settingTwenty-one sites across North America and Europe.

participantsA total of 82 adults with hypoparathyroidism were randomized and received study drug and 78 completed week 52. INTERVENTION(S): All OLE participants received TransCon PTH administered once daily. MAIN OUTCOME MEASURE(S): Multicomponent efficacy endpoint: proportion of participants at week 52 who achieved normal serum calcium (8.3-10.6 mg/dL) and independence from conventional therapy (≤600 mg/day of elemental calcium and no active vitamin D). Other efficacy endpoints included patient-reported outcomes and bone mineral density. Safety was assessed by 24-hour urine calcium and treatment-emergent adverse events.

resultsAt week 52, 81% (63/78) met the multicomponent efficacy endpoint, 95% (74/78) achieved independence from conventional therapy, and none required active vitamin D. Patient-reported outcomes showed sustained improvements in quality of life, physical functioning, and well-being. Mean bone mineral density Z-scores decreased toward age- and sex-matched norms from baseline to week 52. Mean (SD) 24-hour urine calcium excretion decreased from 376 (168) mg/day at baseline to 195 (114) mg/day at week 52. Most treatment-emergent adverse events were mild or moderate and none led to trial discontinuation during the OLE.

conclusionAt week 52 of the PaTHway trial, TransCon PTH showed sustained efficacy, safety, and tolerability in adults with hypoparathyroidism.

Indexed as

HypoparathyroidismParathyroid HormoneAdultAgedBone DensityCalciumDouble-Blind MethodFemaleHumansMaleMiddle AgedQuality of LifeTreatment OutcomeCalciumParathyroid Hormonehormone replacement therapyhypocalcemiahypoparathyroidismpalopegteriparatideparathyroid hormonequality of life

Identifiers

PMID39376010
PMCPMC11913112

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.