Evidence map›Paper›PMID 34347093›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2022

PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism.

Aliya A Khan, Lars Rejnmark, Mishaela Rubin, Peter Schwarz, Tamara Vokes, Bart Clarke, Intekhab Ahmed, Lorenz Hofbauer, Claudio Marcocci, Uberto Pagotto and 9 more

2 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase IIMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 41 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed, 4 pooled it
5.6field-weighted citation impact, top 3% of its field
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.

NCT04009291 phase2completednot on this map

PaTH Forward: A Phase 2, 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

TypeinterventionalSponsorAscendis Pharma A/SRan2019 to 2025Enrolled59ConditionsHypoparathyroidism, Endocrine System Diseases, Parathyroid DiseasesArmsTransCon PTH, Placebo for TransCon PTH
NCT07160348 not yet recruitingnot on this mapstarted 2025, after this paper: background citation

Translation and Validation of the Hypoparathyroidism Patient Experience Scales (HPES) Questionnaire in Patients With Hypoparathyroidism for Use in the Greek Population

TypeobservationalSponsorAristotle University Of ThessalonikiRan2025 to 2026Enrolled200ConditionsHypoparathyroidismArmsQuestionnaire and Physical Exam
3 · Its place in the literature

Who cites it

41 citing papers in PubMed, 4 syntheses or guidelines pooled it, 64 citations in OpenAlex.

  1. Pooled it
  2. Guideline
  3. Autosomal Dominant Hypocalcemia Type 1: A Systematic Review.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2022
    Pooled it
  4. Safety and Efficacy of PTH 1-34 and 1-84 Therapy in Chronic Hypoparathyroidism: A Meta-Analysis of Prospective Trials.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2022
    Pooled it
  5. Palopegteriparatide for adults with chronic hypoparathyroidism: skeletal dynamics through 3 years of the phase 2 PaTH forward trial.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2026
    Trial
  6. Trial
  7. Trial
  8. Efficacy and Safety of Parathyroid Hormone Replacement With TransCon PTH in Hypoparathyroidism: 26-Week Results From the Phase 3 PaTHway Trial.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2023
    Trial
  9. Article
  10. Article
  11. Article
  12. Article
  13. Review
  14. Article
  15. Review
  16. Review
  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. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors at 14 institutions in 6 countries.

Aliya A KhanDepartment of Endocrinology and Metabolism and Geriatrics, McMaster University, Hamilton, Ontario L8S 4L8, Canada.
Lars RejnmarkDepartment of Endocrinology and Internal Medicine, Aarhus University Hospital, 8200 Aarhus N, Denmark.
Mishaela RubinMetabolic Bone Disease Unit, Columbia University, New York, New York 10027, USA.
Peter SchwarzDepartment of Endocrinology, Rigshospitalet, Copenhagen and Faculty of Health Sciences, Copenhagen University, 2200 Copenhagen N, Denmark.
Tamara VokesSection of Endocrinology, Diabetes and Metabolism, University of Chicago, Chicago, Illinois 60637, USA.
Bart ClarkeDivision of Endocrinology, Diabetes, Metabolism, and Nutrition, Mayo Clinic, Rochester, Minnesota 55905, USA.
Intekhab AhmedThomas Jefferson University, Philadelphia, Pennsylvania 19107, USA.
Lorenz HofbauerUniversitätsklinikum Dresden, 01307 Dresden, Germany.
Claudio MarcocciUniversity of Pisa, 56126 Pisa, Italy.ORCID 0000-0001-6568-8588
Uberto PagottoDivision of Endocrinology and Diabetes Prevention and Care, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Department of Medical and Surgical Sciences (DIMEC), Alma Mater Studiorum University of Bologna, 40126 Bologna, Italy.
Andrea PalermoUnit of Endocrinology and Diabetes, Campus Bio-Medico University, 00128 Rome, Italy.ORCID 0000-0002-1143-4926
Erik EriksenOslo University Hospital, Institute of Clinical Medicine, 0372 Oslo, Norway.
Meryl BrodThe Brod Group, Mill Valley, California 94941, USA.
Denka MarkovaAscendis Pharma Inc , Palo Alto, California 94301, USA.
Alden SmithAscendis Pharma Inc , Palo Alto, California 94301, USA.
Susanne PihlAscendis Pharma A/S, 2900 Hellerup, Denmark.
Sanchita MouryaAscendis Pharma Inc , Palo Alto, California 94301, USA.
David B KarpfAscendis Pharma A/S, 2900 Hellerup, Denmark.
Aimee D ShuAscendis Pharma Inc , Palo Alto, California 94301, USA.ORCID 0000-0003-1801-5289
Ascendis Pharma (Denmark) · DKAarhus University Hospital · DKBrod Group · USColumbia University · USKlinik und Poliklinik für Psychotherapie und Psychosomatik · DEMayo Clinic · USMcMaster University · CAOslo University Hospital · NOThomas Jefferson University · USUniversità Campus Bio-Medico · ITUniversity of Bologna · ITUniversity of Chicago · USUniversity of Copenhagen · DKUniversity of Pisa · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

contextHypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism.

objectiveThis work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism.

methodsThis phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 µg PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during which TransCon PTH dose was titrated (6-60 µg PTH[1-34]/day).

resultsBy Week 26, 91% of participants treated with TransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 μg/day and calcium [Ca] ≤ 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events.

conclusionTransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements (≤ 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism.

Indexed as

AdultAgedCalciumDelayed-Action PreparationsDouble-Blind MethodDrug Administration ScheduleFemaleHormone Replacement TherapyHumansHypoparathyroidismMaleMiddle AgedParathyroid HormonePatient Reported Outcome MeasuresPlacebosProdrugsCalciumDelayed-Action PreparationsParathyroid HormonePlacebosProdrugsVitamin Dhypoparathyroidismparathyroid hormoneprodrugPTH(1-34)replacement therapyTransCon PTH

Identifiers

PMID34347093
PMCPMC8684498
OpenAlexW3187660342

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

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.