Impact of treatment with TransCon PTH on Quality of Life, cognitive function, brain structure, and cerebral capillary bloodflow in patients with hypoparathyroidism

2025-523930-14-00 Therapeutic use (Phase IV) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 1 sites

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)
Status Authorised, recruitment pending
Participants planned 40
Countries 1
Sites 1

Hypoparathyroidism

We would like to compare patients with chronic HypoPT treated with TransCon for a long-time period with chronic HypoPT patients treated with conventional treatment (activated vitamin D analogue and calcium supplements) to investigate whether the effect of HypoPT on QoL, neuropsychiatric indices, brain structures, and c…

Key facts

Sponsor
Region Midtjylland
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Hormonal diseases [C19]
Decision date (initial)
2026-04-20
Transition trial
No
Low-intervention
Yes
Rare-disease indication
Yes
Vulnerable population
No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy

We would like to compare patients with chronic HypoPT treated with TransCon for a long-time period with chronic HypoPT patients treated with conventional treatment (activated vitamin D analogue and calcium supplements) to investigate whether the effect of HypoPT on QoL, neuropsychiatric indices, brain structures, and capillary blood flow are reversed with PTH treatment.
We will apply our contrast-enhanced MRI based method to examine whether differences in brain structure and capillary dysfunction depends on the treatment used.

Conditions and MedDRA coding

Hypoparathyroidism

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

  1. Male or female with age between 18 and 80 years.
  2. A low endogenous PTH production is verified by low plasma levels of intact PTH, necessitating treatment with 1-αhydroxylated vitamin D analogs.
  3. Stable P-calcium levels 1 month prior to inclusion
  4. Stable thyroid function as measured by thyroid stimulating hormone (TSH)
  5. Speaks and reads Danish

Exclusion criteria 11

  1. Reduced kidney function (eGFR < 30 mL/min/1.73m2).
  2. Unwillingness to participate
  3. Uncontrolled diabetes type 1 or 2
  4. Uncontrolled hypertension
  5. Clinical suspicion of major depression (also if treated)
  6. Clinical suspicion of ongoing alcohol abuse (or alcohol-related dementia)
  7. Other organic or psychiatric cause the patients’ symptoms
  8. Contraindications to contrast-enhanced MRI.
  9. Metal implants close to the head, which will interfere with the MRI or pacemaker. The patients will complete a metal scheme
  10. Claustrophobia
  11. Pregnancy

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Difference in microvascular hemodynamic between PTH- and conventionally treated patients with chronic HypoPT

Secondary endpoints 3

  1. Correlation between cognitive impairment measured by neuropsychological assessment and capillary transit time heterogeneity, cortical blood volume and hemodynamic parameters.
  2. Status of cognitive function in PTH treated compared to conventional treated controls
  3. Difference in groups in size of structures in the brain.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Yorvipath 168 micrograms/0.56 mL solution for injection in pre‑filled pen

PRD10961896 · Product

Active substance
Palopegteriparatide
Substance synonyms
ACP-014, Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinyl]thio]hexyl]amino]ethyl]amino]carbonyl]-2-methylalanyl-teriparatide (2:1), TERIPARATIDE CONJUGATED TO A MULTI-ARM POLYETHYLENE GLYCOL CARRIER MOLECULE THROUGH A CLEAVABLE LINKER, TRANSCON PTH
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS INJECTION
Max daily dose
420 µg microgram(s)
Max total dose
999 µg microgram(s)
Max treatment duration
999 Month(s)
Authorisation status
Authorised
ATC code
H05AA05 — -
Marketing authorisation
EU/1/23/1766/001
MA holder
ASCENDIS PHARMA BONE DISEASES A/S
MA country
Liechtenstein
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/20/2350
Modified vs. Marketing Authorisation
No

Comparator 1

Alfacalcidol Stada 1 micrograme capsule moi

PRD10562012 · Product

Active substance
Alfacalcidol
Pharmaceutical form
CAPSULE, SOFT
Route of administration
ORAL
Max daily dose
10 µg microgram(s)
Max total dose
10 µg microgram(s)
Max treatment duration
999 Month(s)
Authorisation status
Authorised
ATC code
A11CC03 — ALFACALCIDOL
Marketing authorisation
15039/2023/07
MA holder
STADA M&D S.R.L.
MA country
Romania
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Region Midtjylland

Sponsor organisation
Region Midtjylland
Address
Palle Juul-Jensens Boulevard 99
City
Aarhus N
Postcode
8200
Country
Denmark

Scientific contact point

Organisation
Region Midtjylland
Contact name
tanja tvistholm sikjær

Public contact point

Organisation
Region Midtjylland
Contact name
tanja tvistholm sikjær

Third parties 1

OrganisationCity, countryDuties
GCP enhederne
ORL-000014084
AARHUS N, Denmark On site monitoring

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Authorised, recruitment pending 40 1
Rest of world 0

Investigational sites

Denmark

1 site · Authorised, recruitment pending
Aarhus Universitet
Department of Endocrinology and Internal Medicien, Palle Juul-Jensens Boulevard 82, 8200, Aarhus N

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) Protocol version 1 1.3
Recruitment arrangements (for publication) 9 screeningssprgsmal version 1_0 1
Recruitment arrangements (for publication) Recruitment arrangements version 1 1.2
Recruitment arrangements (for publication) Recruitment material letter version 1 1.1
Subject information and informed consent form (for publication) Dine rettigheder som forsgsperson i forsg med medicin 1
Subject information and informed consent form (for publication) L_SIS and ICF version 1_2 1.3
Subject information and informed consent form (for publication) Tillg til samtykkeblanket - Retten til ikke-viden 1
Summary of Product Characteristics (SmPC) (for publication) Alfacalcidol 1
Summary of Product Characteristics (SmPC) (for publication) yorvipath-epar-product-information 1
Synopsis of the protocol (for publication) D1 protocol synopsis danish version1 1
Synopsis of the protocol (for publication) D1 protocol synopsis engelsk version 1 1

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-12-22 Denmark Acceptable
2026-04-16
2026-04-20