Natpara (recombinant human parathyroid hormone 1-84) / Takeda - LARVOL DELTA

Preliminary Results from a Prospective Cohort Study on Real-World Data of Chronic Hypoparathyroidism Treated with Trans-Con PTH (ESPE-ESE 2025) - "Background: Chronic hypoparathyroidism (HypoPT) is a rare endocrine disorder characterized by deficient parathyroid hormone (PTH) levels, leading to hypocalcemia and dependency on calcium and active vitamin D., Trans-conPTH 1-34 formulation (Yorvipath), has emerged as a potential therapeutic option for patients with uncontrolled hypoparathyroidism or those previously treated with rhPTH(1-84)...Additionally, 15 patients (40%) were on hydrochlorothiazide for hypercalciuria... Preliminary findings suggest that Yorvipath is effective in reducing or eliminating the need for calcium and activated vitamin-D treatment in patients with HypoPT. Most patients achieved Ca/active VitD independence within the first few weeks of treatment. Further studies with longer follow-up are warranted to confirm the long-term efficacy and safety of Yorvipath in this patient population."

Clinical • Real-world • Real-world evidence • Endocrine Cancer • Endocrine Disorders • Genetic Disorders • Hypoparathyroidism • Immunology • Metabolic Disorders • Oncology • Sarcoidosis • Solid Tumor • Thyroid Gland Carcinoma

Palopegteriparatide for the treatment of Autosomal Dominant Hypocalcemia type 1 (ADH1) (ESPE-ESE 2025) - "He was treated with calcium carbonate 500mg 1x2, alfacalcidol 1 mcg 1x1, cholecalciferol 800UI 1x1, hydrochlorothiazide 25mg 1x1, rhPTH (Natpar) 75-100 mcg 1x1 and magnesium supplements. He also received dasatinib for the CML... Until the launch of the calcilytics, palopegteriparatide (Yorvipath) may represent a valuable alternative for the management of ADH1."

Chronic Myeloid Leukemia • Endocrine Disorders • Hematological Malignancies • Hypoparathyroidism • Leukemia • Oncology • CASR

Switch from rhPTH1-84 to TransCon PTH in patients with hypoparathyroidism normalizes phosphate, magnesium metabolism, and bone turnover. (ESPE-ESE 2025) - "Switching treatment from rhPTH1-84 to TransCon PTH was followed by significant changes towards normalization in calcium, phosphate, and magnesium metabolism and bone turnover during the first six months of treatment."

Clinical • Cataract • Endocrine Disorders • Hypoparathyroidism • Metabolic Disorders • Nephrology • Ophthalmology • Rare Diseases • Renal Calculi • Renal Disease • FGF23

Switch from rhPTH1-84 to TransCon PTH with individual Dose Adjustment in Adult Hypoparathyroidism – results for 40 patients one month after treatment transition. (ESPE-ESE 2025) - "Switching treatment from rhPTH1-84 to TransCon PTH using the initial dose of 18 µg proved to be efficient independently of the prior rhPTH1-84 dose. Adverse events were frequent, albeit mild to moderate and no patient withdrew from treatment."

Clinical • CNS Disorders • Endocrine Disorders • Fatigue • Gastrointestinal Disorder • Hypoparathyroidism • Musculoskeletal Pain • Pain • Sleep Disorder

Preoperative stroke predicts new postoperative clinically significant stroke in patients undergoing surgery for left-sided infective endocarditis. (PubMed, JTCVS Open) - "Among patients with LSIE undergoing surgery, PREOS is a risk factor for PCS and new renal failure. Patients with PREOS may require greater level of care upon discharge and may benefit from more stringent preoperative evaluation."

Journal • Cardiovascular • Hematological Disorders • Renal Disease

Evaluating SinergiAPS, an intervention based on patient feedback to improve patient safety in primary care: a cluster randomized trial. (PubMed, BMC Med) - P=N/A | "In the context of the health emergency caused by the COVID-19 pandemic, SinergiAPS did not improve patient safety in Spanish PHC centres."

Clinical • Journal • Infectious Disease • Novel Coronavirus Disease

NATPARA KAB: A Survey to Assess Participants' and Physicians' Knowledge, Attitudes and Behavior When Using NATPARA (clinicaltrials.gov) - P=N/A | N=200 | Recruiting | Sponsor: Shire | Trial completion date: Dec 2024 ➔ Jan 2026 | Trial primary completion date: Dec 2024 ➔ Jan 2026

Trial completion date • Trial primary completion date • Endocrine Disorders • Hypoparathyroidism

Personalized parathyroid hormone therapy for hypoparathyroidism: Insights from pharmacokinetic-pharmacodynamic modelling. (PubMed, Br J Clin Pharmacol) - "The PKPD model for PTH was completed with the unique data of a single patient who received PTH according to various dosing regimens, including continuous infusion. Continuous administration of PTH is favoured because it permanently increases the phosphate clearance and therefore needs to be further investigated."

Journal • PK/PD data • Endocrine Disorders • Hypoparathyroidism • Nephrology • Renal Calculi

Management of autosomal dominant hypocalcemia type 1: Literature review and clinical practice recommendations. (PubMed, J Endocrinol Invest) - "We provide an overview of conventional and new treatments for ADH1 patients. Based on these data, we propose practical recommendations to assist clinicians in the management of ADH1 patients."

Journal • Review • Endocrine Disorders • Hypoparathyroidism • Nephrology • Renal Calculi

Paramedic or GP consultations in primary care: prospective study comparing costs and outcomes. (PubMed, Br J Gen Pract) - "Well-designed training and supervision are needed to ensure PGPs have the right knowledge and can clearly convey health care plans to patients. While PGPs may reduce GP workload pressure, they do not necessarily reduce NHS costs."

Journal

HEXT (Hypo EXTended): Effect of PTH on Skeleton in Hypoparathyroidism (clinicaltrials.gov) - P3 | N=62 | Completed | Sponsor: Columbia University | Unknown status ➔ Completed

Trial completion • Endocrine Disorders • Hypoparathyroidism • Renal Disease

Validation of the patient reported experiences and outcomes of safety in primary care compact Form Brazil. (PubMed, PLoS One) - "The Brazilian version of the PREOS-PC Compact Form Brazil composed of four domains (PrA, PA, EPaS and OPaS) and 16 items presented evidence of validation of its psychometric properties and can be used to evaluate the experiences and results of patient safety in Primary Health Care in the Brazilian context."

Journal

Patient perspectives of general practice consultation for musculoskeletal disorders: A qualitative study. (PubMed, Musculoskeletal Care) - "Patients commented that FCPP consultations provided quick and accurate diagnoses and targeted advice. Recommendations for future practice included prompt access to face-to-face appointments, phone calls to be answered more quickly, improved communication for test results and follow ups, patients to feel listened to with a more individualised approach, and better continuity of care."

Journal • Musculoskeletal Diseases

Open-label extension of a randomized trial investigating safety and efficacy of rhPTH(1-84) in hypoparathyroidism. (PubMed, JBMR Plus) - P3 | "The effects observed on urine calcium and the safety profile are consistent with previous findings. NCT03364738."

Clinical • Endocrine Disorders • Hypoparathyroidism • Rare Diseases • Renal Disease

Evaluation of SINERGIAPS, an intervention to improve patient safety in primary healthcare centers in Spain based on patients' perceptions and experiences: a protocol for a hybrid type I randomized clinical trial. (PubMed, Front Public Health) - P3 | "The intervention group will receive two audits (baseline and intermediate at 12 months) based on information collected through the administration of the PREOS-PC questionnaire (a measure of patient-reported patient safety) to a convenience sample of 100 patients per center...This study will expand the scarce body of evidence existing regarding the effects and implementation of interventions aimed at promoting patient and family engagement in primary healthcare, specifically for enhancing patient safety. The study has the potential to produce an impact on clinical practice, healthcare systems, and population health.Clinical Trial Registration: https://clinicaltrials.gov/study/NCT05958108?term=sinergiAPS&rank=1 (NCT05958108)."

Clinical • Journal

Continuous Subcutaneous Delivery of rhPTH(1-84) and rhPTH(1-34) by Pump in Adults With Hypoparathyroidism. (PubMed, J Endocr Soc) - "In this case series of 12 patients with chronic hypoparathyroidism treated with rhPTH(1-84)/rhPTH(1-34) administered via a pump, improvement in clinical and biochemical parameters were observed in the majority of the patients. Our observations indicate benefits of pump administration of rhPTH that warrant further investigation."

Journal • Endocrine Disorders • Hypoparathyroidism • Renal Disease

THE USE OF AN INSULIN PUMP IN THE TREATMENT OF A PATIENT WITH SEVERE POSTOPERATIVE HYPOPARATHYROIDISM (ATTD 2024) - "The insulin pump, as a technology that was developed exclusively for the treatment of diabetes, can be very successfully used in other diseases. We do not have an answer as to why the use of a tethered insulin pump led to such a large oscillation of the findings."

Clinical • Diabetes • Endocrine Disorders • Hypoparathyroidism • Metabolic Disorders • Renal Disease

An industry perspective on current QSP trends in drug development. (PubMed, J Pharmacokinet Pharmacodyn) - "2023 marks the 10th anniversary of Natpara's submission to the US FDA, which led to the first recorded regulatory interaction where a decision was supported by Quantitative and Systems Pharmacology (QSP) simulations...What level of validation is required to inform drug development decisions? This article reflects on all these questions, in particular addressing those audiences with limited line-of-sight into the drug industry decision-making machinery."

Journal

Evaluation of the psychometric performance of the Spanish and Catalan versions of the patient reported experiences and Outcomes of Safety in Primary Care (PREOS-PC)-Compact questionnaire. (PubMed, Eur J Gen Pract) - "IRT models showed disordered thresholds for 'Practice activation' and 'Harm burden' but showed excellent fit after reducing the response categories. The Spanish and Catalan versions of the PREOS-PC-Compact are broadly valid and reliable tools to measure patient safety in Spanish primary care centres; confirmation of lower-than-expected test-rest reliability merits further examination ."

Journal

Transitory Activation and Improved Transition from Erosion to Formation within Intracortical Bone Remodeling in Hypoparathyroid Patients Treated with rhPTH(1-84). (PubMed, JBMR Plus) - "© 2023 The Authors. JBMR Plus published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research"

Clinical • Endocrine Disorders • Hypoparathyroidism • Renal Disease

Extended Treatment with rhPTH(1-84) in Adult Patients with Chronic Hypoparathyroidism. (PubMed, Endocr Pract) - P3, P4 | "The effects of rhPTH(1-84) on biochemical, skeletal, and health-related quality-of-life parameters did not wane with extended use."

Journal • Endocrine Disorders • Hypoparathyroidism • Renal Disease

NATPARA KAB: A Survey to Assess Participants' and Physicians' Knowledge, Attitudes and Behavior When Using NATPARA (clinicaltrials.gov) - P=N/A | N=200 | Recruiting | Sponsor: Shire | Trial completion date: Dec 2023 ➔ Dec 2024 | Trial primary completion date: Dec 2023 ➔ Dec 2024

Trial completion date • Trial primary completion date • Endocrine Disorders • Hypoparathyroidism • Renal Disease

Patients' Perceptions of Safety in Primary Healthcare Settings: A Cross-Sectional Study in the Qassim Region of Saudi Arabia. (PubMed, Healthcare (Basel)) - "The Patient-Reported Experiences and Outcomes of Safety in Primary Care (PREOS-PC) questionnaire was used to measure patients' perceived safety and experience in primary healthcare settings within the past year...Patient-reported safety experiences reported in our study offer valuable insights into primary care safety in Saudi Arabia. Collecting routine patient feedback is crucial for addressing identified safety problems and implementing standardized procedures."

Journal • Observational data

A Phase I Randomized Trial of Once-Daily Versus Twice-Daily Recombinant Human Parathyroid Hormone (1-84) for Hypoparathyroidism. (PubMed, JBMR Plus) - "Recombinant human parathyroid hormone (1-84), rhPTH(1-84), is an approved adjunctive treatment to oral calcium and active vitamin D for adult patients with hypoparathyroidism; however, there is limited information on the effect of twice daily (BID) dosing of rhPTH(1-84).."

Clinical • Journal • P1 data • Endocrine Disorders • Hypoparathyroidism • Renal Disease

Chronic Asymptomatic Hypoparathyroidism Due To Two Separate Single Nucleotide Heterozygous Mutations In The CaSR Gene (ENDO 2023) - "Due to lack of specific symptoms, patient was not initiated on specific therapies such as calcitriol or Natpara (recombinant PTH) and was advised to continue taking the daily calcium/vitamin D3 supplementation...Hypoparathyroidism due to CASR gene mutation has a varied clinical presentation. One-third of cases do not develop any clinical symptoms, such as our patient and hence do not warrant any specific treatment other than periodic clinical and biochemical assessment.*Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins."

Endocrine Disorders • Hypoparathyroidism • Immunology • Movement Disorders • Renal Disease • CASR