Factor XII-associated Cold Autoinflammatory Syndrome (FACAS) Linked to Kallikrein-kinin Pathology: Proof of Concept Treatment With Lanadelumab (DX-2930)

This is a Phase 2, exploratory, proof-of-concept, single-center, open-label pilot study to assess the effects and safety of Lanadelumab in patients with FXII-associated cold autoinflammatory syndrome (FACAS).

Start Date05 Oct 2020

Sponsor / Collaborator

Charité - Universitätsmedizin Berlin

[+1]

NCT04234490

/ CompletedNot Applicable

Longitudinal Evaluation of Patient Outcomes and Impact Assessment on Family Members of Home Parenteral Nutrition: New Directions for Research

Patients with type 3 intestinal failure are completely reliant on artificial feeding and often manage this at home; home parenteral nutrition (HPN). HPN therapy is life saving for these patients.
The Parenteral Nutrition Impact Questionnaire (PNIQ) is a proven tool for measuring quality of life when receiving HPN.
This study will recruit HPN patients across the United Kingdom (UK) and ask them to complete the PNIQ survey at several different time points. This will assess the impact of HPN over time. Family members involved in the participants HPN care will also be asked to complete a carer burden survey (at one time point) to assess the impact of HPN on carers.

Start Date02 Mar 2020

Sponsor / Collaborator

The University of Manchester

[+1]

NCT03230149

/ CompletedNot ApplicableIIT

Prevalence and Characteristics of Fabry Disease (FD) in Patients With Stroke or Small Fiber Neuropathy

FD is pan-ethnic. Its reported annual incidence of 1 in 100,000 may underestimate the true prevalence of the disease. Indeed, recently, in addition with affected males FD developing a "classic" phenotype, " cardiac variant " and " renal variant " have been reported for FD patients with predominant or exclusive cardiac or renal involvement. " Neurologic variant " could exist.
Nervous system can be affect by FD leading to cerebrovascular diseases (ischemic or haemorrhagic strokes, TIA (Transient Ischemic Attacks) or peripheral neuropathy (acroparesthesias and pain).
Aims will be to determine the prevalence of Fabry disease in patients with stroke or small fiber neuropathy, and their characteristics

Start Date12 Mar 2018

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

[+1]

100 Clinical Results associated with Shire International GmbH

0 Patents (Medical) associated with Shire International GmbH

Literatures (Medical) associated with Shire International GmbH

01 Jun 2021·Clinical Nutrition ESPEN

Predictors and timing of response to teduglutide in patients with short bowel syndrome dependent on parenteral support

Article

Author: Seidner, Douglas L ; Mu, Fan ; Kelkar, Sneha S ; Joly, Francisca ; Olivier, Clement ; Chen, Kristina ; Xie, Jipan

BACKGROUND AND AIMSThis study aimed to identify predictors and estimate time to teduglutide response among adult patients with short bowel syndrome with intestinal failure (SBS-IF) dependent on parenteral support (PS).METHODSPost-hoc analysis was performed on individual patient data from teduglutide-treated patients in the phase III teduglutide trial STEPS and the STEPS-2 extension. Response was defined as ≥20% PS volume reduction from baseline for two consecutive visits. Early responders experienced the reduction at 20 and 24 weeks during STEPS while late responders experienced the reduction during STEPS-2. Timing and predictors for response were assessed among the treated population using Cox proportional hazard model. Time to response was compared in aetiological subgroups using Kaplan-Meier analysis. Patient characteristics and time to response were compared between early vs. late responders.RESULTSA total of 34 patients were included in this analysis; overall median time to response was 4.3 months. The presence of stoma predicted a positive response to teduglutide (hazard ratio [HR]: 5.6; 95% confidence interval [CI]: 1.4-21.9; p = 0.013). Vascular disease (vs. inflammatory bowel disease [IBD]) as cause of major intestinal resection (HR: 0.2; 95% CI: 0.0-0.8; p = 0.015), presence of ileocecal valve (HR: 0.1; 95% CI: 0.0-0.8; p = 0.047), and female sex (HR: 0.3; 95% CI: 0.1-1.0; p = 0.026) are negatively associated with response. In subgroup analyses, patients with IBD (vs. vascular disease), with (vs. without) a stoma, and without (vs. with) colon-in-continuity had a shorter time to response (all p < 0.05). The mean times to response were 3.6 (standard deviation (SD): 1.1) months for early responders (n = 27) and 10.0 (SD: 6.1) months for late responders (n = 7). Fewer early responders had colon-in-continuity (51.9%) and ileocecal valve (0.0%) compared to late responders (100% and 28.6%, respectively; both p < 0.05). Early responders had a lower mean percentage of colon remaining compared to late responders (24.6% vs. 57.1%, respectively; p = 0.016).CONCLUSIONSTime to response to teduglutide depends on bowel anatomy and SBS-IF aetiology. IBD, presence of a stoma, and absence of ileocecal valve were associated with earlier response to teduglutide. These findings may enhance management of patients with SBS-IF; however, due to sample size limitations, additional studies are needed to confirm these findings.

01 Jun 2021·European Journal of EndocrinologyQ1 · MEDICINE

Characterization of patients with adrenal insufficiency and frequent adrenal crises

Q1 · MEDICINE

Article

Author: Marelli, Claudio ; Quinkler, Marcus ; Murray, Robert D ; Isidori, Andrea M ; Ekman, Bertil ; Zhang, Pinggao ; Petermann, Robert ; _, _

ObjectiveThis study aimed to characterize the clinical and biochemical features of patients with primary (PAI) and secondary (SAI) adrenal insufficiency who developed adrenal crises (ACs) and estimate the incidence of ACs in these patients.DesignRetrospective case-control analysis of the European Adrenal Insufficiency Registry (EU-AIR; NCT01661387).MethodsTwo thousand six hundred and ninety-four patients with AI (1054 PAI; 1640 SAI) enrolled in EU-AIR. Patients who developed ≥ 1 AC were matchd 1:3 with patients without ACs for age, sex and AI type. Data were collected at baseline and follow-up (mean ± s.d.: PAI 3.2 ± 1.7 years; SAI 2.9 ± 1.7 years).ResultsOne hundred and forty-eight out of 2694 patients (5.5%; n = 84 PAI; n = 64 SAI) had an AC during the study: 6.53 (PAI) and 3.17 (SAI) ACs/100 patient-years. Of patients who experienced an AC, 16% (PAI) and 9.4% (SAI) experienced ≥ 1 AC/year. The incidence of adverse events, infectious intercurrent illnesses and infectious serious adverse events were higher in patients with ACs than without ACs. No differences were observed in BMI, HbA1c, blood pressure and frequencies of diabetes mellitus or hypertension between subgroups (PAI and SAI, with and without ACs). At baseline, PAI patients with AC had higher serum potassium (4.3 ± 0.5 vs 4.2 ± 0.4 mmol/L; P = 0.03) and lower sodium (138.5 ± 3.4 vs 139.7 ± 2.9 mmol/L; P = 0.004) than patients without AC. At last observation, SAI patients with AC had higher hydrocortisone doses than patients without AC (11.9 ± 5.1 vs 10.1 ± 2.9 mg/m2; P < 0.001).ConclusionsThese results demonstrate that concomitant diseases and cardiovascular risk factors do not feature in the risk profile of AC; however, patients with AC had a higher incidence of infectious events.

01 Mar 2021·Cardiovascular ToxicologyQ4 · MEDICINE

Cardiovascular Safety of Anagrelide Hydrochloride versus Hydroxyurea in Essential Thrombocythaemia

Q4 · MEDICINE

Article

Author: Achenbach, Heinrich ; Gotic, Mirjana ; Kvasnicka, Hans Michael ; Wu, Jingyang ; Egyed, Miklos ; Gercheva, Liana ; Warzocha, Krzysztof

AbstractEssential thrombocythaemia (ET) is a rare myeloproliferative neoplasm. This multicentre, Phase 3b, randomised, open-label, non-inferiority study investigated the cardiac safety, efficacy and tolerability of first-line treatment with anagrelide or hydroxyurea in high-risk ET patients for up to 3 years. Eligible patients aged ≥ 18 years with a diagnosis of high-risk ET confirmed by bone marrow biopsy within 6 months of randomisation received anagrelide (n = 75) or hydroxyurea (n = 74), administered twice daily. Treatment dose for either compound was titrated to the lowest dose needed to achieve a response. Planned primary outcome measures were change in left ventricular ejection fraction from baseline over time and platelet count at Month 6. Planned secondary outcome measures were platelet count change from baseline at Months 3 and 36; percentage of patients with complete or partial response; time to complete or partial response; number of patients with thrombohaemorrhagic events; and changes in white blood cell count or red blood cell count over time. Neither treatment altered cardiac function. There were no significant differences in adverse events between treatment groups, and no reports of malignant transformation. The incidence of disease-related thrombotic or haemorrhagic events was numerically higher in anagrelide-treated patients. Both treatments controlled platelet counts at 6 months, with the majority of patients experiencing complete or partial responses. In conclusion, these results suggest that long-term treatment with anagrelide is not associated with adverse effects on cardiac function. This is one of the few studies using left ventricular ejection fraction assessment and central biopsy reading to confirm the diagnosis of ET.Trial registration number: Clinicaltrials.gov NCT00202644

100 Deals associated with Shire International GmbH

100 Translational Medicine associated with Shire International GmbH

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Drug(Targets)	Indications	Global Highest Phase

Lanadelumab ( Kallikreins )	Familial cold urticaria More	Phase 2

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