Clinical Research Directory

ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly

The primary objective of this study is to evaluate the efficacy of 15-week treatment with ALXN2420 versus placebo for decreasing insulin-like growth factor IGF-1 levels, when administered in combination with somatostatin analog (SSA) therapy to adult participants with acromegaly.

A Study to Evaluate the Long-Term Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (ACROBAT Advance)

A phase 2, open label, long-term extension study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with acromegaly.

A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (PATHFNDR-2)

A randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with non-pharmacologically treated acromegaly.

A Study to Assess the Efficacy and Safety of Debio 4126 in Participants With Acromegaly Previously Treated With Somatostatin Analogs

The primary purpose of this study is to assess the effect of Debio 4126 in the maintenance of the levels of insulin-like growth factor 1 (IGF-1) ≤1x upper limit of normal (ULN) in the double-blind period (Period 1) in comparison to placebo at week 36.

A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly

A randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (also known as CRN00808; an orally administered nonpeptide somatostatin agonist) in subjects with acromegaly previously treated with somatostatin receptor ligand (SRL) based treatment regimens.

Study of MAR002 in Healthy Men

Evaluate the safety and tolerability of subcutaneous (SC) administration of MAR002 in healthy men

Effects of Pasireotide Lar Therapy on Bone Metabolism

Acromegaly, a chronic condition characterized by growth hormone (GH) and, in turn, insulin-like growth factor-1 (IGF-I) excess, is burdened by a series of systemic and metabolic comorbidities that strongly impair quality of life (QoL) and life expectancy. Amongst them, a specific acromegalic osteopathy has been discovered, characterized by fragility fractures associated with high bone turnover, which need to be early detected, according to most recent guidelines, since they are very frequent and related to chronic pain and reduced QoL. Morphometric vertebral fractures (VFs) are an emerging landmark of skeletal fragility in general population as well as in clinical trials, and are highly prevalent in acromegaly, being reported to affect from 30 up to 60% of patients and represent an early and common event in disease history. Until now, same groups of patients with higher risk of vertebral fractures were identified, such as those carrying incident vertebral fractures, or affected by biochemical active acromegaly, concomitant hypogonadism, or diabetes mellitus. The main aim in the management of patients with acromegaly is to normalize IGF-I levels and restore acromegaly related symptoms. To aim this treatment objective, the first line of treatment of acromegaly, when feasible, is neurosurgery. In cases where surgical intervention fails to achieve biochemical control, medical therapy is recommended, with the objective of reaching normal levels of IGF-1 and GH age-corrected. Octreotide LAR and Lanreotide are the first-line medical therapy. In patients who have not achieved adequate control with standard doses of octreotide LAR and Lanreotide, increasing the dose and/or frequency of administration can lead to improved biochemical control. In patients who are unable to achieve control even with this approach, a switch to Pasireotide LAR may be considered. In instances where patients fail to achieve biochemical control with maximal doses of SRL, or in the presence of contraindications, the use of Pegvisomant as a second-line therapy may be considered. In addition, a combination of Pegvisomant and SRL represents a potential avenue for treating patients. Prevention of VFs in acromegaly remains an open issue. It has been shown that use of GH/IGF-I lowering treatments with first-generation SSA and Pegvisomant, may reduce the risk of VFs, while improving disease control. Moreover, in a retrospective and observational multicenter study, it was recently proved that patients treated with second generation SRLs (Pasireotide-LAR) developed less frequently VFs then patients treated with Pegvisomant.

Effects of Therapies in the Acromegaly Disease: Acral Morpho-functional Study

Acromegaly is a rare chronic disease due to excessive secretion of growth hormone (GH) and insulin-like growth factor-I (IGF-I), caused in over 98% of cases by GH-secreting pituitary adenoma. Prolonged exposure to GH/IGF-I excess is the cause of increased mortality and morbidity in these patients. Arthropathy occurs in about 75% of acromegalic patients. Any joint may be affected, with the development of osteoarthritis, arthralgia, and an increase in fracture risk. The aims of the present project are to evaluate the dimensions of hands and feet with the 3D scanner method and to perform a quantitative analysis of movement through Gait Analysis technique in de novo patients with acromegaly (group # 1) and in patients with different disease status (group #2).

Korean Regulatory Post Marketing Surveillance for Somavert

Non-interventional observational study, to identify safety and effectiveness of Somavert during the post-marketing period based on the Korean RMP as required by the regulations of Ministry of Food and Drug Safety (MFDS)

Genetics of Endocrine Tumours - Familial Isolated Pituitary Adenoma - FIPA

The research is aimed at identifying new predisposition genes for endocrine tumours. Our focus initially is on pituitary adenomas including growth hormone-secreting tumors (somatotrophinomas) and prolactin secreting tumours (prolactinomas), but we wish to extend work to other pituitary tumour cases/families. The recruitment process will be as follows. 1. We will recruit patients from our own Endocrine outpatient clinics and inpatient wards. In addition we will ask colleagues in other Endocrinology Departments (or other specialties such as Clinical Genetics,Pathology, General Medicine ) to identify potentially suitable patients with endocrine \& pituitary tumours from their records. We shall focus on patients with good evidence of inheritance of their condition: relatively early onset; or multiple lesions; or other affected family members. Conditions where the predisposing genes have been identified (principally MEN) will be excluded from study. Patients directly contacting us can also enter the study. 2. The Consultant looking after the patient will contact the patient to initially inform him/her of the study. 3. We will then contact the patient (generally by telephone) to discuss the study and what it would entail in terms of information and samples. 4. Subject to agreement in (3), patient will receive 'Information Sheet for patients with pituitary tumour' and 'Consent Form' and will have blood sampling in Consultant's clinic. 5. We will contact additional family members (if appropriate) after an initial approach by the family member already recruited to the study. The additional family members may have developed tumours similar to those of the proband, or may be unaffected individuals who provide useful information for gene identification purposes (for example, spouses may greatly aid the power of gene mapping by linkage. They will receive the "Information Sheet for family members". analysis). 8\. Archival tissue will be obtained from HTA licensed tissue banks. This is an established bank whose licence is primarily for diagnosis but can be used for research. 9. We will undertake laboratory work, such as genetic linkage analysis, candidate gene mutation screening and studies of loss of heterozygosity in tumours, to identify the genes predisposing to the condition, such as the AIP gene. In addition we would like to screen other genes related to the chaperon AIP molecule, such as AhR, and other genes currently identified (PDE4A5, survivin and Tom20 protein) or may not been identified. Blood samples for DNA and RNA will coded with unique ID numbers. Pituitary and other endocrine tumour samples will be collected at surgery and kept in liquid nitrogen or -80 C. They will be coded with unique ID numbers. Candidate gene sequencing will be performed in the Barts and the London Medical School Genome Centre. RNA expression studies from blood or adenoma tissue samples will be performed by RT-PCR. Protein expression studies will be performed by Western blotting or immunohistochemistry. The first gene we wish to study causes familial acromegaly, a disease resulting from a pituitary adenoma secreting growth hormone. To establish if the candidate gene is also causing possibly sporadic (not familial) cases of the disease, samples (blood and tissue) will be collected from patients with sporadic disease and will be analysed as above.

The RApid Switch From 1st Generation Somatostatin Analogues to PaSireOtiDe In Acromegaly

This is an observational, retrospective, national multicenter study aimed to evaluate the impact and efficacy of Time To Switch (TTS) from first-line to second-line medical therapy in Acromegaly.

Cellular, Molecular and Clinical Determinants of Bone Strength in in Vivo and Human Models of GH Excess.

Prospective observational clinical, molecular, translational study aimed at identifying the main determinants and predictive factors of fragility fracture risck in acromegaly patients

Neuroendocrine Mechanisms in Adiposity: An Integrated Approach to the Characterization of Potential Pharmacological Novel Targets Based on Experimental and Clinical Models

The goal of this observational study is to evaluate, retrospectively and prospectively, the effect of different hormonal and neuropeptide dysfunctions on the body composition of patients suffering from hypothalamic-pituitary pathologies, and to evaluate the potential beneficial effect of surgical and medical treatments with agonists and antagonists of hypothalamic neuropeptides, currently available, on the development and treatment of adiposity and negative cross-talk between adiposity and muscle/bone tissue

Diet in the Management of Acromegaly

Impact of low carbohydrate and low gluten diet on acromegaly progression, symptoms, complications, and treatment outcomes.

The Longitudinal Approach to Acromegaly: A Pattern of Treatment and Comparative Effectiveness Research

This study is a prospective, noninteractive, observational, and longitudinal study aimed at assessing the treatment pattern and clinical outcome of acromegaly in China.

Developing a Simple Recognition System of Acromegaly

We will compare the features of 3D stereophotography of acromegaly patients with that of healthy people. We hope to develop a computerized model to help screening acromegaly patients for early detection and treatment.