Development and Validation of a Self-assessment System Based on a Mobile App to Manage Adult Growth Hormone Deficiency

NCT03525587 | Completed

• 1 other identifier: 346/17
• Study Type: interventional
• Target: 83
• Locations: 1 country
• Sites: 1

Brief Summary

Adult Growth Hormone Deficiency (AGHD) is a recognized clinical entity but several barriers concerning patient-clinician communication, inadequate patients' awareness of the disease, low perceived benefit of replacement therapy and poor compliance still remains. The overall goal of the study is to improve AGHD management through a Smartphone app (MAGHD App: Manage Adult Growth Hormone Deficiency) integrated with a software framework able to merge patients daily data on physical activity, quality of life (QoL), and well-being with clinical data collected in institutional databases. The target population consists of 100 patients with a previous diagnosis of AGHD, whether in treatment with growth hormone or not. In a prospective 24 months study, MAGHD App will be developed and connected to MAGHD Framework. This system will allow to integrate: 1) Physical Activity Data collected by wearable devices, 2) Patient Related Outcomes Data, periodically inserted by the patients through MAGHD App in response to questions extrapolated from validated questionnaires, 3) HCP Data registered in clinical databases and including medical history, biochemical and radiological examination. Data will converge in MAGHD Framework where they will be analyzed and used to create reports visible to patients (in MAGHD App) and clinicians (by a monitoring dashboard). The results are expected to positively influence AGHD management by involving patients in care process and giving clinicians a useful tool for clinical practice.

Conditions

Growth Hormone Deficiency

Keywords

Quality Improvement; Quality of Life; Smartphone APP

Outcome Measures

Primary Outcomes (1)

• Changes of quality of life (QoL) from baseline

  Comparison of changes of QoL score from baseline for each patient within the three different groups. QoL score will be evaluated and compared before and after the use of the integrated system MAGHD App/MAGHD Framework.

  24 months

Secondary Outcomes (13)

• Changes of well-being from baseline

  24 months

• Changes of physical activity from baseline

  24 months

• Changes of sleep quality from baseline

  24 months

• Changes of sexual function from baseline

  24 months

• Changes of insulin-like growth factor 1 (IGF-1) levels from baseline

  24 months

Study Arms (3)

Ongoing r-hGH therapy

ACTIVE COMPARATOR

Patients on long-term r-hGH therapy. Intervention: Use of "MAGHD App/MAGHD Framework"

Other: Use of "MAGHD App/MAGHD Framework"

Previous r-hGH therapy

ACTIVE COMPARATOR

Patients previously treated with r-hGH, who had stopped the treatment for any reason (age, concomitant adverse reactions, contraindications or personal will). Intervention: Use of "MAGHD App/MAGHD Framework"

Other: Use of "MAGHD App/MAGHD Framework"

Never treated

ACTIVE COMPARATOR

Patients never treated for any reason (according to age, contraindications or lack of patient's consent). Intervention: Use of "MAGHD App/MAGHD Framework"

Other: Use of "MAGHD App/MAGHD Framework"

Interventions

Use of "MAGHD App/MAGHD Framework" OTHER

Patients daily related outcomes will be recorded thanks to the development, implementation and validation of a Smartphone app (MAGHD App) integrated with a software framework able to merge patient's daily activities data on well-being status, QoL, physical activities and sexual function with clinical data collected in their record chart (extrapolated from already available Institutional Databases).

Never treated; Ongoing r-hGH therapy; Previous r-hGH therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Having a documented diagnosis of AGHD, according to the latest Endocrine Society clinical practice guidelines
• Being at least 18 years old
• Having a good Italian understanding
• Owing smartphones with either an Android or iPhone operating system
• Patients with other pituitary deficits will be enrolled only if the other hormonal deficiencies are well controlled by replacement treatments since six months in order to avoid the overlap of effects due to different therapies.

You may not qualify if:

• \) Patients who do not plan to continue follow-up at the Endocrinology Unit of Modena 2) Patients with a diagnosis of biochemical GHD outside the appropriate clinical context of pituitary disease 3) Patients with major psychiatric diseases, chronic highly invalidating diseases (these patients will be considered not eligible for being enrolled in the study due to the impact of the underlying disease on well-being and daily activities).

Sponsors & Collaborators

• Azienda Ospedaliero-Universitaria di Modena: lead
• Pfizer: collaborator

Study Sites (1)

Azienda Ospedaliero - Universitaria di Modena

Modena, Modena, 41124, Italy

Location

Related Publications (19)

• Ho KK; 2007 GH Deficiency Consensus Workshop Participants. Consensus guidelines for the diagnosis and treatment of adults with GH deficiency II: a statement of the GH Research Society in association with the European Society for Pediatric Endocrinology, Lawson Wilkins Society, European Society of Endocrinology, Japan Endocrine Society, and Endocrine Society of Australia. Eur J Endocrinol. 2007 Dec;157(6):695-700. doi: 10.1530/EJE-07-0631.

  PMID: 18057375 BACKGROUND

• Molitch ME, Clemmons DR, Malozowski S, Merriam GR, Vance ML; Endocrine Society. Evaluation and treatment of adult growth hormone deficiency: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab. 2011 Jun;96(6):1587-609. doi: 10.1210/jc.2011-0179.

  PMID: 21602453 BACKGROUND

• Woodhouse LJ, Mukherjee A, Shalet SM, Ezzat S. The influence of growth hormone status on physical impairments, functional limitations, and health-related quality of life in adults. Endocr Rev. 2006 May;27(3):287-317. doi: 10.1210/er.2004-0022. Epub 2006 Mar 16.

  PMID: 16543384 BACKGROUND

• van Bunderen CC, van Varsseveld NC, Erfurth EM, Ket JC, Drent ML. Efficacy and safety of growth hormone treatment in adults with growth hormone deficiency: a systematic review of studies on morbidity. Clin Endocrinol (Oxf). 2014 Jul;81(1):1-14. doi: 10.1111/cen.12477. Epub 2014 May 12.

  PMID: 24750271 BACKGROUND

• Hoybye C, Christiansen JS. Growth hormone replacement in adults - current standards and new perspectives. Best Pract Res Clin Endocrinol Metab. 2015 Jan;29(1):115-23. doi: 10.1016/j.beem.2014.09.006. Epub 2014 Oct 2.

  PMID: 25617177 BACKGROUND

• Newman CB, Carmichael JD, Kleinberg DL. Effects of low dose versus high dose human growth hormone on body composition and lipids in adults with GH deficiency: a meta-analysis of placebo-controlled randomized trials. Pituitary. 2015 Jun;18(3):297-305. doi: 10.1007/s11102-014-0571-z.

  PMID: 24810900 BACKGROUND

• Bengtsson BA, Abs R, Bennmarker H, Monson JP, Feldt-Rasmussen U, Hernberg-Stahl E, Westberg B, Wilton P, Wuster C. The effects of treatment and the individual responsiveness to growth hormone (GH) replacement therapy in 665 GH-deficient adults. KIMS Study Group and the KIMS International Board. J Clin Endocrinol Metab. 1999 Nov;84(11):3929-35. doi: 10.1210/jcem.84.11.6088.

  PMID: 10566630 BACKGROUND

• Attanasio AF, Bates PC, Ho KK, Webb SM, Ross RJ, Strasburger CJ, Bouillon R, Crowe B, Selander K, Valle D, Lamberts SW; Hypoptiuitary Control and Complications Study International Advisory Board. Human growth hormone replacement in adult hypopituitary patients: long-term effects on body composition and lipid status--3-year results from the HypoCCS Database. J Clin Endocrinol Metab. 2002 Apr;87(4):1600-6. doi: 10.1210/jcem.87.4.8429.

  PMID: 11932289 BACKGROUND

• Alexopoulou O, Abs R, Maiter D. Treatment of adult growth hormone deficiency: who, why and how? A review. Acta Clin Belg. 2010 Jan-Feb;65(1):13-22. doi: 10.1179/acb.2010.002.

  PMID: 20373593 BACKGROUND

• Lazure P, Bartel RC, Biller BM, Molitch ME, Rosenthal SM, Ross JL, Bernsten BD, Hayes SM. Contextualized analysis of a needs assessment using the Theoretical Domains Framework: a case example in endocrinology. BMC Health Serv Res. 2014 Jul 24;14:319. doi: 10.1186/1472-6963-14-319.

  PMID: 25060235 BACKGROUND

• Buijink AW, Visser BJ, Marshall L. Medical apps for smartphones: lack of evidence undermines quality and safety. Evid Based Med. 2013 Jun;18(3):90-2. doi: 10.1136/eb-2012-100885. Epub 2012 Aug 25.

  PMID: 22923708 BACKGROUND

• Cui M, Wu X, Mao J, Wang X, Nie M. T2DM Self-Management via Smartphone Applications: A Systematic Review and Meta-Analysis. PLoS One. 2016 Nov 18;11(11):e0166718. doi: 10.1371/journal.pone.0166718. eCollection 2016.

  PMID: 27861583 BACKGROUND

• Chin SO, Keum C, Woo J, Park J, Choi HJ, Woo JT, Rhee SY. Successful weight reduction and maintenance by using a smartphone application in those with overweight and obesity. Sci Rep. 2016 Nov 7;6:34563. doi: 10.1038/srep34563.

  PMID: 27819345 BACKGROUND

• Ryan EA, Holland J, Stroulia E, Bazelli B, Babwik SA, Li H, Senior P, Greiner R. Improved A1C Levels in Type 1 Diabetes with Smartphone App Use. Can J Diabetes. 2017 Feb;41(1):33-40. doi: 10.1016/j.jcjd.2016.06.001. Epub 2016 Aug 25.

  PMID: 27570203 BACKGROUND

• Tennant R, Hiller L, Fishwick R, Platt S, Joseph S, Weich S, Parkinson J, Secker J, Stewart-Brown S. The Warwick-Edinburgh Mental Well-being Scale (WEMWBS): development and UK validation. Health Qual Life Outcomes. 2007 Nov 27;5:63. doi: 10.1186/1477-7525-5-63.

  PMID: 18042300 BACKGROUND

• Rosen RC, Riley A, Wagner G, Osterloh IH, Kirkpatrick J, Mishra A. The international index of erectile function (IIEF): a multidimensional scale for assessment of erectile dysfunction. Urology. 1997 Jun;49(6):822-30. doi: 10.1016/s0090-4295(97)00238-0.

  PMID: 9187685 BACKGROUND

• Rosen R, Brown C, Heiman J, Leiblum S, Meston C, Shabsigh R, Ferguson D, D'Agostino R Jr. The Female Sexual Function Index (FSFI): a multidimensional self-report instrument for the assessment of female sexual function. J Sex Marital Ther. 2000 Apr-Jun;26(2):191-208. doi: 10.1080/009262300278597.

  PMID: 10782451 BACKGROUND

• Craig CL, Marshall AL, Sjostrom M, Bauman AE, Booth ML, Ainsworth BE, Pratt M, Ekelund U, Yngve A, Sallis JF, Oja P. International physical activity questionnaire: 12-country reliability and validity. Med Sci Sports Exerc. 2003 Aug;35(8):1381-95. doi: 10.1249/01.MSS.0000078924.61453.FB.

  PMID: 12900694 BACKGROUND

• Curcio G, Tempesta D, Scarlata S, Marzano C, Moroni F, Rossini PM, Ferrara M, De Gennaro L. Validity of the Italian version of the Pittsburgh Sleep Quality Index (PSQI). Neurol Sci. 2013 Apr;34(4):511-9. doi: 10.1007/s10072-012-1085-y. Epub 2012 Apr 13.

  PMID: 22526760 BACKGROUND

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Study Officials

• Vincenzo Rochira, Professor

  Azienda Ospedaliero-Universitaria di Modena

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD - Associate Professor of Endocrinology

Model Details: Patients with a previous documented AGHD fitting the inclusion/exclusion criteria will be enrolled and grouped as follows: * Group 1: patients on long-term r-hGH therapy; * Group 2: patients previously treated with r-hGH, who had stopped the treatment for any reason (age, concomitant adverse reactions, contraindications or personal will); * Group 3: patients never treated for any reason (according to age, contraindications or lack of patient's consent). The whole cohort of AGHD patients (n=100) will undergo 2 consecutive phases: in the first phase (12 months) patients will be evaluated as usual every six months according to our standard clinical practice without using the App and the wearable device (baseline visit, visit 1 and visit 2), while in the second phase (following 12 months) they will be evaluated every six months during the use of the App (visit 3 and visit 4).

Study Record Dates

• First Submitted: April 17, 2018
• First Posted: May 15, 2018
• Study Start: January 17, 2018
• Primary Completion: June 30, 2020
• Study Completion: December 31, 2024
• Last Updated: March 14, 2025

Record last verified: 2025-03

Locations

IT (1)