Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults

Cystic Fibrosis Clinical Trial

— ORK-AJA  

Official title:

Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03659214
• Other study ID #: APHP180003
• Secondary ID: 2018-A01561-54
• Status: Completed
• Start date: December 1, 2017
• Est. completion date: January 19, 2022

Study information

• Verified date: October 2022
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The aim of this project is to evaluate the psychological reshuffle induced by ORKAMBI. The particular focus of this study is the consequence of its introduction on anxiety, depression, quality of life and adherence to all cystic fibrosis (CF) treatment. To answer this question investigators will monitor the psychological function of CF adolescents and young adults treated with ORKAMBI and compare them to CF adolescents and young adults not treated with ORKAMBI.

Description:

Introduction of KALYDECO then ORKAMBI, as a personalized medicine in the therapeutic strategy of Cystic Fibrosis (CF) may deeply modify the prognosis of patients with CF. These approaches realize the dream of short-circuiting the molecular mechanism of the disease and therefore opens the perspective of an extended and functional survival. It suddenly offers the possibility of a completely new future to the patient and his family, and therefore must deeply impact on the psychological function of all involved. Moreover, those new therapies may also modify the adherence to CF basic treatment, as it brings a new feeling of good health and the hope of a longer life. For that reason there is a need to investigate modifications of the psychological state of mind induced by the treatment with ORKAMBI in CF adolescents and young adults.

This project aims to understand the psychological impact and to study the consequences of the introduction of ORKAMBI on adherence to CF treatment, anxiety, depression and quality of life.

To answer this question, investigators will evaluate the psychological function of adolescents and young adults, aged 12 to 20 years, with CF treated with ORKAMBI, and compare it to other adolescents and young CF adults not treated with ORKAMBI.

This is a cohort prospective study, with a 1 year follow-up.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: January 19, 2022
• Est. primary completion date: January 19, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 20 Years

Eligibility

Inclusion Criteria:

• Treated group: Patients with proven CF (sweat-test > 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI

• Control group: Patients not carrying 2 DF508 CF causing mutations, not treated with ORKAMBI, and not carrying the G551D, G178R, S549N, S549R, G551S, G1244E,S1251N, S1255P or G1349D mutation or treated with Kalydeco

Exclusion Criteria:

• Transplanted patients

• Patients younger than 12 years

• Patients older than 20 years

Related Conditions & MeSH terms

• Cystic Fibrosis
• Depression
• Fibrosis

Intervention

Behavioral:
• Questionnaires
Answering 4 questionnaires : anxiety, depression, quality of life and adherence

Locations

Country	Name	City	State
France	Department of Pneumology -CRCM - Necker - Enfants maladies Hospital (AP-HP)	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	VERTEX Pharmaceutical

Country where clinical trial is conducted

France, 

References & Publications (10)

Arias Llorente RP, Bousono Garcia C, Diaz Martin JJ. Treatment compliance in children and adults with cystic fibrosis. J Cyst Fibros. 2008 Sep;7(5):359-67. doi: 10.1016/j.jcf.2008.01.003. Epub 2008 Mar 4. — View Citation

Blackwell LS, Marciel KK, Quittner AL. Utilization of patient-reported outcomes as a step towards collaborative medicine. Paediatr Respir Rev. 2013 Sep;14(3):146-51. doi: 10.1016/j.prrv.2013.04.003. Epub 2013 May 27. — View Citation

Bucks RS, Hawkins K, Skinner TC, Horn S, Seddon P, Horne R. Adherence to treatment in adolescents with cystic fibrosis: the role of illness perceptions and treatment beliefs. J Pediatr Psychol. 2009 Sep;34(8):893-902. doi: 10.1093/jpepsy/jsn135. Epub 2009 Feb 5. — View Citation

Butner J, Berg CA, Osborn P, Butler JM, Godri C, Fortenberry KT, Barach I, Le H, Wiebe DJ. Parent-adolescent discrepancies in adolescents' competence and the balance of adolescent autonomy and adolescent and parent well-being in the context of Type 1 diabetes. Dev Psychol. 2009 May;45(3):835-49. doi: 10.1037/a0015363. — View Citation

Girerd X, Radauceanu A, Achard JM, Fourcade J, Tournier B, Brillet G, Silhol F, Hanon O. [Evaluation of patient compliance among hypertensive patients treated by specialists]. Arch Mal Coeur Vaiss. 2001 Aug;94(8):839-42. French. — View Citation

Kroenke K, Spitzer RL, Williams JB. The PHQ-9: validity of a brief depression severity measure. J Gen Intern Med. 2001 Sep;16(9):606-13. doi: 10.1046/j.1525-1497.2001.016009606.x. — View Citation

Marciel KK, Saiman L, Quittell LM, Dawkins K, Quittner AL. Cell phone intervention to improve adherence: cystic fibrosis care team, patient, and parent perspectives. Pediatr Pulmonol. 2010 Feb;45(2):157-64. doi: 10.1002/ppul.21164. — View Citation

Pendleton DA, David TJ. The compliance conundrum in cystic fibrosis. J R Soc Med. 2000;93 Suppl 38(Suppl 38):9-13. No abstract available. — View Citation

Spitzer RL, Kroenke K, Williams JB, Lowe B. A brief measure for assessing generalized anxiety disorder: the GAD-7. Arch Intern Med. 2006 May 22;166(10):1092-7. doi: 10.1001/archinte.166.10.1092. — View Citation

Zindani GN, Streetman DD, Streetman DS, Nasr SZ. Adherence to treatment in children and adolescent patients with cystic fibrosis. J Adolesc Health. 2006 Jan;38(1):13-7. doi: 10.1016/j.jadohealth.2004.09.013. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7)	to assess the impact on Anxiety	24 months
Secondary	Score of Patient Health Questionnaire-9 (PHQ-9 )	To assess the impact on Depression	24 months
Secondary	Scores of Cystic Fibrosis Questionnaire 14+ (CFQ 14+)	Disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms (Quality of Life)	24 months
Secondary	GIRERD Scale	The 6 items are assessed using a YES/NO answer .The scale was validated in patients with chronic disease to assess medication adherence	24 months