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

NCT03659214 | Completed

• 2 other identifiers: APHP1800032018-A01561-54
• Study Type: observational
• Target: 19
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; Orkambi; Adherence; Adolescent; Anxiety; Depression; Psychology

Outcome Measures

Primary Outcomes (1)

• Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7)

  to assess the impact on Anxiety

  24 months

Secondary Outcomes (3)

• Score of Patient Health Questionnaire-9 (PHQ-9 )

  24 months

• Scores of Cystic Fibrosis Questionnaire 14+ (CFQ 14+)

  24 months

• GIRERD Scale

  24 months

Study Arms (2)

Treated group

Patients with proven CF (sweat-test \> 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI)

Behavioral: Questionnaires

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

Behavioral: Questionnaires

Interventions

Questionnaires BEHAVIORAL

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

Control group; Treated group

Eligibility Criteria

• Age: 12 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

CF Center patients

You may qualify if:

• 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

You may not qualify if:

• Transplanted patients
• Patients younger than 12 years
• Patients older than 20 years

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• VERTEX Pharmaceutical: collaborator
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Department of Pneumology -CRCM - Necker - Enfants maladies Hospital (AP-HP)

Paris, 75015, France

Location

Related Publications (10)

• 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.

  PMID: 10911813 BACKGROUND

• 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.

  PMID: 19196850 BACKGROUND

• 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.

  PMID: 16387243 BACKGROUND

• 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.

  PMID: 19413435 BACKGROUND

• 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.

  PMID: 18304896 BACKGROUND

• 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.

  PMID: 20054860 BACKGROUND

• 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.

  PMID: 23721854 BACKGROUND

• 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.

  PMID: 11575214 BACKGROUND

• 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.

  PMID: 16717171 BACKGROUND

• 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.

  PMID: 11556941 BACKGROUND

MeSH Terms

Conditions

Cystic Fibrosis; Anxiety Disorders; Depression

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Mental Disorders; Behavioral Symptoms; Behavior

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Maya KIRSZENBAUM, Psychologist

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 3, 2018
• First Posted: September 6, 2018
• Study Start: December 1, 2017
• Primary Completion: January 19, 2022
• Study Completion: January 19, 2022
• Last Updated: November 20, 2025

Record last verified: 2025-10

Locations

FR (1)