Cystic FibrosisORK-AJANCT03659214
Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults
An observational study of Cystic Fibrosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 12 to 20 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Questionnaires
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 19 participants
- Where
- 1 siteFrance
- Study dates
- 1 Dec 2017to 19 Jan 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: CF Center patients
Who may be able to join
- 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
Who cannot join
- Transplanted patients
- Patients younger than 12 years
- Patients older than 20 years
Trial locations and contacts
1 site in 1 country.
France1 site
Department of Pneumology -CRCM - Necker - Enfants maladies Hospital (AP-HP)
Paris, 75015
About this study
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.
Read the detailed 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.
- Conditions
- Cystic Fibrosis
- Being tested
- Questionnaires
- Keywords
- Cystic Fibrosis, Orkambi, Adherence, Adolescent, Anxiety, Depression, Psychology
- Medical subject headings
- Cystic Fibrosis, Anxiety Disorders, Depression
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnairesBehavioural
Answering 4 questionnaires : anxiety, depression, quality of life and adherence
| Study group | Role | What they receive |
|---|---|---|
| Treated group | Patients with proven CF (sweat-test > 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI) Receives: 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 Receives: Behavioral: Questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7)
to assess the impact on Anxiety
Measured: 24 months
Secondary outcomes (3)
Score of Patient Health Questionnaire-9 (PHQ-9 )
To assess the impact on Depression
Measured: 24 months
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)
Measured: 24 months
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
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2018
- Study start1 Dec 2017
- Primary completion19 Jan 2022
- Study completion19 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- VERTEX Pharmaceutical, URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Maya KIRSZENBAUM, Psychologist, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03659214
- Sponsor's protocol ID
- APHP180003
- Other IDs
- 2018-A01561-54
- Acronym
- ORK-AJA
- Status last verified
- Oct 2025
- First submitted
- 3 Sep 2018
- Last updated
- 20 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 10911813Bucks 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.
PubMed 19196850Zindani 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.
PubMed 16387243Butner 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.
PubMed 19413435Arias 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.
PubMed 18304896Marciel 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.
PubMed 20054860Blackwell 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.
PubMed 23721854Girerd 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.
PubMed 11575214Spitzer 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.
PubMed 16717171Kroenke 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.
PubMed 11556941
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults
- ClinicalTrials.gov ID
- NCT03659214
- Acronym / sponsor ID
- ORK-AJA / APHP180003
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Orkambi, Adherence, Adolescent, Anxiety, Depression, Psychology
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- VERTEX Pharmaceutical, URC-CIC Paris Descartes Necker Cochin
- Study start
- 1 Dec 2017 (actual)
- Primary completion
- 19 Jan 2022 (actual)
- Study completion
- 19 Jan 2022 (actual)
- First posted
- 6 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treated group | – | Behavioral: QuestionnairesPatients with proven CF (sweat-test > 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI) |
| Control group | – | Behavioral: QuestionnairesPatients 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 |
Who may qualify
- Ages
- 12 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- CF Center patients
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (3 listed)
- Transplanted patients
- Patients younger than 12 years
- Patients older than 20 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QuestionnairesAnswering 4 questionnaires : anxiety, depression, quality of life and adherence | Behavioural | Control group, Treated group |
Primary outcome
Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7)
Time frame: 24 months
to assess the impact on Anxiety
3 secondary outcomes
Score of Patient Health Questionnaire-9 (PHQ-9 )
Time frame: 24 months
Scores of Cystic Fibrosis Questionnaire 14+ (CFQ 14+)
Time frame: 24 months
GIRERD Scale
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2025.
- Background: PubMed 10911813Pendleton DA, David TJ. The compliance conundrum in cystic fibrosis. J R Soc Med. 2000;93 Suppl 38(Suppl 38):9-13. No abstract available.
- Background: PubMed 19196850Bucks 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.
- Background: PubMed 16387243Zindani 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.
- Background: PubMed 19413435Butner 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.
- Background: PubMed 18304896Arias 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.
- Background: PubMed 20054860Marciel 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.
- Background: PubMed 23721854Blackwell 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.
- Background: PubMed 11575214Girerd 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.
- Background: PubMed 16717171Spitzer 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.
- Background: PubMed 11556941Kroenke 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maya KIRSZENBAUM, Psychologist principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7) (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03659214, which also lists 10 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2025.