Kallmann SyndromeNCT01914172
Health Needs of Patients With Kallmann Syndrome
An observational study of Kallmann Syndrome and Congenital Hypogonadotropic Hypogonadism, run by Centre Hospitalier Universitaire Vaudois at 1 site in Switzerland.
Trial summary
Kallmann syndrome (KS), also known as congenital hypogonadotropic hypogonadism (CHH), is a rare endocrine disorder that is characterized by failure to undergo puberty combined with infertility. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Kallmann Syndrome, Congenital Hypogonadotropic Hypogonadism, Idiopathic Hypogonadotropic Hypogonadism
- What is being tested?
- online questionairres
- How many people
- 249 participants
- Where
- 1 siteSwitzerland
- Study dates
- Jul 2013to 27 Sep 2017
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults diagnosed with congenital hypogonadotropic hypogonadism/Kallmann syndrome will be recuited internationally.
Who may be able to join
- Diagnosed with congenital hypogonadotropic hypogonadism: i.e. Kallmann syndrome or idiopathic hypogonadotropic hypogonadism
- Primary language is English/capable of responding to a written questionnaire in English
- Consenting to participate in the study
Who cannot join
- other diagnosis of hypogonadism: i.e. hypergonadotropic hypogonadism (Klinefelter syndrome), adult onset hypogonadism, etc.
Trial locations and contacts
1 site in 1 country.
Switzerland1 site
Centre Hospitalier Universitaire Vaudois (CHUV)
Lausanne, Canton of Vaud, 1011
About this study
Official title: Factors Affecting Health Promoting Behavior in Rare Disease Patients: A Mixed Methods Study of Men With Congenital Hypogonadotropic Hypogonadism (CHH)
Kallmann syndrome (KS), also known as congenital hypogonadotropic hypogonadism (CHH), is a rare endocrine disorder that is characterized by failure to undergo puberty combined with infertility. KS/CHH patients face a number of psychosocial burdens related to delays in diagnosis, inadequate access to expert care, and lack of information about the condition. As such, there is some evidence to suggest that KS/CHH patients have unmet health needs. This study aims to identify the needs of patients and understand the issues that must be overcome to achieve improved health and quality of life.
Read the detailed description
This study aims to examine the experiences of patients diagnosed with Kallmann syndrome (KS)/congenital hypogonadotropic hypogonadism (CHH).
The study includes two parts:
- online survey (less than 30 minutes to complete)
- focus groups with KS/CHH patients
The aim of this project is to better understand what health needs are not presently being met for these patients and to identify targets for improving the care of patients diagnosed with KS/CHH
- Conditions
- Kallmann Syndrome, Congenital Hypogonadotropic Hypogonadism, Idiopathic Hypogonadotropic Hypogonadism
- Being tested
- online questionairres
- Keywords
- rare disease, community-based participatory research, health promotion, quantitative research, qualitative research, focus group
- Medical subject headings
- Kallmann Syndrome, Idiopathic Hypogonadotropic Hypogonadism, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
online questionairresOther
Also called patient focus groups
see group descriptions
| Study group | Role | What they receive |
|---|---|---|
| Online web-based questionnaire | Up to 200 patients with KS/CHH will be recruited to complete an online web-based questionnaire (less than 30 minutes to complete) Receives: Other: online questionairres | |
| Patient focus group | Focus groups (90-120 minutes in duration) with 6-12 patients. Up to 36 patients total | |
| Online web-based evaluation of patient education materials | Up to 100 patients with KS/CHH will be recruited to complete an online web-based questionnaire to evaluate patient education materials (less than 15 minutes to complete) Receives: Other: online questionairres |
What is being measured
The primary outcome is the main question the study is designed to answer.
illness perception
subjective self-assessment questionnaire of how KS/CHH impacts a patient's life
Measured: baseline
depression symptoms
self-rated questionnaire of depression symptoms
Measured: baseline
adherence to treatment
self-report of adherence to medication treatment and periods without treatment or healthcare
Measured: baseline
understandability and actionability of patient education materials
completion of Patient Education Materials Assessment Tool (PEMAT)
Measured: baeline
Secondary outcomes (2)
coping
Focus groups will be conducted to evaluate how KS/CHH impacts patients quality of life, the barriers to better health/quality of life, and how patients cope with living with KS/CHH
Measured: baseline
Interactions wth healthcare
A questionnaire reporting the quality and type of interactions with healthcare providers and the healthcare system
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2013 (estimated)
- Study startJul 2013
- Primary completionMar 2014
- Study completion27 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Kallmann SyndromeStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire VaudoisThe 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
- Cross-sectional
- Enrolment
- 249 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire Vaudois
- Sponsor type
- University, hospital or other
- Collaborators
- University of Lausanne
- Responsible party
- Principal investigator, Andrew Dwyer, Centre Hospitalier Universitaire Vaudois
- Investigators
- Andrew Dwyer, PhD, FNP-BC, Principal investigator, Centre Hositalier Universitaire Vaudois (CHUV)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01914172
- Sponsor's protocol ID
- 233/13
- Other IDs
- 233/13
- Status last verified
- Sep 2017
- First submitted
- 31 Jul 2013
- Last updated
- 29 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No: Patient characteristics will be reported in aggregate (no identifiers)
Publications
Dwyer AA, Quinton R, Morin D, Pitteloud N. Identifying the unmet health needs of patients with congenital hypogonadotropic hypogonadism using a web-based needs assessment: implications for online interventions and peer-to-peer support. Orphanet J Rare Dis. 2014 Jun 11;9:83. doi: 10.1186/1750-1172-9-83.
PubMed 24915927Dwyer AA, Quinton R, Pitteloud N, Morin D. Psychosexual development in men with congenital hypogonadotropic hypogonadism on long-term treatment: a mixed methods study. Sex Med. 2015 Mar;3(1):32-41. doi: 10.1002/sm2.50.
PubMed 25844173Dwyer AA, Tiemensma J, Quinton R, Pitteloud N, Morin D. Adherence to treatment in men with hypogonadotrophic hypogonadism. Clin Endocrinol (Oxf). 2017 Mar;86(3):377-383. doi: 10.1111/cen.13236. Epub 2017 Jan 11.
PubMed 27647266Dzemaili S, Tiemensma J, Quinton R, Pitteloud N, Morin D, Dwyer AA. Beyond hormone replacement: quality of life in women with congenital hypogonadotropic hypogonadism. Endocr Connect. 2017 Aug;6(6):404-412. doi: 10.1530/EC-17-0095. Epub 2017 Jul 11.
PubMed 28698240COST Action BM1105; Badiu C, Bonomi M, Borshchevsky I, Cools M, Craen M, Ghervan C, Hauschild M, Hershkovitz E, Hrabovszky E, Juul A, Kim SH, Kumanov P, Lecumberri B, Lemos MC, Neocleous V, Niedziela M, Djurdjevic SP, Persani L, Phan-Hug F, Pignatelli D, Pitteloud N, Popovic V, Quinton R, Skordis N, Smith N, Stefanija MA, Xu C, Young J, Dwyer AA. Developing and evaluating rare disease educational materials co-created by expert clinicians and patients: the paradigm of congenital hypogonadotropic hypogonadism. Orphanet J Rare Dis. 2017 Mar 20;12(1):57. doi: 10.1186/s13023-017-0608-2.
PubMed 28320476
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Factors Affecting Health Promoting Behavior in Rare Disease Patients: A Mixed Methods Study of Men With Congenital Hypogonadotropic Hypogonadism (CHH)
- ClinicalTrials.gov ID
- NCT01914172
- Acronym / sponsor ID
- 233/13
- Conditions
- Kallmann Syndrome, Congenital Hypogonadotropic Hypogonadism, Idiopathic Hypogonadotropic Hypogonadism
- Keywords
- rare disease, community-based participatory research, health promotion, quantitative research, qualitative research, focus group
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 249 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire VaudoisUniversity, hospital or other
- Collaborators
- University of Lausanne
- Study start
- Jul 2013
- Primary completion
- Mar 2014 (actual)
- Study completion
- 27 Sep 2017 (actual)
- First posted
- 2 Aug 2013 (estimated)
- 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.
Kallmann syndrome (KS), also known as congenital hypogonadotropic hypogonadism (CHH), is a rare endocrine disorder that is characterized by failure to undergo puberty combined with infertility. KS/CHH patients face a number of psychosocial burdens related to delays in diagnosis, inadequate access to expert care, and lack of information about the condition. As such, there is some evidence to suggest that KS/CHH patients have unmet health needs. This study aims to identify the needs of patients and understand the issues that must be overcome to achieve improved health and quality of life.
Detailed description
This study aims to examine the experiences of patients diagnosed with Kallmann syndrome (KS)/congenital hypogonadotropic hypogonadism (CHH).
The study includes two parts:
- online survey (less than 30 minutes to complete)
- focus groups with KS/CHH patients
The aim of this project is to better understand what health needs are not presently being met for these patients and to identify targets for improving the care of patients diagnosed with KS/CHH
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Online web-based questionnaire | – | Other: online questionairresUp to 200 patients with KS/CHH will be recruited to complete an online web-based questionnaire (less than 30 minutes to complete) |
| Patient focus group | – | –Focus groups (90-120 minutes in duration) with 6-12 patients. Up to 36 patients total |
| Online web-based evaluation of patient education materials | – | Other: online questionairresUp to 100 patients with KS/CHH will be recruited to complete an online web-based questionnaire to evaluate patient education materials (less than 15 minutes to complete) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults diagnosed with congenital hypogonadotropic hypogonadism/Kallmann syndrome will be recuited internationally.
Key inclusion criteria (3 listed)
- Diagnosed with congenital hypogonadotropic hypogonadism: i.e. Kallmann syndrome or idiopathic hypogonadotropic hypogonadism
- Primary language is English/capable of responding to a written questionnaire in English
- Consenting to participate in the study
Key exclusion criteria (1 listed)
- other diagnosis of hypogonadism: i.e. hypergonadotropic hypogonadism (Klinefelter syndrome), adult onset hypogonadism, etc.
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 |
|---|---|---|
| online questionairresAlso: patient focus groupssee group descriptions | Other | Online web-based evaluation of patient education materials, Online web-based questionnaire |
Primary outcome
illness perception
Time frame: baseline
subjective self-assessment questionnaire of how KS/CHH impacts a patient's life
depression symptoms
Time frame: baseline
self-rated questionnaire of depression symptoms
adherence to treatment
Time frame: baseline
self-report of adherence to medication treatment and periods without treatment or healthcare
understandability and actionability of patient education materials
Time frame: baeline
completion of Patient Education Materials Assessment Tool (PEMAT)
2 secondary outcomes
coping
Time frame: baseline
Interactions wth healthcare
Time frame: baseline
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 29 Sep 2017.
- Results publication: PubMed 24915927Dwyer AA, Quinton R, Morin D, Pitteloud N. Identifying the unmet health needs of patients with congenital hypogonadotropic hypogonadism using a web-based needs assessment: implications for online interventions and peer-to-peer support. Orphanet J Rare Dis. 2014 Jun 11;9:83. doi: 10.1186/1750-1172-9-83.
- Results publication: PubMed 25844173Dwyer AA, Quinton R, Pitteloud N, Morin D. Psychosexual development in men with congenital hypogonadotropic hypogonadism on long-term treatment: a mixed methods study. Sex Med. 2015 Mar;3(1):32-41. doi: 10.1002/sm2.50.
- Results publication: PubMed 27647266Dwyer AA, Tiemensma J, Quinton R, Pitteloud N, Morin D. Adherence to treatment in men with hypogonadotrophic hypogonadism. Clin Endocrinol (Oxf). 2017 Mar;86(3):377-383. doi: 10.1111/cen.13236. Epub 2017 Jan 11.
- Results publication: PubMed 28698240Dzemaili S, Tiemensma J, Quinton R, Pitteloud N, Morin D, Dwyer AA. Beyond hormone replacement: quality of life in women with congenital hypogonadotropic hypogonadism. Endocr Connect. 2017 Aug;6(6):404-412. doi: 10.1530/EC-17-0095. Epub 2017 Jul 11.
- Results publication: PubMed 28320476COST Action BM1105; Badiu C, Bonomi M, Borshchevsky I, Cools M, Craen M, Ghervan C, Hauschild M, Hershkovitz E, Hrabovszky E, Juul A, Kim SH, Kumanov P, Lecumberri B, Lemos MC, Neocleous V, Niedziela M, Djurdjevic SP, Persani L, Phan-Hug F, Pignatelli D, Pitteloud N, Popovic V, Quinton R, Skordis N, Smith N, Stefanija MA, Xu C, Young J, Dwyer AA. Developing and evaluating rare disease educational materials co-created by expert clinicians and patients: the paradigm of congenital hypogonadotropic hypogonadism. Orphanet J Rare Dis. 2017 Mar 20;12(1):57. doi: 10.1186/s13023-017-0608-2.
- Individual participant dataSharing planned: No. Patient characteristics will be reported in aggregate (no identifiers)
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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.
Andrew Dwyer, PhD, FNP-BC principal investigator
Centre Hositalier Universitaire Vaudois (CHUV)
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 Kallmann Syndrome and Congenital Hypogonadotropic Hypogonadism, run by Centre Hospitalier Universitaire Vaudois at 1 site in Switzerland.
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 3 inclusion and 1 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 29 Sep 2017. 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 Switzerland. 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?
illness perception (time frame: baseline). The study lists 4 primary outcomes in all. 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 NCT01914172, which also lists 5 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 29 Sep 2017.