Cartilage-hair HypoplasiaPhase 4NCT02383797
Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox
A Phase 4 study testing Varilrix in people with Cartilage-hair Hypoplasia, run by Helsinki University Central Hospital.
Trial summary
The investigatoris will carefully select cartilage-hair hypoplasia patients unexposed to varicella (VZV) and immunize patients in a controlled manner with VZV vaccine. Patients will be selected on the basis of disease severity and the degree of immunodeficiency (including CD4+ T-cell counts). Read more about this study
- Who is it for?
- Ages 12 months and older
- Conditions the study is for
- Cartilage-hair Hypoplasia
- What is being tested?
- Varilrix
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- Feb 2015to Feb 2020
- Phase
- Phase 4
- 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 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- genetically confirmed cartilage-hair hypoplasia diagnosis
- age >12 months
- no history of chickenpox
- informed concent of the patient/caregiver.
Who cannot join
- positive serum IgG for varicella zoster virus
- low CD4+ cell counts (<15% or <200 cells/mm3)
- clinical or laboratory signs of severe immunodeficiency
- ongoing intravenous or subcutaneous immunoglobulin treatment
About this study
Official title: Immunodeficiency in Cartilage-hair Hypoplasia: Correlation With Pulmonary Disease, Infections and Malignancy
The investigatoris will carefully select cartilage-hair hypoplasia patients unexposed to varicella (VZV) and immunize patients in a controlled manner with VZV vaccine. Patients will be selected on the basis of disease severity and the degree of immunodeficiency (including CD4+ T-cell counts). Any potential complication of VZV immunization, such as rash, will be discussed with the patients/caregivers beforehand and acyclovir will be used to treat any VZV related symptoms, consistent with the current practices. The investigators will verify the development of immune response to vaccination by testing for VZV antibodies and cell-mediated immunity.
- Conditions
- Cartilage-hair Hypoplasia
- Being tested
- Varilrix
- Medical subject headings
- Cartilage-hair hypoplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VarilrixBiological
Also called ATC J07BK01
Vaccination against varicella in selected cartilage-hair hypoplasia patients
| Study group | Role | What they receive |
|---|---|---|
| Cartilage-hair hypoplasia (CHH) | Experimental | Selected CHH patients will be vaccinated against varicella with Varilrix, one dose of 0,5 ml subcutaneously. If no response is documented to the first dose, the second dose of 0,5 ml can be administered. Receives: Biological: Varilrix |
What is being measured
The primary outcome is the main question the study is designed to answer.
Humoral response to vaccination
Measured: 4-6 weeks post-vaccination
Cell-mediated response to vaccination
Measured: 4-6 wks post-vaccination
Number and severity of adverse events to vaccination
Measured: 0-60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2015 (estimated)
- Study startFeb 2015
- Primary completionOct 2019
- Study completionFeb 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cartilage-hair HypoplasiaStudies of the same condition, open and under way.
- More trials run by Helsinki University Central HospitalThe 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Helsinki University Central Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Paula Klemetti, Helsinki University Central Hospital
- Investigators
- Outi Mäkitie, PhD, Study director, Helsinki University Central Hospital and University of Helsinki, Folkhälsan Research Centre, Helsinki Finland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02383797
- Sponsor's protocol ID
- TYH2013336
- Status last verified
- Oct 2019
- First submitted
- 10 Feb 2015
- Last updated
- 22 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
Vakkilainen S, Kleino I, Honkanen J, Salo H, Kainulainen L, Grasbeck M, Kekalainen E, Makitie O, Klemetti P. The Safety and Efficacy of Live Viral Vaccines in Patients With Cartilage-Hair Hypoplasia. Front Immunol. 2020 Aug 11;11:2020. doi: 10.3389/fimmu.2020.02020. eCollection 2020.
PubMed 32849667
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immunodeficiency in Cartilage-hair Hypoplasia: Correlation With Pulmonary Disease, Infections and Malignancy
- ClinicalTrials.gov ID
- NCT02383797
- Acronym / sponsor ID
- TYH2013336
- Conditions
- Cartilage-hair Hypoplasia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Helsinki University Central HospitalUniversity, hospital or other
- Study start
- Feb 2015
- Primary completion
- Oct 2019 (actual)
- Study completion
- Feb 2020 (estimated)
- First posted
- 9 Mar 2015 (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.
The investigatoris will carefully select cartilage-hair hypoplasia patients unexposed to varicella (VZV) and immunize patients in a controlled manner with VZV vaccine. Patients will be selected on the basis of disease severity and the degree of immunodeficiency (including CD4+ T-cell counts). Any potential complication of VZV immunization, such as rash, will be discussed with the patients/caregivers beforehand and acyclovir will be used to treat any VZV related symptoms, consistent with the current practices. The investigators will verify the development of immune response to vaccination by testing for VZV antibodies and cell-mediated immunity.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cartilage-hair hypoplasia (CHH) | Experimental | Biological: VarilrixSelected CHH patients will be vaccinated against varicella with Varilrix, one dose of 0,5 ml subcutaneously. If no response is documented to the first dose, the second dose of 0,5 ml can be administered. |
Who may qualify
- Ages
- 12 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- genetically confirmed cartilage-hair hypoplasia diagnosis
- age >12 months
- no history of chickenpox
- informed concent of the patient/caregiver.
Key exclusion criteria (4 listed)
- positive serum IgG for varicella zoster virus
- low CD4+ cell counts (<15% or <200 cells/mm3)
- clinical or laboratory signs of severe immunodeficiency
- ongoing intravenous or subcutaneous immunoglobulin treatment
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 |
|---|---|---|
| VarilrixAlso: ATC J07BK01Vaccination against varicella in selected cartilage-hair hypoplasia patients | Biological | Cartilage-hair hypoplasia (CHH) |
Primary outcome
Humoral response to vaccination
Time frame: 4-6 weeks post-vaccination
Cell-mediated response to vaccination
Time frame: 4-6 wks post-vaccination
Number and severity of adverse events to vaccination
Time frame: 0-60 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2019.
- Derived from this study: PubMed 32849667Vakkilainen S, Kleino I, Honkanen J, Salo H, Kainulainen L, Grasbeck M, Kekalainen E, Makitie O, Klemetti P. The Safety and Efficacy of Live Viral Vaccines in Patients With Cartilage-Hair Hypoplasia. Front Immunol. 2020 Aug 11;11:2020. doi: 10.3389/fimmu.2020.02020. eCollection 2020.
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.
Outi Mäkitie, PhD study director
Helsinki University Central Hospital and University of Helsinki, Folkhälsan Research Centre, Helsinki Finland
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Varilrix in people with Cartilage-hair Hypoplasia, run by Helsinki University Central Hospital.
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 4 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 22 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Humoral response to vaccination (time frame: 4-6 weeks post-vaccination). The study lists 3 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 NCT02383797, which also lists 1 linked publication.
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 22 Oct 2019.