Sickle Cell DiseasePhase 2NCT05470270
Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
A Phase 2 study testing Hydroxycarbamide in people with Sickle Cell Disease, run by Theravia at 3 sites in France.
Trial summary
This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg… Read more about this study
- Who is it for?
- Ages 2 to 6 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Hydroxycarbamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 3 sitesFrance
- Study dates
- 8 Jul 2022to 28 Oct 2022
- Phase
- Phase 2
- 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 2 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children,
- Child with sickle cell disease, treated with 100 mg and/or 1000 mg Siklos® film-coated tablets at the same daily dose for more than 4 weeks,
- Child aged between 2 and 6 years old,
- Parents capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements,
- Children affiliated to a social security plan (including universal health coverage) or beneficiary of a similar insurance plan.
Who cannot join
- Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding study inclusion,
- Known hypersensitivity or allergy to the excipients,
- Any surgical or medical condition or any significant illness (of which severe hepatic impairment (Child-Pugh classification C), severe renal impairment, toxic ranges of myelosuppression) that, in the opinion of the investigator, constitutes a risk or a contraindication to the participation of the patient to the study, or that may interfere with the objectives, conduct or evaluation of the study.
Trial locations and contacts
3 sites in 1 country.
France3 sites
InterCommunal Hospital Centre of Creteil
Créteil, 94000
Necker University Hospital
Paris, 75015
Robert Debré Hospital
Paris, 75019
About this study
Official title: An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg and/or 1000 mg film-coated tablets).
- Conditions
- Sickle Cell Disease
- Being tested
- Hydroxycarbamide
- Keywords
- hydroxycarbamide, pediatric, acceptability
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxycarbamideDrug
Single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide.
| Study group | Role | What they receive |
|---|---|---|
| New formulation of hydroxycarbamide | Experimental | Single arm study with a single administration of hydroxycarbamide Receives: Drug: Hydroxycarbamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Acceptability score
Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)
Measured: At Day 1 (inclusion visit at study drug administration)
Secondary outcomes (6)
Percentage of children with acceptable acceptability score (neutral to positive scores)
Neutral to positive scores reported by the parent(s) of the child (2-6 years old), and by the child (4-6 years old)
Measured: At Day 1 (inclusion visit at study drug administration)
Distribution of the scores related to the ease of administration
Score reported by parent(s), based on a 5-point Likert scale,
Measured: At Day 1 (inclusion visit at study drug administration)
Distribution of the scores related to the ease of preparation including the ease of constitution of the liquid form and the ease to follow the prescription
Score reported by the parent(s) based on a 5-point Likert scale,
Measured: At Day 1 (inclusion visit at study drug administration)
Score related to the usefulness of the dispersible form, compared with the tablets currently used
Score reported by the parent(s), based on a 5-point Likert scale
Measured: At Day 1 (inclusion visit at study drug administration)
Free comments collected by the investigator
Questions from child/parent, reactions before/after drug intake
Measured: At Day 1 (inclusion visit at study drug administration)
Number of adverse events
Number of adverse events and percentage of patients reporting at least one adverse event
Measured: At Day 1 (inclusion visit at study drug administration)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2022
- Study start8 Jul 2022
- Primary completion28 Oct 2022
- Study completion28 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Theravia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bérengère Koel, MD, Principal investigator, Hopital Universitaire Robert-Debre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05470270
- Sponsor's protocol ID
- SIK-FR-22-1
- Status last verified
- Nov 2022
- First submitted
- 19 Jul 2022
- Last updated
- 23 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
- ClinicalTrials.gov ID
- NCT05470270
- Acronym / sponsor ID
- SIK-FR-22-1
- Conditions
- Sickle Cell Disease
- Keywords
- hydroxycarbamide, pediatric, acceptability
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- TheraviaIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2022 (actual)
- Primary completion
- 28 Oct 2022 (actual)
- Study completion
- 28 Oct 2022 (actual)
- First posted
- 22 Jul 2022 (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.
This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg and/or 1000 mg film-coated tablets).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| New formulation of hydroxycarbamide | Experimental | Drug: HydroxycarbamideSingle arm study with a single administration of hydroxycarbamide |
Who may qualify
- Ages
- 2 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children,
- Child with sickle cell disease, treated with 100 mg and/or 1000 mg Siklos® film-coated tablets at the same daily dose for more than 4 weeks,
- Child aged between 2 and 6 years old,
- Parents capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements,
- Children affiliated to a social security plan (including universal health coverage) or beneficiary of a similar insurance plan.
Key exclusion criteria (3 listed)
- Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding study inclusion,
- Known hypersensitivity or allergy to the excipients,
- Any surgical or medical condition or any significant illness (of which severe hepatic impairment (Child-Pugh classification C), severe renal impairment, toxic ranges of myelosuppression) that, in the opinion of the investigator, constitutes a risk or a contraindication to the participation of the patient to the study, or that may interfere with the objectives, conduct or evaluation of the study.
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 |
|---|---|---|
| HydroxycarbamideSingle administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide. | Drug | New formulation of hydroxycarbamide |
Primary outcome
Acceptability score
Time frame: At Day 1 (inclusion visit at study drug administration)
Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)
6 secondary outcomes
Percentage of children with acceptable acceptability score (neutral to positive scores)
Time frame: At Day 1 (inclusion visit at study drug administration)
Distribution of the scores related to the ease of administration
Time frame: At Day 1 (inclusion visit at study drug administration)
Distribution of the scores related to the ease of preparation including the ease of constitution of the liquid form and the ease to follow the prescription
Time frame: At Day 1 (inclusion visit at study drug administration)
Score related to the usefulness of the dispersible form, compared with the tablets currently used
Time frame: At Day 1 (inclusion visit at study drug administration)
Free comments collected by the investigator
Time frame: At Day 1 (inclusion visit at study drug administration)
Number of adverse events
Time frame: At Day 1 (inclusion visit at study drug administration)
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 23 Nov 2022.
- Individual participant dataSharing planned: No
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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.
Bérengère Koel, MD principal investigator
Hopital Universitaire Robert-Debre
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxycarbamide in people with Sickle Cell Disease, run by Theravia at 3 sites 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 5 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 23 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Acceptability score (time frame: At Day 1 (inclusion visit at study drug administration)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Nov 2022.