Immune-mediated Thrombocytopenic PurpuraNCT05263193
Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)
An observational study of Immune-mediated Thrombocytopenic Purpura, run by Sanofi at 1 site in France.
Trial summary
The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Immune-mediated Thrombocytopenic Purpura
- What is being tested?
- Caplacizumab
- How many people
- 4 participants
- Where
- 1 siteFrance
- Study dates
- 8 Jun 2022to 28 Oct 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric patients with Immune-mediated Thrombotic Thrombocytopenic Purpura treated with Caplacizumab
Who may be able to join
- Patient's aged ≤18 years at start of caplacizumab treatment initiation
- Patient has a diagnosis of iTTP documented in the medical records
- Patient was treated with caplacizumab within the eligibility period
Who cannot join
- Patient declined use of data for study (where local regulations require patient notification of planned study)
- Patient's medical chart is missing or not retrievable
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab
The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP.
Read the detailed description
Pediatric patients who received caplacizumab will be identified for enrollment in the chart review. The eligibility period starts on August 30, 2018 in the United Kingdom (UK) and France and February 6, 2019 for the United States (US).
Data collection is fully retrospective and will be anchored to the patient's index event date. The index event date is defined as the date the patient initiated caplacizumab treatment. The study period begins at the index date and ends at the earliest date of chart abstraction initiation, 12 weeks after last dose of caplacizumab treatment, date of death, or loss to follow-upwhich ever comes first .
- Conditions
- Immune-mediated Thrombocytopenic Purpura
- Being tested
- Caplacizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CaplacizumabDrug
Also called Cablivi®
as in real world practice
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet count response
defined as time from caplacizumab initiation to initial platelet count ≥ 150×109/L with subsequent stop of daily plasma exchange (PE) within 5 days
Measured: From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with refractory iTTP
defined as lack of doubling of platelet count after four days of caplacizumab treatment and a lactate dehydrogenase (LDH) level that remained above the upper limit of normal (ULN) range
Measured: From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with recurrent disease
Proportion of subjects with iTTP exacerbation (defined as recurrence within 30 days after last PE) and Proportion of subjects with iTTP relapse (defined as recurrence more than 30 days after last PE)
Measured: From index date up to 12 weeks after last dose of caplacizumab
Time to normalization of organ damage marker levels
Defined asLDH ≤ 2 x ULN, Serum creatinine ≤ 1 x ULN, Cardiac troponin I ≤ 1 x ULN
Measured: From index date up to 12 weeks after last dose of caplacizumab
Total duration of hospitalization stays
Measured: From index date up to 12 weeks after last dose of caplacizumab
Duration of intensive care unit (ICU) stay
Measured: From index date up to 12 weeks after last dose of caplacizumab
Duration of therapeutic PE
Measured: From index date up to 12 weeks after last dose of caplacizumab
Proportion of patients achieving clinical response
defined as a normal platelet countand LDH < 2 ULN for at least 48 hours following initial normalization or response of platelet count
Measured: From index date up to 12 weeks after last dose of caplacizumab
Time to ADAMTS13 activity ≥ 20%
where available and feasible
Measured: From index date up to 12 weeks after last dose of caplacizumab
Number of participants with Adverse event
including serious adverse events
Measured: From index date up to 12 weeks after last dose of caplacizumab
Secondary outcomes (2)
Treatment pattern of caplacizumab therapy
Dosing and duration
Measured: From index date up to 12 weeks after last dose of caplacizumab
Types and duration of concomitant medications
Measured: From index date up to 12 weeks after last dose of caplacizumab
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2022
- Study start8 Jun 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 Immune-mediated Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by SanofiThe 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
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences and Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05263193
- Sponsor's protocol ID
- OBS17325
- Status last verified
- Dec 2022
- First submitted
- 22 Feb 2022
- Last updated
- 14 Dec 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab
- ClinicalTrials.gov ID
- NCT05263193
- Acronym / sponsor ID
- OBS17325
- Conditions
- Immune-mediated Thrombocytopenic Purpura
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 8 Jun 2022 (actual)
- Primary completion
- 28 Oct 2022 (actual)
- Study completion
- 28 Oct 2022 (actual)
- First posted
- 2 Mar 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.
The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP.
Detailed description
Pediatric patients who received caplacizumab will be identified for enrollment in the chart review. The eligibility period starts on August 30, 2018 in the United Kingdom (UK) and France and February 6, 2019 for the United States (US).
Data collection is fully retrospective and will be anchored to the patient's index event date. The index event date is defined as the date the patient initiated caplacizumab treatment. The study period begins at the index date and ends at the earliest date of chart abstraction initiation, 12 weeks after last dose of caplacizumab treatment, date of death, or loss to follow-upwhich ever comes first .
Study design
- Observational model
- Case-control
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric patients with Immune-mediated Thrombotic Thrombocytopenic Purpura treated with Caplacizumab
Key inclusion criteria (3 listed)
- Patient's aged ≤18 years at start of caplacizumab treatment initiation
- Patient has a diagnosis of iTTP documented in the medical records
- Patient was treated with caplacizumab within the eligibility period
Key exclusion criteria (2 listed)
- Patient declined use of data for study (where local regulations require patient notification of planned study)
- Patient's medical chart is missing or not retrievable
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 |
|---|---|---|
| CaplacizumabAlso: Cablivi®as in real world practice | Drug | – |
Primary outcome
Platelet count response
Time frame: From index date up to 12 weeks after last dose of caplacizumab
defined as time from caplacizumab initiation to initial platelet count ≥ 150×109/L with subsequent stop of daily plasma exchange (PE) within 5 days
Proportion of subjects with refractory iTTP
Time frame: From index date up to 12 weeks after last dose of caplacizumab
defined as lack of doubling of platelet count after four days of caplacizumab treatment and a lactate dehydrogenase (LDH) level that remained above the upper limit of normal (ULN) range
Proportion of subjects with recurrent disease
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with iTTP exacerbation (defined as recurrence within 30 days after last PE) and Proportion of subjects with iTTP relapse (defined as recurrence more than 30 days after last PE)
Time to normalization of organ damage marker levels
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Defined asLDH ≤ 2 x ULN, Serum creatinine ≤ 1 x ULN, Cardiac troponin I ≤ 1 x ULN
Total duration of hospitalization stays
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Duration of intensive care unit (ICU) stay
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Duration of therapeutic PE
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Proportion of patients achieving clinical response
Time frame: From index date up to 12 weeks after last dose of caplacizumab
defined as a normal platelet countand LDH < 2 ULN for at least 48 hours following initial normalization or response of platelet count
Time to ADAMTS13 activity ≥ 20%
Time frame: From index date up to 12 weeks after last dose of caplacizumab
where available and feasible
Number of participants with Adverse event
Time frame: From index date up to 12 weeks after last dose of caplacizumab
including serious adverse events
2 secondary outcomes
Treatment pattern of caplacizumab therapy
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Types and duration of concomitant medications
Time frame: From index date up to 12 weeks after last dose of caplacizumab
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 14 Dec 2022.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
Clinical Sciences and Operations study director
Sanofi
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 Immune-mediated Thrombocytopenic Purpura, run by Sanofi 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 3 inclusion and 2 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 14 Dec 2022. 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?
Platelet count response (time frame: From index date up to 12 weeks after last dose of caplacizumab). The study lists 10 primary outcomes in all. 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 14 Dec 2022.