Wiskott-Aldrich SyndromeNCT04350164
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
An observational study of Wiskott-Aldrich Syndrome, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
Trial summary
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with genetically confirmed Wiskott-Aldrich syndrome (WAS). Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Wiskott-Aldrich Syndrome
- What is being tested?
- Romiplostim
- Type of treatment
- Injection or infusion
- How many people
- 67 participants
- Where
- 1 siteRussia
- Study dates
- 1 Apr 2012to Jun 2020
- 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: patients of the immunology department
Who may be able to join
- Age under < 18 years
- Subject/legal representative has signed written informed consent. ?
- Subjects diagnosed with WAS based on genetic findings.
- Subjects with thrombocytopenia (platelet count of less than 70 x 109/L).
- Subjects with a history of bleeding.
- Subjects received treatment with romiplostim 8-9 µg /kg for at least 30 days
- Available records of the points of analysis
Who cannot join
- Patients, who do not meet the inclusion criteria.
Trial locations and contacts
1 site in 1 country.
Russia1 site
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117997
About this study
Official title: Retrospective Chart Review of Children With Wiskott-Aldrich Syndrome Who Received Romiplostim in Treatment of Thrombocytopenia.
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with genetically confirmed Wiskott-Aldrich syndrome (WAS).
Read the detailed description
Thrombocytopenia is a life-threatening symptom in WAS patients. Subjects with WAS are at increased risk of debilitating and\ or life-threatening bleedings due to low platelet numbers. Hematopoietic stem cell transplantation is an effective treatment of WAS and all its symptoms yet requires time for donor search and is not widely utilized in cases with mild WAS with isolated thrombocytopenia. TPO-RAs have been used in individual WAS patients, wherein publications describing large WAS cohorts treated with TPO-RAs are lacking.
Based on the previous reports, WAS patients in our Center have been receiving treatment with TPO-RA romiplostim since 2012.
The aim of the study is to retrospective analyze patients' data in order to asses treatment efficacy and safety of romiplostim in WAS thrombocytopenia.
The study will collect and analyze information that is already in the patients' medical records. Information about clinical data (assessment of bleeding tendency with a modified World Health Organization (WHO) Bleeding Scale), laboratory values (such as clinical and biochemical analysis of blood) will be included.
Evaluation of the efficacy therapy was based on the results of physical examination, including bleeding events at the time of diagnosis and after 6-month TPO-RA was initiated and platelet response. A complete response was defined as a platelet count >100 x 109/L in the absence of bleeding symptoms, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L. Non-response was defined as not achieving a platelet count of > 30 x 109/L from the baseline count.
- Conditions
- Wiskott-Aldrich Syndrome
- Being tested
- Romiplostim
- Medical subject headings
- Wiskott-Aldrich Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called Nplate
romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
| Study group | Role | What they receive |
|---|---|---|
| treatment | romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year. Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
The percentage of participants with overall platelet response (complete response + partial response)
A complete response defined as a platelet count >100 x 109/L, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L.
Measured: 1 month (30 day +/- 14 days)
Secondary outcomes (3)
Percentage of patients with a platelet response
Measured: until discontinuation, from at least one month to one year
Number of participants with bleeding events and severity of bleeding
The incidence and severity of bleeding events evaluated with a modified World Health Organization (WHO) Bleeding Scale.
(G1=Petechiae, epistaxis <30 min, G2=Mild blood loss, hematomas, epistaxis >30 min, melanotic stool G3=Gross blood loss, requiring blood transfusions, G4=Fatal bleeding).
Measured: until discontinuation, from at least one month to one year
Number of participants with adverse events
Measured: until discontinuation, from at least one month to one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2020
- Study start1 Apr 2012
- Primary completion27 Dec 2019
- Study completionJun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wiskott-Aldrich SyndromeStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anna Shcherbina, MD, PhD, Principal investigator, National Research Center for Pediatric Hematology , Moscow, Russian Federation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04350164
- Sponsor's protocol ID
- NCPHOI-2020-02
- Status last verified
- Dec 2020
- First submitted
- 10 Apr 2020
- Last updated
- 28 Dec 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Chart Review of Children With Wiskott-Aldrich Syndrome Who Received Romiplostim in Treatment of Thrombocytopenia.
- ClinicalTrials.gov ID
- NCT04350164
- Acronym / sponsor ID
- NCPHOI-2020-02
- Conditions
- Wiskott-Aldrich Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyUniversity, hospital or other
- Study start
- 1 Apr 2012 (actual)
- Primary completion
- 27 Dec 2019 (actual)
- Study completion
- Jun 2020 (actual)
- First posted
- 16 Apr 2020 (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 rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with genetically confirmed Wiskott-Aldrich syndrome (WAS).
Detailed description
Thrombocytopenia is a life-threatening symptom in WAS patients. Subjects with WAS are at increased risk of debilitating and\ or life-threatening bleedings due to low platelet numbers. Hematopoietic stem cell transplantation is an effective treatment of WAS and all its symptoms yet requires time for donor search and is not widely utilized in cases with mild WAS with isolated thrombocytopenia. TPO-RAs have been used in individual WAS patients, wherein publications describing large WAS cohorts treated with TPO-RAs are lacking.
Based on the previous reports, WAS patients in our Center have been receiving treatment with TPO-RA romiplostim since 2012.
The aim of the study is to retrospective analyze patients' data in order to asses treatment efficacy and safety of romiplostim in WAS thrombocytopenia.
The study will collect and analyze information that is already in the patients' medical records. Information about clinical data (assessment of bleeding tendency with a modified World Health Organization (WHO) Bleeding Scale), laboratory values (such as clinical and biochemical analysis of blood) will be included.
Evaluation of the efficacy therapy was based on the results of physical examination, including bleeding events at the time of diagnosis and after 6-month TPO-RA was initiated and platelet response. A complete response was defined as a platelet count >100 x 109/L in the absence of bleeding symptoms, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L. Non-response was defined as not achieving a platelet count of > 30 x 109/L from the baseline count.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment | – | Drug: Romiplostimromiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- patients of the immunology department
Key inclusion criteria (7 listed)
- Age under < 18 years
- Subject/legal representative has signed written informed consent. ?
- Subjects diagnosed with WAS based on genetic findings.
- Subjects with thrombocytopenia (platelet count of less than 70 x 109/L).
- Subjects with a history of bleeding.
Key exclusion criteria (1 listed)
- Patients, who do not meet the inclusion criteria.
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 |
|---|---|---|
| RomiplostimAlso: Nplateromiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year. | Drug | treatment |
Primary outcome
The percentage of participants with overall platelet response (complete response + partial response)
Time frame: 1 month (30 day +/- 14 days)
A complete response defined as a platelet count >100 x 109/L, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L.
3 secondary outcomes
Percentage of patients with a platelet response
Time frame: until discontinuation, from at least one month to one year
Number of participants with bleeding events and severity of bleeding
Time frame: until discontinuation, from at least one month to one year
Number of participants with adverse events
Time frame: until discontinuation, from at least one month to one year
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 28 Dec 2020.
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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.
Anna Shcherbina, MD, PhD principal investigator
National Research Center for Pediatric Hematology , Moscow, Russian Federation
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 Wiskott-Aldrich Syndrome, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
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 7 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 28 Dec 2020. 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 Russia. 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?
The percentage of participants with overall platelet response (complete response + partial response) (time frame: 1 month (30 day +/- 14 days)). 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 28 Dec 2020.