Romiplostim Treatment for Thrombocytopenia in Patients Wi... | NCT04350164

Studies
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.

NCT04350164

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Status

Completed

Last Update Posted

Dec 28, 2020Actual

Enrollment

67Actual

Phase

Not provided

Conditions

Wiskott-Aldrich Syndrome

Interventions

Romiplostim

Countries

Russia

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2020

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 1, 2012Actual

Primary Completion Date

Dec 27, 2019Actual

Completion Date

Jun 2020Actual

First Submitted Date

Apr 10, 2020

First Submission Date that Met QC Criteria

Apr 15, 2020

First Posted Date

Apr 16, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 24, 2020

Last Update Posted Date

Dec 28, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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).

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

67Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
treatment		romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.	Drug: Romiplostim

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Romiplostim	Drug	romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.	treatment

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	1 month (30 day +/- 14 days)

Secondary Outcomes

Measure	Description	Time Frame
Percentage of patients with a platelet response		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).

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

• Patients, who do not meet the inclusion criteria.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

patients of the immunology department

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Anna Shcherbina, MD, PhD	National Research Center for Pediatric Hematology , Moscow, Russian Federation	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology		Moscow		117997	Russia

Condition Browse Module

MeSH Terms

ID	Term
D014923	Wiskott-Aldrich Syndrome

Ancestor Terms

ID	Term
D025861	Blood Coagulation Disorders, Inherited
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C488777	romiplostim

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided