A Study to Evaluate 5 μg/kg Tbo-filgrastim in Infants, Children and Adolescents With Solid Tumors Without Bone Marrow Involvement

Study at a glance

From the registry record, last updated by the sponsor on 10 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Efficacy, and Immunogenicity of Daily Subcutaneous Administration of 5 ?g/kg Tbo-filgrastim in Infants, Children and Adolescents With Solid Tumors Without Bone Marrow Involvement

ClinicalTrials.gov ID

NCT02190721

Acronym / sponsor ID

XM02-ONC-201

Conditions

Neutropenia

Keywords

Neutropenia

Phase

Phase 2

Study type

Interventional

Recruitment status

Completed

Enrolment

50 participants (actual)

Lead sponsor

Teva Branded Pharmaceutical Products R&D, Inc.Industry (pharma, biotech, devices)

Study start

12 May 2015 (actual)

Primary completion

4 Apr 2017 (actual)

Study completion

4 Apr 2017 (actual)

First posted

15 Jul 2014 (estimated)

Results

Posted 31 Jul 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Not applicable

Intervention model

Single group

Masking

None (open label)

Number of arms

1

Who may qualify

Ages

1 month to 16 years

Sex

All

Healthy volunteers

Not accepted

Age groups

Child

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Participants With Adverse Events (AEs)

   Time frame: Non-TEAE: signing of informed consent to Day -1 (last day of CTX in week 1). And > 30 days after last dose of tbo-filgrastim (Day 46+). TEAE timeframe: Day 1 (start of tbo-filgrastim) to <= 30 days after the last dose (up to Day 45)

   An adverse event was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. A treatment-emergent AE (TEAE) is an AE occurring during the timeframe. A non-TEAE is any AE not considered a TEAE. Severity was rated by the investigator using the NCI Common Terminology Criteria for Adverse Events (NCI-CTCAE) scale where 3=severe but not life-threatening, 4=life-threatening and 5=death. Relation of AE to treatment was determined by the investigator (related=reasonable possibility). Serious AEs include death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, OR an important medical event that jeopardized the patient and required medical intervention to prevent the previously listed serious outcomes.

2. Participants With Potentially Clinically Significant Abnormal Serum Chemistry Results

   Time frame: Day 1 (start of tbo-filgrastim administration) up to Day 21

   Serum chemistry tests included alkaline phosphatase, alanine aminotransferase (ALT), aspartate aminotransferase (AST), direct bilirubin, indirect bilirubin, total bilirubin, calcium, creatinine, gammaglutamyl transpeptidase (GGT), glucose, potassium, lactate dehydrogenase (LDH), phosphate, sodium and uric acid. Only tests with potentially clinically significant abnormal results are reported. ULN = upper limit of normal

3. Participants With Potentially Clinically Significant Abnormal Hematology Results

   Time frame: Day 1 (start of tbo-filgrastim administration) up to Day 21

   Hematology tests included Basophils ABS (x 10^9/L), Basophils (%), Eosinophils ABS (x 10^9/L), Eosinophils (%), Hematocrit (%), Hemoglobin (g/L), Lymphocytes Absolute Count (ABS) (x 10^9/L), Lymphocytes (%), Monocytes ABS (x 10^9/L), Monocytes (%), Neutrophils ABS (x 10^9/L), Neutrophils (%), Platelets (x 10^9/L), Red Blood Cell (RBC) (x 10^12/L), White Blood Cell (WBC) (x 10^9/L). Only tests with potentially clinically significant abnormal results are reported. ULN = upper limit of normal

4. Participants With Potentially Clinically Significant Abnormal Vital Signs

   Time frame: Day 1 (start of tbo-filgrastim administration) up to Day 21

   Vital sign tests included Pulse Rate (bpm), Systolic BP (mmHg), Diastolic BP (mmHg), Respiratory Rate (bpm), and Temperature (°C). Only tests with potentially clinically significant abnormal results are reported.

5. Participants With Potentially Clinically Significant Abnormal Electrocardiogram Results

   Time frame: Day 1 (start of tbo-filgrastim administration) pre-dose, 4 hours post dose and 6 hours post dose; Day 21 (end of study visit)

   Triplicate 12-lead ECGs were conducted at screening, predose, 4 and 6 hours postdose on day 1 of tbo-filgrastim administration, and at the end-of-study visit. The ECGs were interpreted by both the investigator and a qualified physician at the central diagnostic center as normal, abnormal not clinically significant, or abnormal clinically significant. The following parameters were measured/derived for each ECG assessment: heart rate, PR interval, RR interval, QT interval, corrected QT interval according to Fridericia's formula (QTcF), corrected QT interval according to Bazett's formula (QTcB), QRS duration, and QRS axis. The count of participants with potentially clinically significant ECG findings is reported.

6. Participants With Negative Shifts From Baseline to End of Study in Physical Exam Findings

   Time frame: Baseline: Day -21, Day 21 (end of study visit)

   Physical examination was performed at screening and at the end-of-study visit. The following body systems were marked as normal or abnormal and if abnormal, whether clinically significant: Head, ears, eyes, nose and throat (HEENT), chest and lungs, heart, abdomen, skin, lymph nodes and neurological. Any physical examination finding that is judged by the investigator as a clinically significant change (worsening) compared with a baseline value were considered as an adverse event. Counts of participants with a negative shift from baseline in any of the body systems (including shifts from normal to abnormal, not clinically significant) are presented.

7. Participants With Injection Site Reactions to Tbo-Filgrastim Administration

   Time frame: Day 1 (start of tbo-filgrastim administration) up to Day 14

   Using Local Tolerability Assessment Scale ranging from Pain severity 0 (Absent) to 3 (Spontaneously painful)

8. Participants With Negative Shifts From Baseline to End of Study in Spleen Sonography Findings

   Time frame: Baseline: Day -21, Day 21 (end of study visit)

   The investigator assessed spleen sonography findings as normal, abnormal not clinically significant, or abnormal clinically significant. Data representing counts of participants with a negative shift from baseline in spleen sonography findings (including shifts from normal to abnormal, not clinically significant) are presented.

9. Participants Who Were Alive at the 90 Day Follow-Up

   Time frame: 90 days post end of study visit (111 days from start of tbo-filgrastim administration)

   Summary of participant survival at 90 day follow-up.

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2021.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

33 sites in 8 countries: see every site and its status.