Trial Outcomes & Findings for A Dose Confirmation and Pharmacokinetic Study of Pegcrisantaspase Administered as Intravenous (IV) Infusion in Children and Young Adults With Acute Lymphoblastic Leukemia (ALL) /Lymphoblastic Lymphoma (LBL). Following Hypersensitivity to Pegaspargase (Oncaspar) (NCT NCT02257684)

NCT ID: NCT02257684

Last Updated: 2017-04-04

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: 15 days during Course 1
• Results posted on: 2017-04-04

Participant Flow

Participant milestones

Measure	Pegcrisantaspase IV administration of pegcrisantaspase in Course 1
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	Pegcrisantaspase IV administration of pegcrisantaspase in Course 1
Overall Study Adverse Event	2
Overall Study Lack of Efficacy	1
Overall Study Sponsor's Decision	1

Baseline Characteristics

A Dose Confirmation and Pharmacokinetic Study of Pegcrisantaspase Administered as Intravenous (IV) Infusion in Children and Young Adults With Acute Lymphoblastic Leukemia (ALL) /Lymphoblastic Lymphoma (LBL). Following Hypersensitivity to Pegaspargase (Oncaspar)

Baseline characteristics by cohort

Measure	Pegcrisantaspase n=4 Participants pegcrisantaspase
Age, Continuous	12.95 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 15 days during Course 1

Population: Only 1 of the first 4 patients dosed achieved the predefined serum asparaginase activity (SAA) level above the 0.1 IU/mL therapeutic threshold 14 days following the first IV pegcrisantaspase dose in Course 1 (Primary Objective of the study).

Outcome measures

Measure	Pegcrisantaspase n=1 Participants IV administration of pegcrisantaspase in Course 1
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 0 (Screening)	0.013 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 1 (Pre-dose)	0.013 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 1 (1 hour post)	0.50 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 1 (3 hour post)	0.44 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 1 (5 hour post)	0.41 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 2 (Visit 2)	0.36 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 8 (Visit 3)	0.28 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 11 (Visit 4)	0.24 SAA Level IU/mL
The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1 Course 1; Day 15 (Visit 5)	0.26 SAA Level IU/mL

PRIMARY outcome

Timeframe: 1 Year

Population: Not Applicable, as the study was terminated before this endpoint was analyzed.

Outcome measures

Measure	Pegcrisantaspase IV administration of pegcrisantaspase in Course 1
The Serum Asparaginase Activity 14 Days After the First Infusion of Study Drug and the Adverse Events in All Participants.	0

SECONDARY outcome

Timeframe: 14 Days

Population: Not Applicable, as the study was terminated before this endpoint was analyzed.

Outcome measures

Measure	Pegcrisantaspase IV administration of pegcrisantaspase in Course 1
The Pharmakokinetic (PK) Profile of IV Pegcrisantaspase in Children and Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase. Pharmakokinetic Profiles to be Assessed Are: Half Life, Elimination Rate, Tmax, Cmax, AUC.	0

SECONDARY outcome

Timeframe: 30 Days

Population: Not Applicable, as the study was terminated before this endpoint was analyzed.

Outcome measures

Measure	Pegcrisantaspase IV administration of pegcrisantaspase in Course 1
The SAA Levels Over Time Following Repeated Administration in Children and Young Adults ALL/LBL and Hypersensitivity to Pegaspargase	0

SECONDARY outcome

Timeframe: 30 Days

Population: Not Applicable, as the study was terminated before this endpoint was analyzed.

Outcome measures

Measure	Pegcrisantaspase IV administration of pegcrisantaspase in Course 1
The Immunogenicity of IV Pegcristaspase by Testing Anti-pegcrisantaspase and Anti-PEG Binding and Neutralizing Antibodies	0

Adverse Events

Pegcrisantaspase

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	Pegcrisantaspase n=4 participants at risk IV administration of pegcrisantaspase in Course 1
Blood and lymphatic system disorders Febrile neutropenia	25.0% 1/4
Blood and lymphatic system disorders Pancytopenia	25.0% 1/4
General disorders Pyrexia	25.0% 1/4
Immune system disorders Anaphylactic reaction	25.0% 1/4
Injury, poisoning and procedural complications Infusion related reaction	25.0% 1/4
Neoplasms benign, malignant and unspecified (incl cysts and polyps) T-cell lymphoma recurrent	25.0% 1/4

Other adverse events

Measure	Pegcrisantaspase n=4 participants at risk IV administration of pegcrisantaspase in Course 1
Blood and lymphatic system disorders Anaemia	50.0% 2/4
Blood and lymphatic system disorders Neutropenia	25.0% 1/4
Eye disorders Periorbital oedema	25.0% 1/4
General disorders Mucosal inflammation	25.0% 1/4
Investigations Alanine aminotransferase increased	25.0% 1/4
Investigations Platelet count decreased	25.0% 1/4
Musculoskeletal and connective tissue disorders Muscle spasms	25.0% 1/4
Nervous system disorders Headache	25.0% 1/4
Respiratory, thoracic and mediastinal disorders Nasal congestion	25.0% 1/4
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	25.0% 1/4
Skin and subcutaneous tissue disorders Alopecia	25.0% 1/4

Additional Information

Head of Clinical Development

Jazz Pharmaceuticals

Phone: 650-496-3777

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place