Acute Lymphoblastic LeukemiaPhase 2NCT04067518

A Clinical Study of SHP674 (Pegaspargase) in Participants With Newly Diagnosed, Untreated Acute Lymphoblastic Leukemia

A Phase 2 study testing SHP674 in people with Acute Lymphoblastic Leukemia, run by Institut de Recherches Internationales Servier at 8 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The objectives of the study are to assess the safety and tolerability of a single dose of SHP674 in Japanese participants (dose confirmation) in the tolerability assessment period of Part 1 and to assess the safety, pharmacokinetics and efficacy of SHP674 dose in Part 2 (found to be tolerated in Part 1) in the… Read more about this study

Who is it for?: Ages 1 year to 21 years
Conditions the study is for: Acute Lymphoblastic Leukemia
What is being tested?: SHP674
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 28 participants
Where: 8 sitesJapan
Study dates: 17 Oct 2019to 4 Feb 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 1 year to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Age 1 to ≤21 years at the time of informed consent;
Eastern Cooperative Oncology Group performance status (ECOG PS) 0 to 2;
Newly diagnosed, untreated precursor B-cell ALL
No prior therapy for malignant tumor such as chemotherapy and radiation therapy before signing the informed consent;
Life expectancy of at least 6 months from the date of enrollment;

Who cannot join

Mature B-cell ALL ; Philadelphia chromosome-positive (Ph+) or BCR-ABL1-positive ALL
Preexisting known coagulopathy ;
History of pancreatitis;
Continuous use of corticosteroids;
Prior treatment or possible prior treatment with an L-asparaginase preparation;
History of sensitivity to polyethylene glycol (PEG) or PEG-based drugs;
Pregnant

Trial locations and contacts

8 sites in 1 country.

City, postcode or hospital

8 sites in 1 country

Japan8 sites

Kagoshima University Hospital Department of Pediatrics
Kagoshima
Kobe Children's Hospital Department of Hematology/Oncology
Kobe
Nagoya Medical Center Department of Pediatrics
Nagoya
Niigata Cancer Center Hospital
Niigata
Saitama Children's Medical Center Department of Hematology/Oncology
Saitama
Sapporo Hokuyu Hospital Department of Pediatrics and Adolescent Medicine
Sapporo
National Cancer Center Hospital Department of Pediatric Oncology
Tokyo
St. Luke's International Hospital Department of Pediatrics
Tokyo

Other trials in Japan

About this study

Official title: A Phase 2 Clinical Study of SHP674 in Patients With Newly Diagnosed, Untreated Acute Lymphoblastic Leukemia

Conditions: Acute Lymphoblastic Leukemia
Being tested: SHP674
Keywords: Acute Lymphoblastic Leukemia
Medical subject headings: Precursor Cell Lymphoblastic Leukemia-Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SHP674Biological
Also called Pegaspargase
SHP674: powder for solution for injection, IV (administered by 1 to 2 hours of drip infusion), dose determination : if BSA ≥0.6 m^2: 2500 IU/m^2 every 14 days if BSA <0.6 m^2: 82.5 IU/kg every 14 days

Study groups in NCT04067518
Study group	Role	What they receive
SHP674	Experimental	Part 1: Participants with ALL who were stratified into the standard risk (SR) or intermediate risk (IR) groups received total 3 doses of SHP674 in the 36-week treatment period and who were stratified into the high risk (HR) group received total 8 doses of SHP674 in the 45-week treatment period. Part 2: Participants with ALL who were stratified into the SR or IR groups received total 3 doses of SHP674 in the 41-week treatment period and who were stratified into the HR group received total 8 doses of SHP674 in the 45-week treatment period. Receives: Biological: SHP674

What is being measured

The primary outcome is the main question the study is designed to answer.

Part 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) and SHP-674-Related TEAEs During the Tolerability Assessment Period
An adverse event (AE) is defined as any untoward medical occurrence in a participant after signing informed consent. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease, whether or not it is related to the investigational product. TEAE is defined as any untoward medical occurrence in a participant who received an investigational product which occurs during the period from Day 1 of the pre-treatment phase to 30 (+7) days after the last dose of investigational product, or until the start of a new therapy, whichever occurs first. A related adverse event signifies that there is a reasonable causal relationship between study treatment and an AE.
Measured: Up to 30 days after last dose of study drug (approximately 49 weeks)
Part 2: Percentage of Participants Who Achieved a Plasma Asparaginase Activity of ≥0.1 International Units Per Milliliter (IU/mL) 14 Days (336 Hours) After the First Dose of SHP674
Measured: 14 days after the first dose of SHP674

Secondary outcomes (6)

Percentage of Participants With Anti-Drug (SHP674) Antibody (ADA) (Part 1 and Part 2)
Measured: Predose and 25 days post dose (Part 1 and Part 2)
Percentage of Participants With Anti-Polyethylene Glycol (PEG) Antibody (Part 1 and Part 2)
Measured: Predose and 25 days post dose (Part 1 and part 2)
Part 1: Percentage of Participants Who Achieved a Plasma Asparaginase Activity of ≥0.1 IU/mL 14 Days (336 Hours) After the First Dose of SHP674
Measured: 14 days after the first dose of SHP674
Part 2: Percentage of Participants With Plasma Asparaginase Activity of ≥0.1 IU/mL or <0.1 IU/mL
Measured: Day 1 (pre-dose, 5 min, 4 hours, 24 hours post dose), Days 2, 4, 11, 14, 18, 25 post dose
Survival Rate at 1 Year After the Start of Study Treatment
Survival rate is defined as the percentage of subjects who survived at 1 year after the start of study treatment.
Measured: 1 year after the start of study treatment (from first dose up to 12 months)
Event-free Survival Rate at 1 Year After the Start of Study Treatment
Event-free survival rate is defined as percentage of subjects who did not experience any event and survived at 1 year after the start of study treatment.
Measured: 1 year after the start of study treatment (from first dose up to 12 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

17 Oct 20194 Feb 2022

First posted26 Aug 2019
Study start17 Oct 2019
Primary completion12 Feb 2021
Study completion4 Feb 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Acute Lymphoblastic LeukemiaStudies 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: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: The intervention study model is sequential in results section of record.
Masking: None (open label)
Enrolment: 28 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04067518
Sponsor's protocol ID: SHP674-201/CL1-95014-001
Status last verified: Mar 2023
First submitted: 13 Aug 2019
Last updated: 20 Apr 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Qualified scientific and medical researchers can request access to anonymized participant-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in participants: * sponsored by Servier * with a first participant enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Acute Lymphoblastic Leukemia trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Clinical Study of SHP674 in Patients With Newly Diagnosed, Untreated Acute Lymphoblastic Leukemia
ClinicalTrials.gov ID: NCT04067518
Acronym / sponsor ID: SHP674-201/CL1-95014-001
Conditions: Acute Lymphoblastic Leukemia
Keywords: Acute Lymphoblastic Leukemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 28 participants (actual)
Lead sponsor: Institut de Recherches Internationales ServierUniversity, hospital or other
Collaborators: Kyowa Kirin Co., Ltd., ADIR, a Servier Group company
Study start: 17 Oct 2019 (actual)
Primary completion: 12 Feb 2021 (actual)
Study completion: 4 Feb 2022 (actual)
First posted: 26 Aug 2019 (actual)
Results: Posted 10 Jun 2022

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
Model description: The intervention study model is sequential in results section of record.

Arm	Type	Receives
SHP674	Experimental	Biological: SHP674Part 1: Participants with ALL who were stratified into the standard risk (SR) or intermediate risk (IR) groups received total 3 doses of SHP674 in the 36-week treatment period and who were stratified into the high risk (HR) group received total 8 doses of SHP674 in the 45-week treatment period. Part 2: Participants with ALL who were stratified into the SR or IR groups received total 3 doses of SHP674 in the 41-week treatment period and who were stratified into the HR group received total 8 doses of SHP674 in the 45-week treatment period.

Who may qualify

Ages: 1 year to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (5 listed)

Age 1 to ≤21 years at the time of informed consent;
Eastern Cooperative Oncology Group performance status (ECOG PS) 0 to 2;
Newly diagnosed, untreated precursor B-cell ALL
No prior therapy for malignant tumor such as chemotherapy and radiation therapy before signing the informed consent;
Life expectancy of at least 6 months from the date of enrollment;

Key exclusion criteria (7 listed)

Mature B-cell ALL ; Philadelphia chromosome-positive (Ph+) or BCR-ABL1-positive ALL
Preexisting known coagulopathy ;
History of pancreatitis;
Continuous use of corticosteroids;
Prior treatment or possible prior treatment with an L-asparaginase preparation;

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
SHP674Also: PegaspargaseSHP674: powder for solution for injection, IV (administered by 1 to 2 hours of drip infusion), dose determination : if BSA ≥0.6 m^2: 2500 IU/m^2 every 14 days if BSA <0.6 m^2: 82.5 IU/kg every 14 days	Biological	SHP674

Primary outcome

Part 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) and SHP-674-Related TEAEs During the Tolerability Assessment Period
Time frame: Up to 30 days after last dose of study drug (approximately 49 weeks)
An adverse event (AE) is defined as any untoward medical occurrence in a participant after signing informed consent. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease, whether or not it is related to the investigational product. TEAE is defined as any untoward medical occurrence in a participant who received an investigational product which occurs during the period from Day 1 of the pre-treatment phase to 30 (+7) days after the last dose of investigational product, or until the start of a new therapy, whichever occurs first. A related adverse event signifies that there is a reasonable causal relationship between study treatment and an AE.
Part 2: Percentage of Participants Who Achieved a Plasma Asparaginase Activity of ≥0.1 International Units Per Milliliter (IU/mL) 14 Days (336 Hours) After the First Dose of SHP674
Time frame: 14 days after the first dose of SHP674

6 secondary outcomes

Percentage of Participants With Anti-Drug (SHP674) Antibody (ADA) (Part 1 and Part 2)
Time frame: Predose and 25 days post dose (Part 1 and Part 2)
Percentage of Participants With Anti-Polyethylene Glycol (PEG) Antibody (Part 1 and Part 2)
Time frame: Predose and 25 days post dose (Part 1 and part 2)
Part 1: Percentage of Participants Who Achieved a Plasma Asparaginase Activity of ≥0.1 IU/mL 14 Days (336 Hours) After the First Dose of SHP674
Time frame: 14 days after the first dose of SHP674
Part 2: Percentage of Participants With Plasma Asparaginase Activity of ≥0.1 IU/mL or <0.1 IU/mL
Time frame: Day 1 (pre-dose, 5 min, 4 hours, 24 hours post dose), Days 2, 4, 11, 14, 18, 25 post dose
Survival Rate at 1 Year After the Start of Study Treatment
Time frame: 1 year after the start of study treatment (from first dose up to 12 months)
Event-free Survival Rate at 1 Year After the Start of Study Treatment
Time frame: 1 year after the start of study treatment (from first dose up to 12 months)

Results and safety

Results posted to the registry on 10 Jun 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part 1: SHP674	3	2
Part 2: SHP674	25	20

Primary outcome: Part 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) and SHP-674-Related TEAEs During the Tolerability Assessment Period

Time frame: Up to 30 days after last dose of study drug (approximately 49 weeks). Count of participants. Unit: Participants

Measure	Part 1: SHP674 n = 3
TEAEs	3
SHP-674-Related TEAEs	3

Population analysed: SAF (Safety Analysis Set) included all participants who had received at least one dose of SHP674 in Part 1 or Part 2 of the study. As pre-specified in the protocol, this outcome measure is analyzed only for Part 1.

Primary outcome: Part 2: Percentage of Participants Who Achieved a Plasma Asparaginase Activity of ≥0.1 International Units Per Milliliter (IU/mL) 14 Days (336 Hours) After the First Dose of SHP674

Time frame: 14 days after the first dose of SHP674. Number (95% Confidence Interval). Unit: participants

Measure	Part 2: SHP674 n = 23
Value	100.0 (85.2 to 100.0)

Population analysed: FAS (Full Analysis Set) included all participants who were enrolled and received SHP674 in Part 2 of the study.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Parts 1 and 2: Up to 30 days after last dose of study drug (approximatively 117 weeks). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part 1: SHP674	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Part 2: SHP674	10 of 23 (43.5%)	23 of 23 (100%)	0 of 23

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Find Results on Servier Clinical Trial Data websiteLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Qualified scientific and medical researchers can request access to anonymized participant-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in participants: * sponsored by Servier * with a first participant enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

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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.

Chitose Ogawa, MD principal investigator

National Cancer Center Hospital, Tokyo JAPAN

8 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SHP674 in people with Acute Lymphoblastic Leukemia, run by Institut de Recherches Internationales Servier at 8 sites in Japan.

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 5 inclusion and 7 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 20 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in Japan. 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.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Part 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) and SHP-674-Related TEAEs During the Tolerability Assessment Period (time frame: Up to 30 days after last dose of study drug (approximately 49 weeks)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04067518.

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 20 Apr 2023.