Partial Obstruction of Systemic to Pulmonary Artery ShuntPhase 1NCT02765633

Cangrelor Neonatal PK/PD and Safety Study

A Phase 1 study testing Cangrelor in people with Partial Obstruction of Systemic to Pulmonary Artery Shunt and Complete Obstruction of Systemic to Pulmonary Artery Shunt, run by Chiesi Farmaceutici S.p.A. at 2 sites in United States.

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 purpose of this study is to assess the PK/PD and safety profile of cangrelor in neonatal participants at risk of thrombosis. Read more about this study

Who is it for?: Ages 1 day to 28 days
Conditions the study is for: Partial Obstruction of Systemic to Pulmonary Artery Shunt, Complete Obstruction of Systemic to Pulmonary Artery Shunt
What is being tested?: Cangrelor
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 22 participants
Where: 2 sitesUnited States
Study dates: 3 Jan 2017to 23 Dec 2019
Phase: Phase 1
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 1 day to 28 days
All sexes
No healthy volunteers
Child

Who may be able to join

Males and females with congenital heart disease, and ranging in age from birth through 28 days of life
Postoperative neonatal cardiac participants with placement of systemic-to-pulmonary artery palliative shunts, right ventricle to pulmonary artery palliative shunts, or ductus arteriosus stents who are at risk of thrombotic events after repair for structural congenital heart disease.
Written informed consent from a parent/legal guardian
Life expectancy of at least 15 days at study entry
Participants will be excluded from the study if any of the following exclusion criteria apply:
History of intracerebral bleed (confirmed by a ultrasound (US) of the head prior to surgery), or cerebral arteriovenous malformation, or any prior bleed with neurological deficit

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Gastrointestinal or urinary bleeding
Cerebrovascular accident or any cerebrovascular accident with a residual neurological deficit
Known congenital or acquired bleeding or clotting disorder
Weight less than 2.5 kilograms (kg)
Adjusted gestational age less than 37 weeks
Platelet count less than 100,000 cells/microliter (µL)
Chest and/or mediastinal tube blood output of greater than 3 milliliters (mL)/kg/hour (hr) at the time of cangrelor administration
Participants with evidence of severe hepatic or renal failure [aspartate aminotransferase (AST) or alanine aminotransferase (ALT) greater than three times normal for age or total bilirubin greater than 20 milligrams (mg)/deciliter (dL); creatinine greater than 2 times the normal upper limit]
Known allergy to cangrelor or known sensitivity to any component of cangrelor
Any condition that in the investigator's opinion would constitute a contraindication to participation in the study or cause inability to comply with the study requirements
Participation in another investigational therapeutic drug or investigational therapeutic device trial within 30 days of starting study
Participants who have been receiving warfarin (Coumadin®) therapy

Who cannot join

None listed.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Columbia University Medical Center
New York, New York, 10032
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224

Other trials in United States

About this study

Official title: A Prospective, Open-Label, Single-Arm, Multi-Center Study To Assess The Pharmacokinetics/Pharmacodynamics (PK/PD) And Safety Of Different Cangrelor Doses In Neonatal Subjects At Risk Of Thrombosis

The purpose of this study is to assess the PK/PD and safety profile of cangrelor in neonatal participants at risk of thrombosis.

Conditions: Partial Obstruction of Systemic to Pulmonary Artery Shunt, Complete Obstruction of Systemic to Pulmonary Artery Shunt
Being tested: Cangrelor

What participants receive

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

CangrelorDrug

Study groups in NCT02765633
Study group	Role	What they receive
Cangrelor	Experimental	Cangrelor in up to four (4) dose cohorts consisting of a minimum of five participants in each cohort. One cohort of five participants will be enrolled at a time. Cohort 1 subjects will receive Cangrelor at 0.5 mcg/kg/min. Cohort 2 subjects will receive Cangrelor at 0.25 mcg/kg/min. Subsequent cohort dosing decisions are made at the completion of enrollment in each cohort. Receives: Drug: Cangrelor

What is being measured

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

Total plasma concentrations of cangrelor and its primary metabolite, AR C69712, during administration and after cessation of the infusion in neonates
Measured: During study drug infusion through 8 hours post infusion
Proportion of participants in each cohort who achieve ≥90% inhibition of final platelet aggregation as measured by light transmittance aggregometry (LTA) using 20 micromolar (µM) adenosine diphosphate (ADP) in platelet rich plasma
Measured: During study drug infusion through 1 hour post infusion
Individual recovery of platelet function in neonates after cessation of the infusion
Measured: Up to 1 hour post infusion
Assessment of the safety of cangrelor in neonatal participants by evaluating adverse events (AEs) and serious adverse events (SAEs)
Measured: from start of cangrelor infusion through 48 hours post cangrelor infusion

Timeline

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

3 Jan 201723 Dec 2019

First posted6 May 2016 (estimated)
Study start3 Jan 2017
Primary completion31 Aug 2019
Study completion23 Dec 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Partial Obstruction of Systemic to Pulmonary Artery ShuntStudies of the same condition, open and under way.
More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
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 1
Primary purpose: Prevention
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 22 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02765633
Sponsor's protocol ID: MDCO-CAN-15-01
Other IDs: 2016-000134-22, 2016
Status last verified: Feb 2023
First submitted: 14 Apr 2016
Last updated: 15 Feb 2023
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Vargas D, Zhou H, Yu X, Diamond S, Yeh J, Allada V, Krishnamurthy G, Price M, Allen B, Alexander J, Schmidhofer J, Kreutzer J, Vincent J, Morell V, Bacha E, Diacovo T. Cangrelor PK/PD analysis in post-operative neonatal cardiac patients at risk for thrombosis. J Thromb Haemost. 2021 Jan;19(1):202-211. doi: 10.1111/jth.15141. Epub 2020 Nov 29.
PubMed 33078501

Study at a glance

From the registry record, last updated by the sponsor on 15 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective, Open-Label, Single-Arm, Multi-Center Study To Assess The Pharmacokinetics/Pharmacodynamics (PK/PD) And Safety Of Different Cangrelor Doses In Neonatal Subjects At Risk Of Thrombosis
ClinicalTrials.gov ID: NCT02765633
Acronym / sponsor ID: MDCO-CAN-15-01
Conditions: Partial Obstruction of Systemic to Pulmonary Artery Shunt, Complete Obstruction of Systemic to Pulmonary Artery Shunt
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 22 participants (actual)
Lead sponsor: Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
Study start: 3 Jan 2017 (actual)
Primary completion: 31 Aug 2019 (actual)
Study completion: 23 Dec 2019 (actual)
First posted: 6 May 2016 (estimated)
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 purpose of this study is to assess the PK/PD and safety profile of cangrelor in neonatal participants at risk of thrombosis.

Study design

Primary purpose: Prevention
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Cangrelor	Experimental	Drug: CangrelorCangrelor in up to four (4) dose cohorts consisting of a minimum of five participants in each cohort. One cohort of five participants will be enrolled at a time. Cohort 1 subjects will receive Cangrelor at 0.5 mcg/kg/min. Cohort 2 subjects will receive Cangrelor at 0.25 mcg/kg/min. Subsequent cohort dosing decisions are made at the completion of enrollment in each cohort.

Who may qualify

Ages: 1 day to 28 days
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (18 listed)

Males and females with congenital heart disease, and ranging in age from birth through 28 days of life
Postoperative neonatal cardiac participants with placement of systemic-to-pulmonary artery palliative shunts, right ventricle to pulmonary artery palliative shunts, or ductus arteriosus stents who are at risk of thrombotic events after repair for structural congenital heart disease.
Written informed consent from a parent/legal guardian
Life expectancy of at least 15 days at study entry
Participants will be excluded from the study if any of the following exclusion criteria apply:

Key exclusion criteria (0 listed)

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
Cangrelor	Drug	Cangrelor

Primary outcome

Total plasma concentrations of cangrelor and its primary metabolite, AR C69712, during administration and after cessation of the infusion in neonates
Time frame: During study drug infusion through 8 hours post infusion
Proportion of participants in each cohort who achieve ≥90% inhibition of final platelet aggregation as measured by light transmittance aggregometry (LTA) using 20 micromolar (µM) adenosine diphosphate (ADP) in platelet rich plasma
Time frame: During study drug infusion through 1 hour post infusion
Individual recovery of platelet function in neonates after cessation of the infusion
Time frame: Up to 1 hour post infusion
Assessment of the safety of cangrelor in neonatal participants by evaluating adverse events (AEs) and serious adverse events (SAEs)
Time frame: from start of cangrelor infusion through 48 hours post cangrelor infusion

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 15 Feb 2023.
Results publication: PubMed 33078501Vargas D, Zhou H, Yu X, Diamond S, Yeh J, Allada V, Krishnamurthy G, Price M, Allen B, Alexander J, Schmidhofer J, Kreutzer J, Vincent J, Morell V, Bacha E, Diacovo T. Cangrelor PK/PD analysis in post-operative neonatal cardiac patients at risk for thrombosis. J Thromb Haemost. 2021 Jan;19(1):202-211. doi: 10.1111/jth.15141. Epub 2020 Nov 29.
Study Record on EU Clinical Trials Register including resultsLink provided by the sponsor.
Individual participant dataSharing planned: No

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.

Thomas Diacovo, MD principal investigator

Columbia University

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

Questions about this trial

What is this clinical trial studying?

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 18 inclusion and 0 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 15 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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

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?

Total plasma concentrations of cangrelor and its primary metabolite, AR C69712, during administration and after cessation of the infusion in neonates (time frame: During study drug infusion through 8 hours post infusion). The study lists 4 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 NCT02765633, which also lists 1 linked publication.

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 15 Feb 2023.