Sickle Cell DiseaseESSENTIALNCT01736657

Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients

A study testing Red blood cell exchange in sickle cell in people with Sickle Cell Disease, run by Terumo BCT at 5 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 evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease. Read more about this study

Who is it for?: Ages 12 years and older
Conditions the study is for: Sickle Cell Disease
What is being tested?: Red blood cell exchange in sickle cell
Type of treatment: Medical device
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 73 participants
Where: 5 sitesUnited States
Study dates: Nov 2012to Jun 2013
Phase: Interventional
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 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

At least 12 years old
Enrolled in a program of regular red blood cell exchange (RBCx) to prevent symptoms/complications of sickle cell disease (SCD) or Initiating a program of regular RBCx or Receiving RBCx as a pre-surgical procedure.
Medically stable
Previous documentation of diagnosis by hemoglobin electrophoresis of a type of sickle cell disorder requiring RBCx.
Sufficient vascular access to accommodate the RBCx procedure as determined by the apheresis technician performing the procedure or phlebotomist responsible for obtaining intravenous access.
Availability of sickle trait negative, leukoreduced, ABO blood group, Rhesus factor D (Rh (D)) compatible, unexpired replacement blood. See Glossary for definition of replacement blood.
Able to commit to the study follow-up schedule.
Agree to report adverse events (AEs) during the required reporting period.

Who cannot join

Inability to obtain informed consent/assent from patient, or permission from parent or guardian.
Pregnancy (negative serum pregnancy test required for females of childbearing potential).
Life expectancy is fewer than 30 days from time of procedure.
Incarcerated or a ward of the court.
Refusal of blood products.
Failure to comply with site standard requirements for cessation of medications (e.g., angiotensin converting enzyme (ACE) inhibitors) that interfere with or increase risk of RBCx procedures.
History of drug or alcohol abuse that, in the opinion of the investigator, could affect the ability of the patient to comply with the study requirements Inability to comply with the protocol in the opinion of the investigator.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United States5 sites

University of Colorado at Denver
Aurora, Colorado, 80045
Johns Hopkins Medical
Baltimore, Maryland, 21205
Children's of Alabama
Birmingham, Alabama, 35233
Kosair Children's Hospital
Louisville, Kentucky, 40202
Children's Hospital and Research Center at Oakland
Oakland, California, 94609

Other trials in United States

About this study

Official title: Evaluation of the Spectra Optia Apheresis Red Blood Cell Exchange Protocol in Patients With Sickle Cell Disease.

Read the detailed description

Evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease. Open label design.

Conditions: Sickle Cell Disease
Being tested: Red blood cell exchange in sickle cell
Medical subject headings: Anemia, Sickle Cell

What participants receive

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

Red blood cell exchange in sickle cellDevice
Also called Specta Optia Apheresis System
One Red Blood Cell Exchange using Spectra Optia Apheresis System per enrolled patient

Study groups in NCT01736657
Study group	Role	What they receive
Red cell exchange in sickle cell	Experimental	Open arm; Red cell blood exchange for patients with sickle cell disease Receives: Device: Red blood cell exchange in sickle cell

What is being measured

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

Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS)
The primary endpoint evaluated the mean ratio of the Actual Fraction of Cells Remaining (FCRa: as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp: as predicted by the Spectra Optia system FCR algorithm multiplied by the Pre-Procedure % HbS), in the evaluable population (60 pts). The pre-defined range for the mean ratio of the FCRa to the FCRp was 0.75 to 1.25.
Measured: Length of the procedure

Secondary outcomes (3)

Procedural Success of the Spectra Optia System in the Evaluable Population
The procedural success of the Spectra Optia System is defined as the ability of the device to complete a red blood cell exchange (RBCx) and to obtain a satisfactory exchange by lowering the patient's hemoglobin S, as determined by the investigator in the evaluable population (60 pts).
Measured: Length of the procedure
Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population
Measurement of the patient post-procedure hematocrit compared to the final target hematocrit calculated by the Spectra Optia Apheresis System. Final target hematocrit was calculated by tracking the number of red cells coming into the system versus the number of red cells removed.
Measured: Length of the procedure
Device-related Serious Adverse Events (SAE) in the Full Analysis Set
Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).
Measured: upon signing consent to 24 hours post-procedure

Timeline

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

Nov 2012Jun 2013

First posted29 Nov 2012 (estimated)
Study startNov 2012
Primary completionJun 2013
Study completionJun 2013

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Sickle Cell DiseaseStudies 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 N/A
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 73 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01736657
Sponsor's protocol ID: CTS-5001
Acronym: ESSENTIAL
Status last verified: Jan 2014
First submitted: 27 Nov 2012
Last updated: 14 Jul 2014
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Quirolo K, Bertolone S, Hassell K, Howard T, King KE, Rhodes DK, Bill J. The evaluation of a new apheresis device for automated red blood cell exchange procedures in patients with sickle cell disease. Transfusion. 2015 Apr;55(4):775-81. doi: 10.1111/trf.12891. Epub 2014 Oct 21.
PubMed 25330984

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

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Evaluation of the Spectra Optia Apheresis Red Blood Cell Exchange Protocol in Patients With Sickle Cell Disease.
ClinicalTrials.gov ID: NCT01736657
Acronym / sponsor ID: ESSENTIAL / CTS-5001
Conditions: Sickle Cell Disease
Study type: Interventional
Recruitment status: Completed
Enrolment: 73 participants (actual)
Lead sponsor: Terumo BCTIndustry (pharma, biotech, devices)
Study start: Nov 2012
Primary completion: Jun 2013 (actual)
Study completion: Jun 2013 (actual)
First posted: 29 Nov 2012 (estimated)
Results: Posted 14 Jul 2014

Clinical summary

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

Detailed description

Evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease. Open label design.

Study design

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

Arm	Type	Receives
Red cell exchange in sickle cell	Experimental	Device: Red blood cell exchange in sickle cellOpen arm; Red cell blood exchange for patients with sickle cell disease

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (8 listed)

At least 12 years old
Enrolled in a program of regular red blood cell exchange (RBCx) to prevent symptoms/complications of sickle cell disease (SCD) or Initiating a program of regular RBCx or Receiving RBCx as a pre-surgical procedure.
Medically stable
Previous documentation of diagnosis by hemoglobin electrophoresis of a type of sickle cell disorder requiring RBCx.
Sufficient vascular access to accommodate the RBCx procedure as determined by the apheresis technician performing the procedure or phlebotomist responsible for obtaining intravenous access.

Key exclusion criteria (7 listed)

Inability to obtain informed consent/assent from patient, or permission from parent or guardian.
Pregnancy (negative serum pregnancy test required for females of childbearing potential).
Life expectancy is fewer than 30 days from time of procedure.
Incarcerated or a ward of the court.
Refusal of blood products.

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
Red blood cell exchange in sickle cellAlso: Specta Optia Apheresis SystemOne Red Blood Cell Exchange using Spectra Optia Apheresis System per enrolled patient	Device	Red cell exchange in sickle cell

Primary outcome

Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS)
Time frame: Length of the procedure
The primary endpoint evaluated the mean ratio of the Actual Fraction of Cells Remaining (FCRa: as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp: as predicted by the Spectra Optia system FCR algorithm multiplied by the Pre-Procedure % HbS), in the evaluable population (60 pts). The pre-defined range for the mean ratio of the FCRa to the FCRp was 0.75 to 1.25.

3 secondary outcomes

Procedural Success of the Spectra Optia System in the Evaluable Population
Time frame: Length of the procedure
Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population
Time frame: Length of the procedure
Device-related Serious Adverse Events (SAE) in the Full Analysis Set
Time frame: upon signing consent to 24 hours post-procedure

Results and safety

Results posted to the registry on 14 Jul 2014, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Red Blood Cell Exchange for Patients With Sickle Cell Disease	73	72

Primary outcome: Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS)

Time frame: Length of the procedure. Mean (95% Confidence Interval). Unit: ratio

Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n = 60
Value	0.9 (0.855 to 0.941)

Population analysed: 60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.

3 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Red Blood Cell Exchange for Patients With Sickle Cell Disease	0 of 72 (0%)	10 of 72 (13.9%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2014.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 25330984Quirolo K, Bertolone S, Hassell K, Howard T, King KE, Rhodes DK, Bill J. The evaluation of a new apheresis device for automated red blood cell exchange procedures in patients with sickle cell disease. Transfusion. 2015 Apr;55(4):775-81. doi: 10.1111/trf.12891. Epub 2014 Oct 21.

Related trials for Sickle Cell Disease

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

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.

Keith Quirolo, MD principal investigator

Children's Hospital and Research Center at Oakland

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

Questions about this trial

What is this clinical trial studying?

A study testing Red blood cell exchange in sickle cell in people with Sickle Cell Disease, run by Terumo BCT at 5 sites in United States.

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 8 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 14 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Jul 2014). 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?

Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS) (time frame: Length of the procedure). 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 NCT01736657, 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 14 Jul 2014.