Anemia, Sickle CellPhase 2NCT02672540
A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease Patients
A Phase 2 study testing SANGUINATE 320 mg/kg and Normal Saline in people with Anemia, Sickle Cell, run by Prolong Pharmaceuticals at 4 sites in 4 countries.
Trial summary
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- SANGUINATE 320 mg/kg and Normal Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 34 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Jul 2016to 31 May 2017
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 and ≤ 65 years of age
- Diagnosis of Sickle Cell Disease (Hb-SS or any Genotype)
- Pain-score due to vaso-occlusive pain crisis (VOC) ≥ 8 on a 10 point scale
- VOC pain location ≥ 1 sites typical of vaso-occlusive crisis
- Patients with Priapism, acute chest syndrome, and/or with other Sickle Cell Disease comorbidities can be enrolled with good judgment of the Investigator.
- Signed and dated informed written consent by the subject
- Able to receive intravenous infusion of SANGUINATE or Normal Saline
- Women of childbearing potential with a negative serum pregnancy test and using a reliable method of contraception during the study period and for 30 days thereafter. Male study participants also agree to use contraception for 30 days after the study period
Who cannot join
- In the judgment of the investigator, the patient is not a good candidate for the study
- Females who are lactating and/or breastfeeding
- Fewer than 14 days since prior infusion pain medication treatment for VOC
- Medical history or evidence of moderate to severe renal insufficiency (estimated GFR < 60 mL/min) or chronic kidney disease, or of moderate to severe hepatic disease (ALTs > 5 x ULN)
- Concurrent or prior treatment within 30 days of Screening with an investigational medication.
- Symptoms or electrocardiogram (ECG)-based signs of acute myocardial infarction, unstable angina pectoris, decompensated heart failure, third degree heart block or cardiac arrhythmia associated with hemodynamic instability;
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- Severe or unstable concomitant condition or disease (e.g., known significant neurologic deficit, cancer, hematologic, metabolic or coronary disease), or chronic condition (e.g., psychiatric disorder), that, in the opinion of the Investigator, may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results;
- Evidence or history of regular alcohol abuse
- Screening laboratory result indicating serologic positivity for hepatitis C antibodies or hepatitis B surface antigens, unless explained by a documented vaccination.
- Unable to comply with study attendance, protocol procedures or other study requirements;
- Abnormal Echocardiogram at Study Entry (defined as Tricuspid Regurgitant Jet Velocity >3.1 m/sec).
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: A Phase II, Multicenter, Single-Blind, Randomized Study of the Safety and Effectiveness of SANGUINATE™ Versus Normal Saline in Adult Sickle Cell Disease Patients With Vaso-Occlusive Crisis (VOC)
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment.
Read the detailed description
A single-blind, multicenter, randomized, placebo-control study in which 30 Sickle Cell disease patients having a vaso-occlusive crisis will either receive SANGUINATE 320 mg/kg/patient (8 mL/kg/patient) or Normal Saline on Day 1 (Visit 1) and Day 2 (Visit 2) infused over 2 hours each day. Patients are to remain in the hospital for up to 7 days but can be discharged at any time after receiving the second dose of SANGUINATE, provided their vaso-occlusive crisis has resolved and the patient has completed discharge procedures. Patients will have a follow-up phone call 7 days after discharge from the hospital to obtain safety, concomitant medication and pain assessments.
- Conditions
- Anemia, Sickle Cell
- Being tested
- SANGUINATE 320 mg/kg, Normal Saline
- Keywords
- Sickle Cell, Anemia, VOC, Vaso occlusive crisis
- Medical subject headings
- Anemia, Sickle Cell, Anemia, Vaso-Occlusive Crises
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SANGUINATE 320 mg/kgDrug
Also called pegylated carboxyhemoglobin bovine
Two-hour infusion of SANGUINATE on Day 1 and Day 2
Normal SalineDrug
Two-hour infusion of Normal Saline and Day 1 and Day 2
| Study group | Role | What they receive |
|---|---|---|
| SANGUINATE 320 mg/kg | Experimental | Two-hour infusion of SANGUINATE on Day 1 and Day 2 Receives: Drug: SANGUINATE 320 mg/kg |
| Normal Saline | Placebo comparator | Two-hour infusion of Normal Saline and Day 1 and Day 2 Receives: Drug: Normal Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to readiness for discharge from in hospital stay following treatment with SANGUINATE and Normal Saline.
Defined as the patient's response that their pain episode has improved enough for discharge from the hospital, or the Investigator's assessment that the patient is ready for discharge from the hospital.
Measured: Up to 7 Days
Secondary outcomes (7)
Safety of treatment as defined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, as well as reported increases in pain and other reported adverse events
Composite endpoint with multiple vital signs, ECGs, echocardiograms, clinical assessments and bio-analytical lab measurements over the 7 day time frame
Measured: Up to 7 Days
Proportion of patients who develop acute chest syndrome (ACS) during the study.
Measured: Up to 14 Days
Proportion of patients who are re-hospitalized for their vaso-occlusive crisis episode.
Measured: Up to 7 Days
Total length of stay (LOS) following treatment of SANGUINATE versus Normal Saline.
Measured: Up to 7 Days
Percent reduction in total pain medication required during in-hospital stay following treatment with SANGUINATE and Normal Saline.
Measured: Up to 7 Days
Percent reduction in pain score utilizing a visual analog scale following treatment with SANGUINATE and Normal Saline.
Measured: Up to 7 Days
Reduction in the level of C-Reactive Protein following treatment of SANGUINATE versus Normal Saline.
Measured: Up to 7 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2016 (estimated)
- Study startJul 2016
- Primary completion31 May 2017
- Study completion31 May 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, Sickle CellStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prolong Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hemant Misra, PhD, Study director, Prolong Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02672540
- Sponsor's protocol ID
- SGSC-002
- Status last verified
- May 2018
- First submitted
- 1 Feb 2016
- Last updated
- 23 May 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multicenter, Single-Blind, Randomized Study of the Safety and Effectiveness of SANGUINATE™ Versus Normal Saline in Adult Sickle Cell Disease Patients With Vaso-Occlusive Crisis (VOC)
- ClinicalTrials.gov ID
- NCT02672540
- Acronym / sponsor ID
- SGSC-002
- Conditions
- Anemia, Sickle Cell
- Keywords
- Sickle Cell, Anemia, VOC, Vaso occlusive crisis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Prolong PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jul 2016 (actual)
- Primary completion
- 31 May 2017 (actual)
- Study completion
- 31 May 2017 (actual)
- First posted
- 3 Feb 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.
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment.
Detailed description
A single-blind, multicenter, randomized, placebo-control study in which 30 Sickle Cell disease patients having a vaso-occlusive crisis will either receive SANGUINATE 320 mg/kg/patient (8 mL/kg/patient) or Normal Saline on Day 1 (Visit 1) and Day 2 (Visit 2) infused over 2 hours each day. Patients are to remain in the hospital for up to 7 days but can be discharged at any time after receiving the second dose of SANGUINATE, provided their vaso-occlusive crisis has resolved and the patient has completed discharge procedures. Patients will have a follow-up phone call 7 days after discharge from the hospital to obtain safety, concomitant medication and pain assessments.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SANGUINATE 320 mg/kg | Experimental | Drug: SANGUINATE 320 mg/kgTwo-hour infusion of SANGUINATE on Day 1 and Day 2 |
| Normal Saline | Placebo comparator | Drug: Normal SalineTwo-hour infusion of Normal Saline and Day 1 and Day 2 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age ≥ 18 and ≤ 65 years of age
- Diagnosis of Sickle Cell Disease (Hb-SS or any Genotype)
- Pain-score due to vaso-occlusive pain crisis (VOC) ≥ 8 on a 10 point scale
- VOC pain location ≥ 1 sites typical of vaso-occlusive crisis
- Patients with Priapism, acute chest syndrome, and/or with other Sickle Cell Disease comorbidities can be enrolled with good judgment of the Investigator.
Key exclusion criteria (11 listed)
- In the judgment of the investigator, the patient is not a good candidate for the study
- Females who are lactating and/or breastfeeding
- Fewer than 14 days since prior infusion pain medication treatment for VOC
- Medical history or evidence of moderate to severe renal insufficiency (estimated GFR < 60 mL/min) or chronic kidney disease, or of moderate to severe hepatic disease (ALTs > 5 x ULN)
- Concurrent or prior treatment within 30 days of Screening with an investigational medication.
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 |
|---|---|---|
| SANGUINATE 320 mg/kgAlso: pegylated carboxyhemoglobin bovineTwo-hour infusion of SANGUINATE on Day 1 and Day 2 | Drug | SANGUINATE 320 mg/kg |
| Normal SalineTwo-hour infusion of Normal Saline and Day 1 and Day 2 | Drug | Normal Saline |
Primary outcome
Time to readiness for discharge from in hospital stay following treatment with SANGUINATE and Normal Saline.
Time frame: Up to 7 Days
Defined as the patient's response that their pain episode has improved enough for discharge from the hospital, or the Investigator's assessment that the patient is ready for discharge from the hospital.
7 secondary outcomes
Safety of treatment as defined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, as well as reported increases in pain and other reported adverse events
Time frame: Up to 7 Days
Proportion of patients who develop acute chest syndrome (ACS) during the study.
Time frame: Up to 14 Days
Proportion of patients who are re-hospitalized for their vaso-occlusive crisis episode.
Time frame: Up to 7 Days
Total length of stay (LOS) following treatment of SANGUINATE versus Normal Saline.
Time frame: Up to 7 Days
Percent reduction in total pain medication required during in-hospital stay following treatment with SANGUINATE and Normal Saline.
Time frame: Up to 7 Days
Percent reduction in pain score utilizing a visual analog scale following treatment with SANGUINATE and Normal Saline.
Time frame: Up to 7 Days
Reduction in the level of C-Reactive Protein following treatment of SANGUINATE versus Normal Saline.
Time frame: Up to 7 Days
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 23 May 2018.
- Individual participant dataSharing planned: No
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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.
Hemant Misra, PhD study director
Prolong Pharmaceuticals
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SANGUINATE 320 mg/kg and Normal Saline in people with Anemia, Sickle Cell, run by Prolong Pharmaceuticals at 4 sites in 4 countries.
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 11 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 23 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Colombia, Dominican Republic, Honduras, Panama. 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. Some participants in this study may receive a placebo. 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?
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?
Time to readiness for discharge from in hospital stay following treatment with SANGUINATE and Normal Saline. (time frame: Up to 7 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 May 2018.