Sickle Cell DiseasePhase 1/2NCT00911495
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
A Phase 1/2 study testing GMI-1070 in people with Sickle Cell Disease, run by GlycoMimetics Incorporated at 3 sites in United States.
Trial summary
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- GMI-1070
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesUnited States
- Study dates
- May 2009to Sep 2010
- Phase
- Phase 1/2
- 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 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age 18 to 45 years
- Established diagnosis of SCD-SS or SCD-SB0-thal
- At medical baseline, with no evidence of worsening of disease over the last 3 months
- Available and agree to return for follow-up visits for the full duration of the study
- Able to cooperate with study procedures
- Documented and observed written informed consent
Who cannot join
- Vaso-occlusive crisis
- Recent major surgery, hospitalization, infection, significant bleeding, cerebrovascular accident or seizure, or transfusion
- Currently receiving, or has received within the previous 4 weeks, any other investigational agent
- Pregnant or lactating female; or female of childbearing age unable or unwilling to comply with birth control or abstinence during the course of the study
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Duke Comprehensive Sickle Cell Center
Durham, North Carolina, 27710
Children's Hospital & Research Center Oakland
Oakland, California, 94609
University of California at Davis, CCRC
Sacramento, California, 95817
About this study
Official title: Phase 1/Phase 2 Study of the Safety, Pharmacokinetics, and Microvascular Effect of Titrating Doses of Intravenous GMI-1070, a Pan-Selectin Inhibitor, in Adults With Sickle Cell Disease
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting.
- Conditions
- Sickle Cell Disease
- Being tested
- GMI-1070
- Keywords
- Sickle Cell Disease
- 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.
GMI-1070Drug
Intravenous GMI-1070 given as two doses over the course of one day
| Study group | Role | What they receive |
|---|---|---|
| GMI-1070 | Experimental | Not described Receives: Drug: GMI-1070 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as Measured by the Number of Participants With Adverse Events
Measured: 28 days
Secondary outcomes (4)
Total Plasma Clearance
Measured: 48 hours
Volume of the Central Compartment
Measured: 48 hours
Intercompartmental Clearance
Measured: 48 hours
Volume of the Peripheral Compartment
Measured: 48 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2009 (estimated)
- Study startMay 2009
- Primary completionJul 2010
- Study completionSep 2010
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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- GlycoMimetics Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Helen Thackray, MD, Study director, GlycoMimetics Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00911495
- Sponsor's protocol ID
- GMI-1070-103
- Status last verified
- May 2020
- First submitted
- 27 May 2009
- Last updated
- 11 May 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Wun T, Styles L, DeCastro L, Telen MJ, Kuypers F, Cheung A, Kramer W, Flanner H, Rhee S, Magnani JL, Thackray H. Phase 1 study of the E-selectin inhibitor GMI 1070 in patients with sickle cell anemia. PLoS One. 2014 Jul 2;9(7):e101301. doi: 10.1371/journal.pone.0101301. eCollection 2014.
PubMed 24988449
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/Phase 2 Study of the Safety, Pharmacokinetics, and Microvascular Effect of Titrating Doses of Intravenous GMI-1070, a Pan-Selectin Inhibitor, in Adults With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00911495
- Acronym / sponsor ID
- GMI-1070-103
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- GlycoMimetics IncorporatedIndustry (pharma, biotech, devices)
- Study start
- May 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Sep 2010 (actual)
- First posted
- 2 Jun 2009 (estimated)
- Results
- Posted 3 May 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GMI-1070 | Experimental | Drug: GMI-1070 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Age 18 to 45 years
- Established diagnosis of SCD-SS or SCD-SB0-thal
- At medical baseline, with no evidence of worsening of disease over the last 3 months
- Available and agree to return for follow-up visits for the full duration of the study
- Able to cooperate with study procedures
Key exclusion criteria (4 listed)
- Vaso-occlusive crisis
- Recent major surgery, hospitalization, infection, significant bleeding, cerebrovascular accident or seizure, or transfusion
- Currently receiving, or has received within the previous 4 weeks, any other investigational agent
- Pregnant or lactating female; or female of childbearing age unable or unwilling to comply with birth control or abstinence during the course of the study
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 |
|---|---|---|
| GMI-1070Intravenous GMI-1070 given as two doses over the course of one day | Drug | GMI-1070 |
Primary outcome
Safety as Measured by the Number of Participants With Adverse Events
Time frame: 28 days
4 secondary outcomes
Total Plasma Clearance
Time frame: 48 hours
Volume of the Central Compartment
Time frame: 48 hours
Intercompartmental Clearance
Time frame: 48 hours
Volume of the Peripheral Compartment
Time frame: 48 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 24988449Wun T, Styles L, DeCastro L, Telen MJ, Kuypers F, Cheung A, Kramer W, Flanner H, Rhee S, Magnani JL, Thackray H. Phase 1 study of the E-selectin inhibitor GMI 1070 in patients with sickle cell anemia. PLoS One. 2014 Jul 2;9(7):e101301. doi: 10.1371/journal.pone.0101301. eCollection 2014.
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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.
Helen Thackray, MD study director
GlycoMimetics Incorporated
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GMI-1070 in people with Sickle Cell Disease, run by GlycoMimetics Incorporated at 3 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 6 inclusion and 4 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 11 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 combined 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.
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 3 May 2013). 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?
Safety as Measured by the Number of Participants With Adverse Events (time frame: 28 days). 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 NCT00911495, 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 11 May 2020.