Sickle Cell AnemiaPhase 3NCT00004408
Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease
A Phase 3 study testing poloxamer 188 in people with Sickle Cell Anemia, run by Mast Therapeutics, Inc..
Trial summary
OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease. II. Assess the effect of poloxamer 188 on duration and intensity of pain, total analgesic use, and length of hospitalization of these patients. Read more about this study
- Who is it for?
- Ages 10 to 65 years
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- poloxamer 188
- How many people
- 300 participants
- Where
- No sites listed yet
- Study dates
- Nov 1997to Nov 1999
- Phase
- Phase 3
- 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 10 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Sickle cell disease confirmed by electrophoresis or high pressure liquid chromatography
- At least one prior documented painful crisis episode but no greater than 10 crises per year for the last two years
- Sudden onset of acute pain lasting 4-12 hours and involving at least one site
- Severe crisis pain that requires parenteral analgesics and hospitalization, but not within the preceding 2 weeks
--Prior/Concurrent Therapy--
- Surgery: At least 2 weeks since prior major surgery No concurrent surgery
- Other: At least 2 weeks since prior puncture of noncompressible vessels No prior therapy using poloxamer 188 No concurrent investigational drug No concurrent use of nonsteroidal anti-inflammatory drugs
--Patient Characteristics--
- Hematopoietic: No significant bleeding or bleeding disorder
- Hepatic: ALT no greater than 2 times normal
- Renal: No active renal disease Creatinine no greater than 1.0 mg/dL OR Creatinine clearance greater than 50 mL/min Protein less than 300 mg/dL
- Cardiovascular: No evidence of acute myocardial ischemia or infarction
- Neurologic: At least 6 months since prior cerebrovascular accident or seizure
- Other: Not pregnant Fertile patients must use effective contraception during and for at least 30 days after treatment No history of chronic bacterial osteomyelitis No history of drug or alcohol abuse At least 6 months since prior use of illicit drug Have adequate IV access No crisis with life-threatening complications such as: Hepatic or splenic sequestration Acute chest syndrome Aplastic crisis No known infection or infection with encapsulated organism No evidence of septic shock Not concurrently hospitalized for other conditions Not concurrently on hypertransfusion program
About this study
OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease.
II. Assess the effect of poloxamer 188 on duration and intensity of pain, total analgesic use, and length of hospitalization of these patients.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, multicenter study. Patients are stratified according to hydroxyurea use.
Patients are randomized to treatment poloxamer 188 or placebo. Treatment begins within 12 hours of presentation with crisis. Patients receive poloxamer 188 or placebo by continuous infusion for 48 hours. Pain is assessed before, during, and after treatment.
Patients are followed on days 7-14 and 28-35.
- Conditions
- Sickle Cell Anemia
- Being tested
- poloxamer 188
- Keywords
- disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, pain, rare disease, sickle cell anemia
- Medical subject headings
- Anemia, Sickle Cell, Genetic Diseases, Inborn, Hematologic Diseases, Pain, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
poloxamer 188Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startNov 1997
- Study completionNov 1999
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 Sickle Cell AnemiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
- Enrolment
- 300 participants
Sponsor and investigators
- Lead sponsor
- Mast Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- CytRx
- Responsible party
- Sponsor
- Investigators
- R. Martin Emanuele, Study chair, CytRx
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004408
- Sponsor's protocol ID
- 199/13296
- Other IDs
- CYTRX-C97-1248, CYTRX-FDR001433
- Status last verified
- Mar 2012
- First submitted
- 18 Oct 1999
- Last updated
- 6 Mar 2012
- Results posted
- No
Publications
Adams-Graves P, Kedar A, Koshy M, Steinberg M, Veith R, Ward D, Crawford R, Edwards S, Bustrack J, Emanuele M. RheothRx (poloxamer 188) injection for the acute painful episode of sickle cell disease: a pilot study. Blood. 1997 Sep 1;90(5):2041-6.
PubMed 9292541
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00004408
- Acronym / sponsor ID
- 199/13296
- Conditions
- Sickle Cell Anemia
- Keywords
- disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, pain, rare disease, sickle cell anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 300 participants
- Lead sponsor
- Mast Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- CytRx
- Study start
- Nov 1997
- Study completion
- Nov 1999 (actual)
- First posted
- 19 Oct 1999 (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.
OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease.
II. Assess the effect of poloxamer 188 on duration and intensity of pain, total analgesic use, and length of hospitalization of these patients.
Detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, multicenter study. Patients are stratified according to hydroxyurea use.
Patients are randomized to treatment poloxamer 188 or placebo. Treatment begins within 12 hours of presentation with crisis. Patients receive poloxamer 188 or placebo by continuous infusion for 48 hours. Pain is assessed before, during, and after treatment.
Patients are followed on days 7-14 and 28-35.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
Who may qualify
- Ages
- 10 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| poloxamer 188 | Drug | – |
Primary outcome
Not reported in the source record.
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 6 Mar 2012.
- Background: PubMed 9292541Adams-Graves P, Kedar A, Koshy M, Steinberg M, Veith R, Ward D, Crawford R, Edwards S, Bustrack J, Emanuele M. RheothRx (poloxamer 188) injection for the acute painful episode of sickle cell disease: a pilot study. Blood. 1997 Sep 1;90(5):2041-6.
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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.
R. Martin Emanuele study chair
CytRx
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing poloxamer 188 in people with Sickle Cell Anemia, run by Mast Therapeutics, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 6 Mar 2012. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00004408, 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 6 Mar 2012.