Sickle Cell DiseasePhase 1/2NCT00773890
TRF-1101 Assessment in Sickle Cell Disease
A Phase 1/2 study testing TRF-1101 in people with Sickle Cell Disease, run by TRF Pharma, Inc at 6 sites in United States.
Trial summary
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- TRF-1101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 6 sitesUnited States
- Study dates
- Jul 2008to May 2009
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be 18 years of age or older at the time of informed consent;
- Have a diagnosis of homozygous sickle cell anemia or sickle cell-beta° thalassemia;
- Have had 2 - 10 documented pain crises in the past year (pain crises are defined as visits to a medical clinic, Emergency Department or hospital, being bedridden and requiring constant analgesia at home for at least three days, or having a three-day interruption of life's activities [i.e., school, work, planned leisure activity] because of pain);
- If female and of child bearing potential, have a negative serum or urine pregnancy test and be using an effective birth-control method with a history of reliability for the individual patient (use of mifepristone is not allowed);
- Be properly informed of the nature and risks of the clinical investigation, be willing and able to comply with all clinical investigation-related procedures and assessments, and sign an Institutional Review Board (IRB) approved Informed Consent Form prior to entering the clinical investigation.
Who cannot join
- Have a history of abnormal bleeding, stroke, moya moya vascular malformations, or any other contraindication to anticoagulation;
- Be currently taking anticoagulant or thrombolytic medication;
- Be currently taking an endothelin receptor antagonist, e.g., bosentan (Tracleer®);
- Have a known sensitivity or allergy to heparin or related drugs;
- Have a history of thrombocytopenia (platelet count < 100 x 103/mm3) induced by heparin or related drugs;
- Have had fewer than 2 documented pain crises in the past year;
Show all 19Show fewer
- Have had a pain crisis within one month of screening or randomization;
- If currently on or recently discontinued hydroxyurea treatment, have initiated or discontinued treatment or changed regimen within the past 6 months;
- Have had a transfusion within last 120 days or have HbA% > 15% from prior transfusion;
- Creatinine levels > 1.53 mg/dL (135 umol/L);
- ALT levels ≥ 3 times normal;
- Platelet count < 100 x 103/mm3;
- INR > 2.0;
- Be unable to tolerate oral medications;
- Have unreliable venous access;
- Be noncompliant with regular care;
- Have a positive pregnancy test, be currently lactating, or be trying to become pregnant;
- Have participation in an investigational drug or medical device study within previous 30 days;
- Have any other condition or circumstance that in the opinion of the Investigator makes the patient a poor candidate for participation in the study.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Medical College of Georgia
Augusta, Georgia, 30912
Boston Medical Center
Boston, Massachusetts, 02118
University of North Carolina
Chapel Hill, North Carolina, 27599
University of Illinios Medical Center
Chicago, Illinois, 60612
Wayne State University Medical Center
Detroit, Michigan, 48201
Howard University
Washington D.C., District of Columbia, 20060
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Activity of TRF-1101 on Microvascular Blood Flow, Vascular Endothelial Injury, and Vasoocclusive Pain in Patients With Sickle Cell Disease
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
- Conditions
- Sickle Cell Disease
- Being tested
- TRF-1101, Placebo
- 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.
TRF-1101Drug
once daily treatment with 300 mg orally for 12 weeks
PlaceboDrug
Daily treatment with TRF-1101 vehicle for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| TRF-1101 | Experimental | Daily treatment with TRF-1101 Receives: Drug: TRF-1101 |
| Placebo | Placebo comparator | Daily treatment with placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
endothelial cell injury/inflammation
Measured: Throughout trial
Secondary outcomes (1)
Microvascular blood flow and trends in frequency of vasoocclusive pain
Measured: throughout trial
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2008 (estimated)
- Study startJul 2008
- Primary completionMay 2009
- Study completionMay 2009
Why it stopped: Study was stopped due to perceived futility because the baseline pain score in first 40 patients was too low to be able demonstrate an improvement
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- TRF Pharma, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Stephen H Embury, M.D., Study director, TRF Pharma, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00773890
- Sponsor's protocol ID
- 1101-201
- Status last verified
- May 2009
- First submitted
- 14 Oct 2008
- Last updated
- 11 May 2009
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Activity of TRF-1101 on Microvascular Blood Flow, Vascular Endothelial Injury, and Vasoocclusive Pain in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00773890
- Acronym / sponsor ID
- 1101-201
- Conditions
- Sickle Cell Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was stopped due to perceived futility because the baseline pain score in first 40 patients was too low to be able demonstrate an improvement
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- TRF Pharma, IncIndustry (pharma, biotech, devices)
- Study start
- Jul 2008
- Primary completion
- May 2009 (actual)
- Study completion
- May 2009 (actual)
- First posted
- 16 Oct 2008 (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.
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| TRF-1101 | Experimental | Drug: TRF-1101Daily treatment with TRF-1101 |
| Placebo | Placebo comparator | Drug: PlaceboDaily treatment with placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Be 18 years of age or older at the time of informed consent;
- Have a diagnosis of homozygous sickle cell anemia or sickle cell-beta° thalassemia;
- Have had 2 - 10 documented pain crises in the past year (pain crises are defined as visits to a medical clinic, Emergency Department or hospital, being bedridden and requiring constant analgesia at home for at least three days, or having a three-day interruption of life's activities [i.e., school, work, planned leisure activity] because of pain);
- If female and of child bearing potential, have a negative serum or urine pregnancy test and be using an effective birth-control method with a history of reliability for the individual patient (use of mifepristone is not allowed);
- Be properly informed of the nature and risks of the clinical investigation, be willing and able to comply with all clinical investigation-related procedures and assessments, and sign an Institutional Review Board (IRB) approved Informed Consent Form prior to entering the clinical investigation.
Key exclusion criteria (19 listed)
- Have a history of abnormal bleeding, stroke, moya moya vascular malformations, or any other contraindication to anticoagulation;
- Be currently taking anticoagulant or thrombolytic medication;
- Be currently taking an endothelin receptor antagonist, e.g., bosentan (Tracleer®);
- Have a known sensitivity or allergy to heparin or related drugs;
- Have a history of thrombocytopenia (platelet count < 100 x 103/mm3) induced by heparin or related drugs;
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 |
|---|---|---|
| TRF-1101once daily treatment with 300 mg orally for 12 weeks | Drug | TRF-1101 |
| PlaceboDaily treatment with TRF-1101 vehicle for 12 weeks | Drug | Placebo |
Primary outcome
endothelial cell injury/inflammation
Time frame: Throughout trial
1 secondary outcome
Microvascular blood flow and trends in frequency of vasoocclusive pain
Time frame: throughout trial
Results and safety
The study was stopped early (reason given: Study was stopped due to perceived futility because the baseline pain score in first 40 patients was too low to be able demonstrate an improvement). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2009.
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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.
Stephen H Embury, M.D. study director
TRF Pharma, Inc
6 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 TRF-1101 in people with Sickle Cell Disease, run by TRF Pharma, Inc at 6 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 5 inclusion and 19 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: stopped early, as of the sponsor's last update on 11 May 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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. 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 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?
The study was stopped early (reason given: Study was stopped due to perceived futility because the baseline pain score in first 40 patients was too low to be able demonstrate an improvement). Results are not available in the source record.
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?
endothelial cell injury/inflammation (time frame: Throughout trial). 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 11 May 2009.