Diabetes Mellitus, Type 1Phase 2BRiTENCT00737763
Beta Cell Rescue in New Onset Type 1 Diabetes With Efalizumab
A Phase 2 study testing efalizumab in people with Diabetes Mellitus, Type 1, run by Emory University.
Trial summary
In this single-center therapeutic study, we will study the ability of efalizumab to protect remaining beta cells in teenagers and young adults who have been newly diagnosed with type 1 diabetes mellitus. Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- Diabetes Mellitus, Type 1
- What is being tested?
- efalizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2008to Nov 2009
- 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 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males or females 12-35 years old, no preference nor discrimination will be made based on ethnicity.
- Recent diagnosis of Type 1Diabetes Mellitus, participant can be enrolled in the trial within 6 weeks of diagnosis.
- Positive for at least one diabetes autoantibody. Insulin autoantibody positivity will only be used as a selection criterion if insulin has not been used in at least the preceding 10 days.
- Willingness to provide written informed consent (either the subject or the subject's legally authorized representative)
- Have routine diabetic care under an endocrinologist and ability to follow study protocol for the duration of the 2-year study.
- Although no preference or discrimination will be made based on ethnicity or gender, participants (and family and/or guardians when applicable) must demonstrate comprehension of the trial, including its obligations and potential risks.
- If a female of childbearing potential, a negative pregnancy test and commitment to the use of two forms of effective contraception or abstinence for the duration of the study are necessary.
- If a non-sterile male, commitment to the use of two forms of effective contraception (birth control) for the duration of the study is necessary.
Who cannot join
- Severe allergic allergy or anaphylaxis to human monoclonal antibodies
- Hospital admission for cardiac disease, stroke, or pulmonary disease within the past year
- History of substance abuse within last 5 years
- History of ongoing uncontrolled bacterial, viral, or fungal or atypical mycobacterium infections
- History of opportunistic infections
- Diagnosis with hepatic cirrhosis regardless of cause or severity
Show all 20Show fewer
- Diagnosis, history, or laboratory evidence of Hepatitis B or C infection
- Hepatic enzymes 2 > times the upper limit of normal
- History of active or treatment for tuberculosis or skin test positive
- History of malignancy over the past 5 years
- Recent initiation or change in treatment regimen of beta-blockers, angiotensin-converting enzyme inhibitors, interferons, quinidine anti-malarial drugs, or lithium in the past month
- Seropositivity for human immunodeficiency virus (HIV)
- Serologic or clinical evidence of recent or acute infection with Epstein-Barr Virus or Cytomegalovirus
- Females who are pregnant, lactating, or planning on pregnancy during the 2 year study period
- Progressive hearing loss
- History of organ or bone marrow transplantation, sickle cell disease, cystic fibrosis, autoimmune anemia, seizures, autoimmune thrombocytopenia, leuko/lymphopenia, vasculitis, other autoimmune disease.
- Current use of immunosuppressive medications
- Plan or requirement of receiving new immunization of any type within the first 12 months of the study, or booster or completion vaccines with live or live-attenuated vaccines
- Any condition that, in judgment of the investigator, could jeopardize the subject-safety following exposure to the drug.
- Participation in another simultaneous medical investigation or trial
About this study
Official title: Beta Cell Rescue in New Onset Type 1 Diabetes Mellitus With the LFA-1 Antibody Efalizumab
In this single-center therapeutic study, we will study the ability of efalizumab to protect remaining beta cells in teenagers and young adults who have been newly diagnosed with type 1 diabetes mellitus. Efalizumab is a monoclonal antibody which prevents the activation of antigen specific T lymphocytes to sites of inflammation. Efalizumab was approved by the FDA in 2003 for the treatment of psoriasis. It has been proven to be safe, well tolerated and effective in targeting T cell mediated disorders like those seen in autoimmunity.
Read the detailed description
Since there is data that shows that early intervention can prevent further destruction of insulin producing beta cells, the patients who will be enrolled in this study will have been diagnosed with Type 1 diabetes within 6 weeks of enrolling and starting therapy. Patients who meet the screening criteria will be randomized at a 2 to 1 ratio to either get weekly subcutaneous injections of efalizumab for 26 weeks versus a placebo injection. The researchers and patients will be blinded to the treatment group assignment. All patients will be followed for two years.
The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment. The Mixed Meal Tolerance test will help test the production of insulin by the pancreas. By comparing the results of these tests between the treated group and the placebo group, we hope to be able to show preservation of beta cell function in the group treated with efalizumab.
- Conditions
- Diabetes Mellitus, Type 1
- Being tested
- efalizumab, placebo
- Keywords
- efalizumab, Raptiva, Diabetes, Type 1 Diabetes Mellitus
- Medical subject headings
- Diabetes Mellitus, Type 1, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efalizumabDrug
Also called Raptiva
Enrollees randomized to efalizumab will receive the first dose of 0.7mg/kg subcutaneously given at enrollment, and 1.0 mg/kg subcutaneously weekly for 26 weeks self or family-administered after injection training. This is the FDA-approved initial and subsequent doses of efalizumab used for psoriasis treatment
placeboDrug
Also called none applicable
Enrollees receiving placebo will be given a subcutaneous injection of equal volume and appearance to treatment on the same schedule.
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | This group will receive weekly efalizumab injections for 6 months Receives: Drug: efalizumab |
| B | Placebo comparator | This group will receive placebo injections for 6 months Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2008 (estimated)
- Study startOct 2008
- Primary completionNov 2009
- Study completionNov 2009
Why it stopped: drug withdrawn from market
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetes Mellitus, Type 1Studies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation, Genentech, Inc.
- Responsible party
- Principal investigator, Mark Rigby, Emory University
- Investigators
- Mark R Rigby, MD, PhD, Principal investigator, Emory University, Children's Healthcare of AtlantaEric Felner, MD, Principal investigator, Children's Healthcare of Atlanta, Emory UniversitySol Jacobs, MD, Principal investigator, Emory UniversityChristian Larsen, MD, DPhil, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00737763
- Sponsor's protocol ID
- IRB0007365
- Other IDs
- BRiTE Trial for T1DM
- Acronym
- BRiTE
- Status last verified
- May 2014
- First submitted
- 19 Aug 2008
- Last updated
- 12 May 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Beta Cell Rescue in New Onset Type 1 Diabetes Mellitus With the LFA-1 Antibody Efalizumab
- ClinicalTrials.gov ID
- NCT00737763
- Acronym / sponsor ID
- BRiTE / IRB0007365
- Conditions
- Diabetes Mellitus, Type 1
- Keywords
- efalizumab, Raptiva, Diabetes, Type 1 Diabetes Mellitus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: drug withdrawn from market
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation, Genentech, Inc.
- Study start
- Oct 2008
- Primary completion
- Nov 2009 (actual)
- Study completion
- Nov 2009 (actual)
- First posted
- 20 Aug 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.
In this single-center therapeutic study, we will study the ability of efalizumab to protect remaining beta cells in teenagers and young adults who have been newly diagnosed with type 1 diabetes mellitus. Efalizumab is a monoclonal antibody which prevents the activation of antigen specific T lymphocytes to sites of inflammation. Efalizumab was approved by the FDA in 2003 for the treatment of psoriasis. It has been proven to be safe, well tolerated and effective in targeting T cell mediated disorders like those seen in autoimmunity.
Detailed description
Since there is data that shows that early intervention can prevent further destruction of insulin producing beta cells, the patients who will be enrolled in this study will have been diagnosed with Type 1 diabetes within 6 weeks of enrolling and starting therapy. Patients who meet the screening criteria will be randomized at a 2 to 1 ratio to either get weekly subcutaneous injections of efalizumab for 26 weeks versus a placebo injection. The researchers and patients will be blinded to the treatment group assignment. All patients will be followed for two years.
The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment. The Mixed Meal Tolerance test will help test the production of insulin by the pancreas. By comparing the results of these tests between the treated group and the placebo group, we hope to be able to show preservation of beta cell function in the group treated with efalizumab.
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 |
|---|---|---|
| A | Experimental | Drug: efalizumabThis group will receive weekly efalizumab injections for 6 months |
| B | Placebo comparator | Drug: placeboThis group will receive placebo injections for 6 months |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Males or females 12-35 years old, no preference nor discrimination will be made based on ethnicity.
- Recent diagnosis of Type 1Diabetes Mellitus, participant can be enrolled in the trial within 6 weeks of diagnosis.
- Positive for at least one diabetes autoantibody. Insulin autoantibody positivity will only be used as a selection criterion if insulin has not been used in at least the preceding 10 days.
- Willingness to provide written informed consent (either the subject or the subject's legally authorized representative)
- Have routine diabetic care under an endocrinologist and ability to follow study protocol for the duration of the 2-year study.
Key exclusion criteria (20 listed)
- Severe allergic allergy or anaphylaxis to human monoclonal antibodies
- Hospital admission for cardiac disease, stroke, or pulmonary disease within the past year
- History of substance abuse within last 5 years
- History of ongoing uncontrolled bacterial, viral, or fungal or atypical mycobacterium infections
- History of opportunistic infections
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 |
|---|---|---|
| efalizumabAlso: RaptivaEnrollees randomized to efalizumab will receive the first dose of 0.7mg/kg subcutaneously given at enrollment, and 1.0 mg/kg subcutaneously weekly for 26 weeks self or family-administered after injection training. This is the FDA-approved initial and subsequent doses of efalizumab used for psoriasis treatment | Drug | A |
| placeboAlso: none applicableEnrollees receiving placebo will be given a subcutaneous injection of equal volume and appearance to treatment on the same schedule. | Drug | B |
Primary outcome
The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment.
Time frame: 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 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.
Mark R Rigby, MD, PhD principal investigator
Emory University, Children's Healthcare of Atlanta
Eric Felner, MD principal investigator
Children's Healthcare of Atlanta, Emory University
Sol Jacobs, MD principal investigator
Emory University
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing efalizumab in people with Diabetes Mellitus, Type 1, run by Emory University.
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 20 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: withdrawn, as of the sponsor's last update on 12 May 2014. 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. 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 was withdrawn before enrolling participants, so there are no results.
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?
The primary endpoint for this study will be the difference from baseline in the body's ability to respond to a Mixed Meal Tolerance Test at 12 months after enrollment. (time frame: 2 years). 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 12 May 2014.