Alopecia Totalis/UniversalisPhase 2NCT01314495
Abatacept Costimulatory Blockade in the Treatment of Alopecia Totalis/Universalis
A Phase 2 study testing Abatacept and Inactive infusion in people with Alopecia Totalis/Universalis, run by Julian M. Mackay-Wiggan.
Trial summary
Will Abatacept reduce priming of the hair follicle specific T cells and thereby reduce hair follicle associated infiltration and improve hair growth. This is a double blind placebo controlled study to test the safety and efficacy of Abatacept in the treatment of 64 subjects diagnosed with alopecia totalis or alopecia… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Alopecia Totalis/Universalis
- What is being tested?
- Abatacept and Inactive infusion
- 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
- Sep 2011to Jun 2012
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be between 18 and 75 years of age.
- Must have a diagnosis of alopecia totalis or alopecia universalis
- Must have >75% total scalp hair loss at baseline as measured using the SALT score.
- Duration of hair loss must be between 3 to 12 months.
- There may be no evidence of regrowth present at baseline.
- Subjects may be naïve to treatment or unresponsive to intralesional (IL) steroids or other treatments for alopecia areata.
- Must be willing to avoid live vaccines while on the study medication, and within 3 months of its discontinuation.
Who cannot join
- Patients with a history of or active skin disease on the scalp such as psoriasis or seborrheic dermatitis.
- Patients in whom the diagnosis of alopecia areata is in question.
- Patients with active medical conditions or malignancies (except adequately treated basal or squamous cell carcinoma) that in the opinion of the investigator would increase the risks associated with study participation, including patients with a history of recurrent infections.
- Patients who monitor their blood glucose levels using glucose dehydrogenase pyrrologuinolinequinone (GDH-PQQ) test strips. Blood glucose monitoring using other methods that do not react with maltose, such as the glucose dehydrogenase nicotine adenine dinucleotide (GDH-NAD), glucose oxidase, or glucose hexokinase test methods are permitted.
- Patients taking TNF antagonists or other biological therapy such as anakinra.
- Women of childbearing potential who are unable or unwilling to use two forms of birth control for the study duration.
Show all 19Show fewer
- Women who are pregnant or nursing.
- Patients known to be HIV or hepatitis B or C positive.
- Hepatitis B surface antigen positive or Hepatitis B core antibody positive at screening.
- Patients with latent Mycobacterium tuberculosis infection as indicated by a positive Purified Protein Derivative [PPD] skin test. Subjects with a positive PPD skin test and documented completion of treatment per standard medical practice for latent TB are eligible. Subjects with a positive PPD skin test and not treated or no documentation of completion of treatment are ineligible.
- History of incompletely treated Mycobacterium tuberculosis infection as indicated by:
- Subject's medical records documenting incomplete treatment for Mycobacterium tuberculosis
- Subject's self-reported history of incomplete treatment for Mycobacterium tuberculosis
- Patients with evidence of infection or skin cancer in the treated areas.
- Patients with history or evidence of hematopoietic abnormality.
- Patients with history of immunosuppression or history of recurrent serious infections.
- Patients with a history or likely diagnosis of COPD
- Patients unwilling or unable to discontinue treatments known to affect hair regrowth in alopecia areata.
- Patients who have been treated with intralesional steroids, systemic steroids, anthralin, squaric acid, DPCP (diphenylcycloprophenone), protopic, minoxidil or other medication which in the opinion of the investigator may affect hair regrowth within one month of the baseline visit.
About this study
Official title: A Randomized, Double Blind, Placebo Controlled Clinical Trial to Evaluate the Efficacy of Abatacept Costimulatory Blockade in the Treatment of Alopecia Totalis/Universalis
Will Abatacept reduce priming of the hair follicle specific T cells and thereby reduce hair follicle associated infiltration and improve hair growth.
This is a double blind placebo controlled study to test the safety and efficacy of Abatacept in the treatment of 64 subjects diagnosed with alopecia totalis or alopecia universalis. Subjects will be randomized 1:1 to the placebo or treatment arm and will receive 6 months of treatment with the study medication or placebo, followed by a 6 month observational period.
Read the detailed description
Alopecia Areata is a common autoimmune disease, affecting 1% of the general population resulting from autoimmune attack on the hair follicles and usually presents with patchy hair loss. One third of these patients will experience spontaneous remissions within the first year. However many patients will develop waxing and waning disease with some progressing to alopecia totalis (total scalp hair loss) or alopecia universalis (loss of all body hair). This population that suffers from a disfiguring disease represents a significant unmet medical need. Alopecia totalis/universalis seldom, if ever, remits spontaneously or with current treatment and is classified by the FDA as an Orphan Indication.
There is no FDA approved drug for alopecia areata. A recent Cochrane report concluded that there was no evidence based support for any intervention in this disease. Standard of care remains observation for mild disease and lesional/oral steroids for more advanced cases.
Abatacept is a soluble human fusion protein that selectively modulates the costimulatory signal required for full T-cell activation. It is approved for the treatment of moderately to severely active rheumatoid arthritis. It is also approved for the treatment of moderately to severely active polyarticular juvenile idiopathic arthritis in children 6 years of age or older. Abatacept is a lyophilized powder administered as a 30 minute intravenous infusion. Dosage, as in rheumatoid arthritis, is weight based and is fixed throughout the course of treatment. Abatacept or placebo will be administered as a 30 minute intravenous infusion at baseline, weeks 2, 4, and every 4 weeks for 5 cycles (weeks 8, 12, 16, 20) for a total treatment period of 6 months. There will be a 6 month observational period following the treatment period.
- Conditions
- Alopecia Totalis/Universalis
- Being tested
- Abatacept, Inactive infusion
- Keywords
- Alopecia Areata
- Medical subject headings
- Alopecia Areata
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AbataceptDrug
Abatacept will be administrated as a 30 minute intravenous infusion.
Inactive infusionDrug
Placebo will be administered as a 30 minute intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| Abatacept | Active comparator | Abatacept administered as a 30 minute intravenous infusion Receives: Drug: Abatacept |
| Inactive infusion | Placebo comparator | Placebo will be administered as a 30 minute intravenous infusion. Receives: Drug: Inactive infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
The study's primary endpoint will be the proportion of responders after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by Severity of Alopecia Tool (SALT) score at week 24.
The study's primary endpoint of this intent to treat trial will be the proportion of responders in the treated compared to the control group after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by Severity of Alopecia Tool (SALT) score at week 24. This is a relatively strict definition for defining responders and non-responders and was chosen to minimize responses in the 'vehicle' arm, in which fewer than 10% are expected to achieve this magnitude of hair re-growth spontaneously.
Measured: 24 weeks
Secondary outcomes (1)
Percent hair regrowth from baseline determined by SALT at weeks 4, 8, 12, 20, and 24 during treatment phase and at weeks 30, 36, 42 and 48 during the observational phase.
As secondary endpoints, efficacy will be measured by changes in hair re-growth as a continuous variable as determined by physical exam and photography, as well as subject and physician global evaluation scores. To assess the durability of responses, subjects will continue to be followed for an additional 6 months post treatment. All subjects will be assessed for safety at each study visit for incidence and severity of adverse effects (AE) and incidence of treatment emergent laboratory abnormalities.
Measured: 24 weeks of treatment and 18 weeks observational phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2011 (estimated)
- Study startSep 2011
- Primary completionJun 2012
- Study completionJun 2012
Why it stopped: Halt in funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alopecia Totalis/UniversalisStudies 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Julian M. Mackay-Wiggan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Julian M. Mackay-Wiggan, Columbia University
- Investigators
- Julian Mackay-Wiggan, MD, MS, Principal investigator, Columbia UniversityAngela Christiano, PhD, Study director, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01314495
- Sponsor's protocol ID
- AAAI1530
- Status last verified
- Nov 2012
- First submitted
- 11 Mar 2011
- Last updated
- 14 Nov 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Placebo Controlled Clinical Trial to Evaluate the Efficacy of Abatacept Costimulatory Blockade in the Treatment of Alopecia Totalis/Universalis
- ClinicalTrials.gov ID
- NCT01314495
- Acronym / sponsor ID
- AAAI1530
- Conditions
- Alopecia Totalis/Universalis
- Keywords
- Alopecia Areata
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Halt in funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Julian M. Mackay-WigganUniversity, hospital or other
- Study start
- Sep 2011
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 14 Mar 2011 (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.
Will Abatacept reduce priming of the hair follicle specific T cells and thereby reduce hair follicle associated infiltration and improve hair growth.
This is a double blind placebo controlled study to test the safety and efficacy of Abatacept in the treatment of 64 subjects diagnosed with alopecia totalis or alopecia universalis. Subjects will be randomized 1:1 to the placebo or treatment arm and will receive 6 months of treatment with the study medication or placebo, followed by a 6 month observational period.
Detailed description
Alopecia Areata is a common autoimmune disease, affecting 1% of the general population resulting from autoimmune attack on the hair follicles and usually presents with patchy hair loss. One third of these patients will experience spontaneous remissions within the first year. However many patients will develop waxing and waning disease with some progressing to alopecia totalis (total scalp hair loss) or alopecia universalis (loss of all body hair). This population that suffers from a disfiguring disease represents a significant unmet medical need. Alopecia totalis/universalis seldom, if ever, remits spontaneously or with current treatment and is classified by the FDA as an Orphan Indication.
There is no FDA approved drug for alopecia areata. A recent Cochrane report concluded that there was no evidence based support for any intervention in this disease. Standard of care remains observation for mild disease and lesional/oral steroids for more advanced cases.
Abatacept is a soluble human fusion protein that selectively modulates the costimulatory signal required for full T-cell activation. It is approved for the treatment of moderately to severely active rheumatoid arthritis. It is also approved for the treatment of moderately to severely active polyarticular juvenile idiopathic arthritis in children 6 years of age or older. Abatacept is a lyophilized powder administered as a 30 minute intravenous infusion. Dosage, as in rheumatoid arthritis, is weight based and is fixed throughout the course of treatment. Abatacept or placebo will be administered as a 30 minute intravenous infusion at baseline, weeks 2, 4, and every 4 weeks for 5 cycles (weeks 8, 12, 16, 20) for a total treatment period of 6 months. There will be a 6 month observational period following the treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Abatacept | Active comparator | Drug: AbataceptAbatacept administered as a 30 minute intravenous infusion |
| Inactive infusion | Placebo comparator | Drug: Inactive infusionPlacebo will be administered as a 30 minute intravenous infusion. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Must be between 18 and 75 years of age.
- Must have a diagnosis of alopecia totalis or alopecia universalis
- Must have >75% total scalp hair loss at baseline as measured using the SALT score.
- Duration of hair loss must be between 3 to 12 months.
- There may be no evidence of regrowth present at baseline.
Key exclusion criteria (19 listed)
- Patients with a history of or active skin disease on the scalp such as psoriasis or seborrheic dermatitis.
- Patients in whom the diagnosis of alopecia areata is in question.
- Patients with active medical conditions or malignancies (except adequately treated basal or squamous cell carcinoma) that in the opinion of the investigator would increase the risks associated with study participation, including patients with a history of recurrent infections.
- Patients who monitor their blood glucose levels using glucose dehydrogenase pyrrologuinolinequinone (GDH-PQQ) test strips. Blood glucose monitoring using other methods that do not react with maltose, such as the glucose dehydrogenase nicotine adenine dinucleotide (GDH-NAD), glucose oxidase, or glucose hexokinase test methods are permitted.
- Patients taking TNF antagonists or other biological therapy such as anakinra.
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 |
|---|---|---|
| AbataceptAbatacept will be administrated as a 30 minute intravenous infusion. | Drug | Abatacept |
| Inactive infusionPlacebo will be administered as a 30 minute intravenous infusion. | Drug | Inactive infusion |
Primary outcome
The study's primary endpoint will be the proportion of responders after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by Severity of Alopecia Tool (SALT) score at week 24.
Time frame: 24 weeks
The study's primary endpoint of this intent to treat trial will be the proportion of responders in the treated compared to the control group after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by Severity of Alopecia Tool (SALT) score at week 24. This is a relatively strict definition for defining responders and non-responders and was chosen to minimize responses in the 'vehicle' arm, in which fewer than 10% are expected to achieve this magnitude of hair re-growth spontaneously.
1 secondary outcome
Percent hair regrowth from baseline determined by SALT at weeks 4, 8, 12, 20, and 24 during treatment phase and at weeks 30, 36, 42 and 48 during the observational phase.
Time frame: 24 weeks of treatment and 18 weeks observational phase
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 14 Nov 2012.
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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.
Julian Mackay-Wiggan, MD, MS principal investigator
Columbia University
Angela Christiano, PhD study director
Columbia University
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Abatacept and Inactive infusion in people with Alopecia Totalis/Universalis, run by Julian M. Mackay-Wiggan.
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 7 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: withdrawn, as of the sponsor's last update on 14 Nov 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. 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 study's primary endpoint will be the proportion of responders after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by Severity of Alopecia Tool (SALT) score at week 24. (time frame: 24 weeks). 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 14 Nov 2012.