Adult-onset ImmunodeficiencyPhase 2NCT03103555
Treatment of Adult-Onset Immunodeficiency With Bortezomib
A Phase 2 study testing Bortezomib and Cyclophosphamide in people with Adult-onset Immunodeficiency, run by Mahidol University at 1 site in Thailand.
Trial summary
This is pilot study aimed to investigate the application of proteosome inhibitor, Bortezomib in treatment of patients with neutralizing autoantibody to IFN-γ. The investigators hypothesis is that bortezomib will reduce the antibody level and restore interferon-gamma function, resulting in clinical improvement and… Read more about this study
- Who is it for?
- Ages 21 to 60 years
- Conditions the study is for
- Adult-onset Immunodeficiency
- What is being tested?
- Bortezomib and Cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteThailand
- Study dates
- 27 Feb 2017to 1 Nov 2019
- 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 21 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age 21 - 60 years
- Positive anti-interferon-gamma autoantibody
- Anti-HIV negative
- Past or current infection with opportunistic infection (OI) such as nontuberculous mycobacteria, proven by culture
- Ability to give written consent, informed written consent
- Negative pregnancy test in premenopausal woman
- Receving antimicrobial for treatment of OI for at least one month.
Who cannot join
- Pregnancy or lactation
- Absolute neutrophil count <1.5 × 109/ L or platelet count <100× 109/ L or hemoglobin level < 8 g/dL
- Past history of myocardial infarction or heart failure within 6 months before enrollment or prolonged QT interval > 450 msec at screening
- Renal insufficiency (GFR < 30 ml/min)
- Abnormal liver function test (AST> 3 times of UNL)
- Known cancer or receiving other immunosuppressive agent
- Known intolerability to Bortezomib
Trial locations and contacts
1 site in 1 country.
Thailand1 site, 1 recruiting
- Recruiting
Faculty of Medicine Siriraj Hospital, Mahidol University
Bangkok, Bangkok, 10700
Nasikarn Angkasekwinai, MD, 66818708766, nasikarn@gmail.com
Yupin =Suputtamongkol, MD.
About this study
Official title: Treatment of Anti-Interferon-Gamma Autoantibody Associated Acquired Immunodeficiency Syndrome With Bortezomib: Pilot Study
This is pilot study aimed to investigate the application of proteosome inhibitor, Bortezomib in treatment of patients with neutralizing autoantibody to IFN-γ. The investigators hypothesis is that bortezomib will reduce the antibody level and restore interferon-gamma function, resulting in clinical improvement and should be well-tolerated and safe for use in patients with autoantibody to IFN-γ.
Read the detailed description
The patients with autoantibody to IFN-γ and past or current history of proven opportunistic infection will receive 2 treatment cycles of bortezomib subcutaneously (4 injections of 1.3 mg Bortezomib /m2 body surface per cycle), followed by low dose oral cyclophosphamide for 4 month after completion treatment with bortezomib.
- Conditions
- Adult-onset Immunodeficiency
- Being tested
- Bortezomib, Cyclophosphamide
- Keywords
- Anti-interferon gamma autoantibody
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BortezomibDrug
Also called Velcade
Bortezomib will be subcutaneously administered in 2 treatment cycles with 4 injections of 1.3 mg Bortezomib /m2 body surface per cycle.
CyclophosphamideDrug
Also called Endoxan
Low dose cyclophosphamide will be given orally for 4 month after completion 2 cycles of bortezomib
| Study group | Role | What they receive |
|---|---|---|
| Bortezomib and cyclophosphamide | Experimental | Two cycles of bortezomib administered subcutaneously, followed by 4 months of low dose oral cyclophosphamide. Receives: Drug: Bortezomib; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Titers of anti-interferon-gamma antibody after treatment with bortezomib
Change in titers of anti-interferon-gamma antibody after treatment with
Measured: 8 weeks after first dose of bortezomib (after completion 2 cycles of bortezomib)
Titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
Change in titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
Measured: 1 year after first dose of bortezomib
Adverse event
Grade 4 adverse event (probably related)
Measured: 6 months after first dose of bortezomib (after completion 4 months of cyclophosphamide)
Secondary outcomes (1)
Disease relapse
Worsening of symptoms and signs
Measured: 6 month and 1 year after first dose of bortezomib
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2017
- Study start27 Feb 2017
- Primary completion1 Nov 2018 (estimated)
- Study completion1 Nov 2019 (estimated)
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 Adult-onset ImmunodeficiencyStudies of the same condition, open and under way.
- More trials run by Mahidol 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mahidol University
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, Thailand
- Responsible party
- Sponsor
- Investigators
- Nasikarn Angkasekwinai, MD., Principal investigator, Faculty of Mdicine Siriraj Hospital, Mahidol University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03103555
- Sponsor's protocol ID
- Treatment AOI with Bortezomib
- Status last verified
- Apr 2017
- First submitted
- 1 Apr 2017
- Last updated
- 6 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Angkasekwinai N, Suputtamongkol Y, Tantibhedhyangkul W, Onlamoon N, Phoompoung P, Pithukpakorn M, Karuphong E, Pusuwan P, Angkasekwinai P. Efficacy of Bortezomib for Treating Anti-Interferon-Gamma Autoantibody-Associated Adult-Onset Immunodeficiency Syndrome. Clin Infect Dis. 2024 Apr 10;78(4):1033-1042. doi: 10.1093/cid/ciad676.
PubMed 37947190
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Anti-Interferon-Gamma Autoantibody Associated Acquired Immunodeficiency Syndrome With Bortezomib: Pilot Study
- ClinicalTrials.gov ID
- NCT03103555
- Acronym / sponsor ID
- Treatment AOI with Bortezomib
- Conditions
- Adult-onset Immunodeficiency
- Keywords
- Anti-interferon gamma autoantibody
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Mahidol UniversityUniversity, hospital or other
- Collaborators
- Ministry of Health, Thailand
- Study start
- 27 Feb 2017 (actual)
- Primary completion
- 1 Nov 2018 (estimated)
- Study completion
- 1 Nov 2019 (estimated)
- First posted
- 6 Apr 2017 (actual)
- 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 is pilot study aimed to investigate the application of proteosome inhibitor, Bortezomib in treatment of patients with neutralizing autoantibody to IFN-γ. The investigators hypothesis is that bortezomib will reduce the antibody level and restore interferon-gamma function, resulting in clinical improvement and should be well-tolerated and safe for use in patients with autoantibody to IFN-γ.
Detailed description
The patients with autoantibody to IFN-γ and past or current history of proven opportunistic infection will receive 2 treatment cycles of bortezomib subcutaneously (4 injections of 1.3 mg Bortezomib /m2 body surface per cycle), followed by low dose oral cyclophosphamide for 4 month after completion treatment with bortezomib.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bortezomib and cyclophosphamide | Experimental | Drug: Bortezomib, Drug: CyclophosphamideTwo cycles of bortezomib administered subcutaneously, followed by 4 months of low dose oral cyclophosphamide. |
Who may qualify
- Ages
- 21 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Age 21 - 60 years
- Positive anti-interferon-gamma autoantibody
- Anti-HIV negative
- Past or current infection with opportunistic infection (OI) such as nontuberculous mycobacteria, proven by culture
- Ability to give written consent, informed written consent
Key exclusion criteria (7 listed)
- Pregnancy or lactation
- Absolute neutrophil count <1.5 × 109/ L or platelet count <100× 109/ L or hemoglobin level < 8 g/dL
- Past history of myocardial infarction or heart failure within 6 months before enrollment or prolonged QT interval > 450 msec at screening
- Renal insufficiency (GFR < 30 ml/min)
- Abnormal liver function test (AST> 3 times of UNL)
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 |
|---|---|---|
| BortezomibAlso: VelcadeBortezomib will be subcutaneously administered in 2 treatment cycles with 4 injections of 1.3 mg Bortezomib /m2 body surface per cycle. | Drug | Bortezomib and cyclophosphamide |
| CyclophosphamideAlso: EndoxanLow dose cyclophosphamide will be given orally for 4 month after completion 2 cycles of bortezomib | Drug | Bortezomib and cyclophosphamide |
Primary outcome
Titers of anti-interferon-gamma antibody after treatment with bortezomib
Time frame: 8 weeks after first dose of bortezomib (after completion 2 cycles of bortezomib)
Change in titers of anti-interferon-gamma antibody after treatment with
Titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
Time frame: 1 year after first dose of bortezomib
Change in titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
Adverse event
Time frame: 6 months after first dose of bortezomib (after completion 4 months of cyclophosphamide)
Grade 4 adverse event (probably related)
1 secondary outcome
Disease relapse
Time frame: 6 month and 1 year after first dose of bortezomib
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2017.
- Derived from this study: PubMed 37947190Angkasekwinai N, Suputtamongkol Y, Tantibhedhyangkul W, Onlamoon N, Phoompoung P, Pithukpakorn M, Karuphong E, Pusuwan P, Angkasekwinai P. Efficacy of Bortezomib for Treating Anti-Interferon-Gamma Autoantibody-Associated Adult-Onset Immunodeficiency Syndrome. Clin Infect Dis. 2024 Apr 10;78(4):1033-1042. doi: 10.1093/cid/ciad676.
- Individual participant dataSharing planned: No
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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.
Nasikarn Angkasekwinai, MD. principal investigator
Faculty of Mdicine Siriraj Hospital, Mahidol University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bortezomib and Cyclophosphamide in people with Adult-onset Immunodeficiency, run by Mahidol University at 1 site in Thailand.
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 7 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: status not confirmed, as of the sponsor's last update on 6 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Thailand. 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Nasikarn Angkasekwinai, MD. (6681-8708766, nasikarn@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Titers of anti-interferon-gamma antibody after treatment with bortezomib (time frame: 8 weeks after first dose of bortezomib (after completion 2 cycles of bortezomib)). The study lists 3 primary outcomes in all. 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 NCT03103555, 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 Apr 2017.