Treatment of Adult-Onset Immunodeficiency With Bortezomib

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT03103555
• Date of registration: 01/04/2017
• Prospective Registration: No
• Primary sponsor: Mahidol University
• Public title: Treatment of Adult-Onset Immunodeficiency With Bortezomib
• Scientific title: Treatment of Anti-Interferon-Gamma Autoantibody Associated Acquired Immunodeficiency Syndrome With Bortezomib: Pilot Study
• Date of first enrolment: February 27, 2017
• Target sample size: 5
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03103555
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Thailand

Contacts

• Name: Nasikarn Angkasekwinai, MD

• Name: Nasikarn Angkasekwinai, MD.

• Name: Nasikarn Angkasekwinai, MD.
• Affiliation: Faculty of Mdicine Siriraj Hospital, Mahidol University

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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



Age minimum: 21 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Adult-onset Immunodeficiency

Intervention(s)

Drug: Bortezomib

Drug: Cyclophosphamide

Primary Outcome(s)

Adverse event [Time Frame: 6 months after first dose of bortezomib (after completion 4 months of cyclophosphamide)]

Titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide [Time Frame: 1 year after first dose of bortezomib]

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)]

Secondary Outcome(s)

Disease relapse [Time Frame: 6 month and 1 year after first dose of bortezomib]

Secondary ID(s)

Treatment AOI with Bortezomib

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ministry of Health, Thailand