Treatment of Adult-Onset Immunodeficiency With Bortezomib

Treatment of Anti-Interferon-Gamma Autoantibody Associated Acquired Immunodeficiency Syndrome With Bortezomib: Pilot Study

Status

UNKNOWN

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03103555

Enrollment

Registered

2017-04-06

Start date

2017-02-27

Completion date

2019-11-01

Last updated

2017-04-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult-onset Immunodeficiency

Keywords

Anti-interferon gamma autoantibody

Brief 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 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.

Interventions

DRUGBortezomib

Bortezomib will be subcutaneously administered in 2 treatment cycles with 4 injections of 1.3 mg Bortezomib /m2 body surface per cycle.

DRUGCyclophosphamide

Low dose cyclophosphamide will be given orally for 4 month after completion 2 cycles of bortezomib

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

21 Years to 60 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Titers of anti-interferon-gamma antibody after treatment with bortezomib	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	1 year after first dose of bortezomib	Change in titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
Adverse event	6 months after first dose of bortezomib (after completion 4 months of cyclophosphamide)	Grade 4 adverse event (probably related)

Secondary

Measure	Time frame	Description
Disease relapse	6 month and 1 year after first dose of bortezomib	Worsening of symptoms and signs

Countries

Thailand

Contacts

Primary ContactNasikarn Angkasekwinai, MD.

nasikarn@gmail.com6681-8708766

Backup ContactYupin Suputtamongkol, MD.

ysuputtamongkol@gmail.com662-4197783

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 8, 2026