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Disease

Agammaglobulinemia

Late-stage therapeutic developmentEmerging researchRising momentum
MeSHAgammaglobulinemia
3
Publications
9
Clinical trials
1
Related conditions
2
Related proteins
2025
Latest publication
Current focus
Therapeutic developmentGenetics & risk factorsDisease mechanisms & pathology
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

BTK drives neutrophil activation for sterilizing antifungal immunity.

Research2024-05-02The Journal of clinical investigation

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

Research2021-05-11The New England journal of medicine

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q3 2026.
  • Active recent publication activity.
Upcoming Milestones
Major developments
Upcoming trial readoutImportant
V-IMMUNE® for Primary Immunodeficiency: A Phase III Clinical Trial (VIP Study)
Results expected Q3 20262026-01-20
Research Highlights1View
Clinical Milestones1View
Results expected 1
Activity timeline2

Clinical trials

8 sponsors · 1 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
9
All trials
2
Active
6
Late-stage
6
Completed
Late-stage studies

V-IMMUNE® for Primary Immunodeficiency: A Phase III Clinical Trial (VIP Study)

Phase 3 · Recruiting · On Pharma Importadora, Exportadora e Distribuidora de Medicamentos LTDA.

A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID)

Phase 3 · Completed · CSL Behring

Multicenter, Open-label, Historically Controlled, Phase III Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Kedrion IVIG 10% in Adult and Pediatric Subjects With Primary Immunodeficiency (PID).

Phase 3 · Completed · Kedrion S.p.A.

A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency

Phase 3 · Completed · CSL Behring

IGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient Patients

Phase 3 · Completed · Grifols Therapeutics LLC

A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents

Phase 4 · Completed · Bio Products Laboratory
Recruiting

V-IMMUNE® for Primary Immunodeficiency: A Phase III Clinical Trial (VIP Study)

Phase 3 · Recruiting · On Pharma Importadora, Exportadora e Distribuidora de Medicamentos LTDA.
Recently completed

Multicenter, Open-label, Historically Controlled, Phase III Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Kedrion IVIG 10% in Adult and Pediatric Subjects With Primary Immunodeficiency (PID).

Phase 3 · Completed · Kedrion S.p.A.

A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents

Phase 4 · Completed · Bio Products Laboratory

A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency

Phase 3 · Completed · CSL Behring

A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID)

Phase 3 · Completed · CSL Behring

Prospective Open-Label Study of Pharmacokinetics, Efficacy and Safety of Immune Globulin Intravenous (Human), 10% TVR Solution in Patients With Hypo- or Agammaglobulinemia

Phase 2 · Completed · Baxalta now part of Shire

IGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient Patients

Phase 3 · Completed · Grifols Therapeutics LLC

Research activity

3 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20212025
Most influential

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

The New England journal of medicine · 2021 · 153 cites

BTK drives neutrophil activation for sterilizing antifungal immunity.

The Journal of clinical investigation · 2024 · 24 cites
Major themes8
  • Agammaglobulinaemia Tyrosine Kinase1
  • Agammaglobulinemia1
  • Aspergillosis1
  • Genetic Diseases, X-Linked1
  • Genetic Vectors1
  • Hematopoietic Stem Cell Transplantation1
  • Neurodegenerative Diseases1
  • Neutrophil Activation1
Leading journals3
  • Frontiers in immunology1
  • The Journal of clinical investigation1
  • The New England journal of medicine1
Leading researchers8
  • Adams S1
  • Arduini S1
  • Aufiero MA1
  • Barman P1
  • Bergerson JR1
  • Booth C1
  • Buckland KF1
  • Buckley RH1
Affiliations (unnormalised)6
  • and Pathology and Laboratory Medicine (G.M.C.) and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research (D.B.K.1
  • Beijing Children's Hospital1
  • Centre de Recherche sur l'Inflammation1
  • Children's Hospital Affiliated to Zhengzhou University1
  • Department of Clinical Microbiology and Microbial Pathogenesis1
  • Geisel School of Medicine at Dartmouth1

Disease biology

2 matches

Key proteins & gene products studied in this disease. Number shows shared papers.

Agammaglobulinaemia Tyrosine Kinase2Janus Kinases2

Related conditions

1 match

Diseases frequently studied alongside this one. Number shows shared papers.

Genetic Diseases, X-Linked2

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

An immunologic deficiency state characterized by an extremely low level of generally all classes of gamma-globulin in the blood.

Identifiers
MeSHAgammaglobulinemia
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.