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Condition (MeSH)

Primary Immunodeficiency Diseases

ClinicalTrials.gov lists 538 trials for Primary Immunodeficiency Diseases or a narrower condition beneath it. Shire has the most among industry and government lead sponsors, with 39.

MeSH D000081207 Congenital / Hereditary / Neonatal C16 C20
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Phase 1 Phase 1 41 Phase 1/2 Phase 1/2 48 Phase 2 Phase 2 66 Phase 2/3 Phase 2/3 15 Phase 3 Phase 3 113 Phase 4 Phase 4 31 Not applicable Not applicable 40 Observational Observational 172 Phase 1 Phase 1 41 Phase 1/2 Phase 1/2 48 Phase 2 Phase 2 66 Phase 2/3 Phase 2/3 15 Phase 3 Phase 3 113 Phase 4 Phase 4 31 Not applicable Not applicable 40 Observational Observational 172

Trials per phase

1 more section with rows

210 of the 538 trials are tagged with Primary Immunodeficiency Diseases itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Primary Immunodeficiency Diseases trials start? (35)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 1 trial is registered to start in 2027 or later, and not listed here.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 33

1990: 1 trials (Observational 1) 1991: 1 trials (Phase 2 1) 1992: 1 trials (Phase 1/2 1) 1994: 1 trials (Not applicable 1) 1995: 4 trials (Observational 2, Not applicable 1, Phase 1 1) 1996: 1 trials (Phase 2 1) 1997: 2 trials (Not applicable 1, Phase 1 1) 1999: 2 trials (Phase 2 2) 2000: 8 trials (Observational 4, Not applicable 2, Phase 3 1, Phase 2/3 1) 2001: 4 trials (Observational 2, Phase 1 2) 2002: 11 trials (Observational 1, Not applicable 2, Phase 4 1, Phase 3 3, Phase 2/3 1, Phase 2 3) 2003: 1 trials (Phase 2 1) 2004: 5 trials (Observational 2, Phase 3 2, Phase 1 1) 2005: 14 trials (Observational 3, Phase 3 5, Phase 2/3 2, Phase 2 1, Phase 1 3) 2006: 10 trials (Observational 1, Phase 3 6, Phase 2 1, Phase 1/2 1, Phase 1 1) 2007: 18 trials (Observational 4, Phase 4 1, Phase 3 5, Phase 2/3 1, Phase 2 2, Phase 1/2 4, Phase 1 1) 2008: 11 trials (Not applicable 1, Phase 4 1, Phase 3 5, Phase 2 1, Phase 1/2 1, Phase 1 2) 2009: 11 trials (Observational 5, Phase 4 1, Phase 3 2, Phase 2 3) 2010: 23 trials (Observational 8, Not applicable 2, Phase 4 2, Phase 3 3, Phase 2 4, Phase 1/2 4) 2011: 18 trials (Observational 3, Not applicable 1, Phase 4 3, Phase 3 5, Phase 2/3 2, Phase 2 1, Phase 1/2 3) 2012: 18 trials (Observational 5, Not applicable 2, Phase 3 3, Phase 2/3 2, Phase 2 2, Phase 1/2 3, Phase 1 1) 2013: 20 trials (Observational 4, Not applicable 2, Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 6, Phase 1/2 3, Phase 1 2) 2014: 26 trials (Observational 5, Not applicable 1, Phase 4 1, Phase 3 7, Phase 2/3 1, Phase 2 3, Phase 1/2 3, Phase 1 5) 2015: 20 trials (Observational 8, Not applicable 3, Phase 4 2, Phase 3 4, Phase 1/2 1, Phase 1 2) 2016: 29 trials (Observational 5, Not applicable 2, Phase 4 4, Phase 3 8, Phase 2 5, Phase 1/2 3, Phase 1 2) 2017: 18 trials (Observational 4, Not applicable 1, Phase 4 2, Phase 3 3, Phase 2 2, Phase 1/2 3, Phase 1 3) 2018: 33 trials (Observational 15, Not applicable 2, Phase 4 3, Phase 3 3, Phase 2/3 1, Phase 2 3, Phase 1/2 4, Phase 1 2) 2019: 23 trials (Observational 9, Phase 3 7, Phase 2 4, Phase 1 3) 2020: 30 trials (Observational 12, Not applicable 3, Phase 4 2, Phase 3 3, Phase 2 7, Phase 1/2 1, Phase 1 2) 2021: 32 trials (Observational 12, Not applicable 2, Phase 4 1, Phase 3 9, Phase 2/3 1, Phase 2 3, Phase 1/2 2, Phase 1 2) 2022: 32 trials (Observational 12, Not applicable 2, Phase 4 1, Phase 3 8, Phase 2/3 1, Phase 2 2, Phase 1/2 2, Phase 1 4) 2023: 22 trials (Observational 12, Not applicable 4, Phase 3 1, Phase 2/3 1, Phase 2 2, Phase 1/2 2) 2024: 24 trials (Observational 11, Not applicable 3, Phase 4 1, Phase 3 7, Phase 2 2) 2025: 25 trials (Observational 8, Phase 4 2, Phase 3 9, Phase 2 2, Phase 1/2 4)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 24
2025 25
2024 24
2023 22
2022 32
2021 32
2020 30
2019 23
2018 33
2017 18

Showing 10 of 35 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 29 trials starting in 2026 and about 26 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 41 trials still running at the 2026-10-09 snapshot; registered median of 27 months across 326 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Primary Immunodeficiency Diseases trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 41
Phase 1/2 48
Phase 2 66
Phase 2/3 15
Phase 3 113
Phase 4 31
Not applicable 40
Observational 172
Expanded access 12

How many Primary Immunodeficiency Diseases trials are recruiting? (12)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 16
Recruiting 64
Enrolling by invitation 11
Active, not recruiting 33
Available 5

Showing 5 of 12 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Primary Immunodeficiency Diseases trials? (12)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Shire 39
National Institute of Allergy and Infectious Diseases (NIAID) 35
Takeda 28
CSL Behring 22
BioCryst Pharmaceuticals 17

Showing 5 of 12 sponsors.

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Which drugs are being tested for Primary Immunodeficiency Diseases? (11)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Busulfan 14
C1 esterase inhibitor [human] (C1INH-nf) 12
Icatibant 11
Berotralstat 11
Cytoxan 10

Showing 5 of 11 drugs.

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Interventions as named in the registry (4)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Globulin, Immune 24
SQ Fludarabine 12
Alemtuzumab s.c. 7
Total Body Irradiation 6

These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Primary Immunodeficiency Diseases trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Hereditary angioedema 41 17
Pyrexia 37 10
Pneumonia 31 26
Device related infection 23 6
Sepsis 18 10

Showing 5 of 10 adverse events.

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Which publications cover Primary Immunodeficiency Diseases? (56)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Key outcomes in treatment of activated phosphoinositide 3-kinase delta syndrome: An e-Delphi panel study and responder threshold application. PloS one 2025 Yes
Clinical and functional spectrum of RAC2-related immunodeficiency. Blood 2024 Yes
Facilitated subcutaneous immunoglobulin treatment patterns in pediatric patients with primary immunodeficiency diseases. Immunotherapy 2024 Yes
Long-term treatment with selective PI3Kδ inhibitor leniolisib in adults with activated PI3Kδ syndrome. Blood advances 2024 Yes
A phase 3 randomized trial of mavorixafor, a CXCR4 antagonist, for WHIM syndrome. Blood 2024 Yes

Showing 5 of 56 publications.

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Prevalence (28)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Primary immunodeficiency Point prevalence 1-9 / 100 000 United Kingdom GB Exact
Primary immunodeficiency Point prevalence 1-9 / 100 000 United Kingdom GB Exact
Primary immunodeficiency Point prevalence 1-9 / 100 000 Australia Not stated Exact
Primary immunodeficiency Point prevalence 1-5 / 10 000 France Not stated Exact
Primary immunodeficiency Point prevalence 1-9 / 100 000 Korea, Republic of Not stated Exact

Showing 5 of 28 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Primary immunodeficiency 101997 Exact Orphanet Rare Disease Ontology (ORDO)
Primary immunodeficiency 101997 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (9)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

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