Primary immunodeficiency

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ORPHA:101997
Who is this for?
Show terms as
1FDA treatments19Active trials63Specialists8Treatment centers4Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Primary Immunodeficiency is treated with 1 medication in our database, including ASCENIV. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by ADMA. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Primary Immunodeficiency treatment below.

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Sep 2026Confidential Care and Adolescent HIV Testing

Yale University — NA

TrialNOT YET RECRUITING
May 2026Sexual Health Access at Retail Pharmacies: Advancing Pharmacy-based Delivery of Primary STI and HIV Prevention for Cisgender Women

University of Washington — NA

TrialNOT YET RECRUITING
Mar 2026Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study

Santa Catarina Federal University

TrialENROLLING BY INVITATION
Dec 2025Quality of Life Among Children With Inborn Error of Immunity

Sohag University

TrialNOT YET RECRUITING
Oct 2025Assessing Patient Preference for Infusion Systems

KORU Medical Systems, Inc. — NA

TrialNOT YET RECRUITING
Sep 2025Study of the Prevalence of Transmitted HIV-1 Resistance, Viral Diversity, and Cluster Identification in Patients at the Time of HIV-1 Diagnosis

ANRS, Emerging Infectious Diseases

TrialNOT YET RECRUITING
Jul 2025Efficacy, Safety and Pharmacokinetic of Virchow IVIG in PID Patients

Virchow Group — PHASE3

TrialNOT YET RECRUITING
Mar 2025Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

Children's Hospital of Philadelphia — PHASE1, PHASE2

TrialRECRUITING
Feb 2025A Database Survey to Evaluate the Safety of Immune Globulin Subcutaneous (Human), 20% Solution in Participants With Primary Immunodeficiency

Takeda

TrialRECRUITING
Feb 2025Immune Cell Therapy (CAR-T) for the Treatment of Patients With HIV and B-Cell Non-Hodgkin Lymphoma

AIDS Malignancy Consortium — PHASE1

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

ASCENIV

HUMAN IMMUNOGLOBULIN G· ADMA

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

Clinical Trials

19 recruitingView all trials with filters →
Phase 32 trials
Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)
Phase 3
Active Prior treatment eligible
PI: Chiara Azzari (Azienda Ospedaliero-Universitaria Ospedale Pediatr) · Sites: San Francisco, California; Centennial, Colorado +18 more · Age: 216 yrs
Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases
Phase 3
Active
· Sites: Irvine, California; Centennial, Colorado +22 more · Age: 275 yrs
Phase 23 trials
Leniolisib for Immune Dysregulation in PIDs
Phase 2
Active
PI: Gulbu Uzel, M.D. (National Institutes of Health (NIH)) · Sites: Bethesda, Maryland · Age: 1275 yrs
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children
Phase 2
Actively Recruiting
PI: Deepak Chellapandian, MD (Johns Hopkins All Children's Hospital) · Sites: St. Petersburg, Florida · Age: 021 yrs
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Phase 2
Actively Recruiting
PI: Paul Szabolcs, MD (University of Pittsburgh) · Sites: Pittsburgh, Pennsylvania · Age: 055 yrs
N/A7 trials
Investigation of the Effects of Pulmonary Rehabilitation in Children With Primary Immunodeficiency
N/A
Actively Recruiting
PI: Betül Yoleri, Pt. MsC (Gazi University) · Sites: Ankara, Çankaya · Age: 618 yrs
Study of Populations at Risk of Developing Chronic Hepatitis Linked to Chronic Enteric Virus Infection in Patients With Primary Immunodeficiency and Secondary Humoral Deficiency
N/A
Actively Recruiting
PI: Victor Michel, MD (Assistance Publique - Hôpitaux de Paris) · Sites: Paris; Paris +1 more
Metabolic Profiling of Immune Responses in Immune-mediated Diseases
N/A
Actively Recruiting
PI: Ian A Myles, M.D. (National Institute of Allergy and Infectious Disea) · Sites: Bethesda, Maryland · Age: 1875 yrs
Ex Vivo Evaluation of JAK-inhibitor and Gene Therapeutical Approach in JAK-STAT Related Disorders
N/A
Actively Recruiting
PI: Rik Schrijvers, MD, PhD (UZ Leuven) · Sites: Leuven, Vlaams-Brabant · Age: 1899 yrs
NIAID Centralized Sequencing Protocol
N/A
Actively Recruiting
PI: Morgan N Similuk (National Institute of Allergy and Infectious Disea) · Sites: Washington D.C., District of Columbia; Bethesda, Maryland · Age: 1100 yrs
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
N/A
Actively Recruiting
PI: Paul Szabolcs, MD (UPMC Children's Hospital of Pittsburgh) · Sites: Pittsburgh, Pennsylvania · Age: 260 yrs
Virtual Reality on Pain and Fear Levels of Children With Primary Immunodeficiency
N/A
Actively Recruiting
PI: Hatice Dönmez, PhD · Sites: Karaman · Age: 411 yrs
Other6 trials
Genetic Analysis of Immune Disorders
Actively Recruiting
PI: Gulbu Uzel, M.D. (National Institute of Allergy and Infectious Disea) · Sites: Bethesda, Maryland · Age: 0101 yrs
Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation
Actively Recruiting
PI: Frédéric Rieux-Laucat (Institut Imagine) · Sites: Paris · Age: 118 yrs
A Database Survey to Evaluate the Safety of Immune Globulin Subcutaneous (Human), 20% Solution in Participants With Primary Immunodeficiency
Actively Recruiting
· Sites: Tokyo, Tokyo
Send-In Sample Collection to Achieve Genetic and Immunologic Characterization of Primary Immunodeficiencies
Actively Recruiting
PI: Ottavia M Delmonte, M.D. (National Institute of Allergy and Infectious Disea) · Sites: Bethesda, Maryland; Pavia +1 more · Age: 099 yrs
Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders
Enrolling by Invitation
PI: Helen C Su, M.D. (National Institute of Allergy and Infectious Disea) · Sites: Bethesda, Maryland · Age: 285 yrs
The Mechanistic Biology of Primary Immunodeficiency Disorders
Actively Recruiting
PI: Luigi D Notarangelo, M.D. (National Institute of Allergy and Infectious Disea) · Sites: Washington D.C., District of Columbia; Bethesda, Maryland · Age: 090 yrs

Specialists

Showing 25 of 63View all specialists →
CA
Chiara Azzari
San Francisco, California
Specialist

Rare Disease Specialist

PI on 1 active trial21 Primary immunodeficiency publications
HP
Hatice Dönmez, PhD
Karaman
Specialist

Rare Disease Specialist

MS
Morgan N Similuk
Washington D.C., District of Columbia
Specialist

Rare Disease Specialist

5 Primary immunodeficiency publications
MP
Michael Borte, MD, PhD
Specialist
PI on 2 active trials
RM
Robert Yarchoan, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 13 active trials
PM
Paul Szabolcs, MD
Pittsburgh, Pennsylvania
Specialist

Rare Disease Specialist

PI on 7 active trials
MM
Mitchell Cairo, MD
HAWTHORNE, NY
Specialist
PI on 11 active trials
MM
Mitchell S Cairo, MD
HAWTHORNE, NY
Specialist
PI on 12 active trials
UD
Urielle Desalbres
Specialist
PI on 21 active trials1 Primary immunodeficiency publication
SM
Suk S De Ravin, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 6 active trials
CM
Corina E Gonzalez, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 2 active trials
JM
John Horan, MD
Birmingham, Alabama
Specialist

Rare Disease Specialist

PI on 4 active trials
HM
Heather J Symons, MD, MHS
Baltimore, Maryland
Specialist

Rare Disease Specialist

PI on 1 active trial
JM
James Moy, MD
Specialist
PI on 2 active trials
NV
Nathalie COLIN de VERDIERE
Specialist
PI on 2 active trials
DM
Donald B. Kohn, M.D.
NEW HAVEN, CT
Specialist
PI on 2 active trials
IM
Isaac Melamed, MD
CENTENNIAL, CO
Specialist
PI on 3 active trials1 Primary immunodeficiency publication
JM
Jordan Milner, MD
GAINESVILLE, FL
Specialist
PI on 5 active trials

Treatment Centers

8 centers
⚗️ Trial Site

National Institutes of Health Clinical Center

📍 Bethesda, Maryland

👤 Payal P Khincha, M.D.

👤 Christopher Grunseich, M.D.

⚗️ Trial Site

University of Michigan

📍 Ann Arbor, Michigan

👤 Janssen Research & Development, LLC Clinicaltrial

⚗️ Trial Site

Johns Hopkins University

📍 Baltimore, Maryland

⚗️ Trial Site

UPMC Children's Hospital of Pittsburgh

📍 Pittsburgh, Pennsylvania

⚗️ Trial Site

Hôpital Necker-Enfants Malades

📍 Paris

👤 Julien Rossignol, Dr

⚗️ Trial Site

Hôpital Saint-Louis

📍 La Defense, Paris

👤 Study Physician +46 08-697-20 00, medical.info@sobi.com

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

Financial Resources

1 resources

Gamimune N

Bayer Corporation

Gamimune N — Contact Bayer Corporation

Unverified — confirm before calling
Patient Assistance
Manufacturer Program
Accepting applications

3 travel grants are also available for Primary immunodeficiency patients — see Travel Grants below ↓

Travel Grants

3 grants

Primary Immunodeficiency Financial Assistance Program

The Assistance Fund

Apply ↗

Accessia Health Primary Immune Deficiency - Public Insurance Financial Assistance

Accessia Health

Apply ↗

Immune Deficiency Foundation Other Support Resources

Immune Deficiency Foundation

Apply ↗

Community

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Latest news about Primary immunodeficiency

1 articles
NewsUNITERAREApr 3, 2026
Fund Update: PAN Foundation — Primary immunodeficiency Fund Primary immunodeficiency fund is currently closed
The PAN Foundation's financial assistance program for people with primary immunodeficiency is currently closed and not accepting new applications. Primary immun
See all news about Primary immunodeficiency

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Primary immunodeficiency

What is Primary immunodeficiency?

Primary Immunodeficiency is treated with 1 medication in our database, including ASCENIV. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by ADMA. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Primary Immunodeficiency treatment below.

Are there clinical trials for Primary immunodeficiency?

Yes — 19 recruiting clinical trials are currently listed for Primary immunodeficiency on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Primary immunodeficiency?

25 specialists and care centers treating Primary immunodeficiency are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Primary immunodeficiency?

1 FDA-approved treatment and 1 patient support program are currently tracked on UniteRare for Primary immunodeficiency. See the treatments and support programs sections for copay assistance, eligibility, and contact details.