Pittsburgh, PANCT01962415Now EnrollingIRB Ready

Primary Immunodeficiency (PID) Clinical Trial in Pittsburgh, PA

Access cutting-edge primary immunodeficiency (pid) treatment through this phase 2 clinical trial at a research site in Pittsburgh. Study-provided care at no cost to qualified participants.

Sponsored by Paul Szabolcs

Quick Self-Assessment

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Expert Care in Pittsburgh

Access primary immunodeficiency (pid) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related primary immunodeficiency (pid) treatment provided free

Apply for This Pittsburgh Location

Check if you qualify for this primary immunodeficiency (pid) clinical trial in Pittsburgh, PA

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Pittsburgh

    Convenient for PA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Pittsburgh site if eligible
  4. 4Begin participation

About This Primary Immunodeficiency (PID) Study in Pittsburgh

The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.

Sponsor: Paul Szabolcs

Who Can Participate

Inclusion Criteria

A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level matched unrelated donor bone marrow or peripheral blood progenitor graft.
Adequate organ function as measured by:
Creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2.
Hepatic transaminases (ALT/AST) ≤ 4 x upper limit of normal (ULN).
Adequate cardiac function by echocardiogram or radionuclide scan (shortening fraction \> 26% or ejection fraction \> 40% or \> 80% of normal value for age).
Pulmonary evaluation testing demonstrating CVC or FEV1/FVC of ≥ 50% of predicted for age and/or resting pulse oximeter ≥ 92% on room air or clearance by the pediatric or adult pulmonologist. For adult patients DLCO (corrected for hemoglobin) should be ≥ 50% of predicted if the DLCO can be obtained.
Written informed consent and/or assent according to FDA guidelines.
Negative pregnancy test if pubertal and/or menstruating.
HIV negative.
A non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to:
Primary Immunodeficiency syndromes including but not limited to:
Severe Combined Immune Deficiency (SCID) with NK cell activity
Omenn Syndrome
Bare Lymphocyte Syndrome (BLS)
Combined Immune Deficiency (CID) syndromes
Combined Variable Immune Deficiency (CVID) syndrome
Wiskott-Aldrich Syndrome
Leukocyte adhesion deficiency
Chronic granulomatous disease (CGD)
X-linked Hyper IgM (XHIM) syndrome
IPEX syndrome
Chediak - Higashi Syndrome
Autoimmune Lymphoproliferative Syndrome (ALPS)
Hemophagocytic Lymphohistiocytosis (HLH) syndromes
Lymphocyte Signaling defects
Other primary immune defects where hematopoietic stem cell transplantation may be beneficial
Congenital bone marrow failure syndromes including but not limited to:
Dyskeratosis Congenita (DC)
Congenital Amegakaryocytic Thrombocytopenia (CAMT)
Osteopetrosis
Inherited Metabolic Disorders (IMD) including but not limited to:
Mucopolysaccharidoses
Hurler syndrome (MPS I)
Hunter syndrome (MPS II)
Leukodystrophies
Krabbe Disease, also known as globoid cell leukodystrophy
Metachromatic leukodystrophy (MLD)
X-linked adrenoleukodystrophy (ALD)
Hereditary diffuse leukoencephalopathy with spheroids (HDLS)
Other inherited metabolic disorders
alpha mannosidosis
Gaucher Disease
Other inheritable metabolic diseases where hematopoietic stem cell transplantation may be beneficial.
Hereditary anemias
Thalassemia major
Sickle cell disease (SCD) - patients with sickle disease must have one or more of the following:
Overt or silent stroke
Pain crises ≥ 2 episodes per year for past year
One or more episodes of acute chest syndrome
Osteonecrosis involving ≥ 1 joints
Diamond Blackfan Anemia (DBA)
Other congenital transfusion dependent anemias
Inflammatory Conditions
Crohn's Disease/Inflammatory Bowel Disease

Exclusion Criteria

Allogeneic hematopoietic stem cell transplant within the previous 6 months.
Any active malignancy or MDS.
Severe acquired aplastic anemia.
Uncontrolled bacterial, viral or fungal infection (currently taking medication and with progression of clinical symptoms).
Pregnancy or nursing mother.
Poorly controlled pulmonary hypertension.
Any condition that precludes serial follow-up.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Pittsburgh?

Yes, this clinical trial (NCT01962415) has an active research site in Pittsburgh, PA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Primary Immunodeficiency (PID) Treatment Options in Pittsburgh, PA

If you're searching for primary immunodeficiency (pid) treatment options in Pittsburgh, PA, this clinical trial (NCT01962415) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Pittsburgh research site is actively enrolling participants for this phase 2 clinical trial. You'll receive care from experienced primary immunodeficiency (pid) specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all primary immunodeficiency (pid) clinical trials near you to find additional studies recruiting in your area.

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