Is NCT03653338 recruiting in Pittsburgh, PA?

Yes, NCT03653338 is currently recruiting participants at its Pittsburgh, PA location. This sickle cell anemia clinical trial is actively enrolling qualified participants.

How do I join the sickle cell anemia clinical trial in Pittsburgh?

To join this clinical trial in Pittsburgh, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Pittsburgh clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Sickle Cell Anemia Clinical Trial in Pittsburgh, PA

About This Sickle Cell Anemia Study in Pittsburgh

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.

Sponsor: Paul Szabolcs

Who Can Participate

Inclusion Criteria

Patient, parent, or legal guardian must have given written informed consent and/or assent according to FDA guidelines.

Ages 5 years to 40 years, at time of consent.

Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following:

Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years.

Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.

Stroke or neurologic event lasting \> 24 hours with an accompanying infarct on MRI in any patient for all ages; Brain MRI with silent infarct without clinical event in patients ≤ 16 years.

Chronic transfusion therapy defined as \> 8 packed red blood cell transfusions per year in the year prior to enrollment and/or evidence of red blood cell alloimmunization.

Elevated transcranial Doppler velocities - \> 200 cm/s, via the non-imaging technique or \> 185 cm/s by the imaging technique measured on 2 separate occasions ≥ 1-month apart

Elevated TRV \> 2.6m/s in patients ≥ 16 years old.

Sickle-related renal insufficiency and/or sickle hepatopathy and/or any irreversible end-organ damage in patients ≥ 16 years old. OR Diagnosis of beta-thalassemia or Diamond-Blackfan anemia complicated by transfusion dependence with evidence of iron overload.

A minimum donor match of 4/8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci in the related setting or minimum donor match of 6/8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci (with the DRB1 locus as a full match requirement). An unrelated donor and cord blood search must have been completed without an eligible 8/8 matched unrelated donor or 6/8 cord blood unit available. Patients who may have acceptable cord blood donor options (4/6 or better) but are limited by cell dose of a single cord will also be eligible for the proposed study.

Adequate function of other organ systems as measured by:

Creatinine clearance or GFR ≥ 45 ml/min/1.73m.

Hepatic transaminases (ALT/AST) ≤ 3 x upper limit of normal.

Liver MR imaging for iron content should be performed in all patients with Ferritin \> 500 ng/mL. If hepatic iron content \> 10mg Fe/g liver should have hepatology consultation and liver biopsy to confirm absence of cirrhosis, fibrosis or hepatitis.

Adequate cardiac function as measure by echocardiogram (shortening fraction \> 26% or ejection fraction \> 40% or \>80% of age-specific normal).

Pulmonary evaluation testing demonstrating FEV1/FVC ≥ 60% of predicted for age and/or resting pulse oximeter ≥ 92% on room air.

Cardiology clearance to proceed with conditioning regimen and HSCT.

Pulmonology clearance to proceed with conditioning regimen and HSCT.

Subjects must be human immunodeficiency virus (HIV) negative by PCR.

Negative pregnancy test for females ≥10 years old or who have reached menarche, unless surgically sterilized.

All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.

Subject and/or parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting (Refer to section,

Hydroxyurea must have been trialed and failed in patients with sickle cell disease. Patient

Exclusion Criteria

Patients with alternate, superior donor options (matched sibling donor or matched unrelated donor).

Patients who have undergone stem cell transplantation in the 6 months prior to anticipated conditioning.

Patients with history of a central nervous system (CNS) event within six months prior to start of conditioning (patient will be delayed until eligible).

Patients who are pregnant or lactating

Patients with uncontrolled bacterial, viral or fungal infection

Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.

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 (NCT03653338) 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.

Sickle Cell Anemia Treatment Options in Pittsburgh, PA

If you're searching for sickle cell anemia treatment options in Pittsburgh, PA, this clinical trial (NCT03653338) 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 clinical trial. You'll receive care from experienced sickle cell anemia 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 sickle cell anemia clinical trials near you to find additional studies recruiting in your area.

Sickle Cell Anemia Clinical Trial in Pittsburgh, PA

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