Is NCT07599176 recruiting in Bethesda, MD?

Yes, NCT07599176 is currently recruiting participants at its Bethesda, MD location. This sickle cell disease clinical trial is actively enrolling qualified participants.

How do I join the sickle cell disease clinical trial in Bethesda?

To join this clinical trial in Bethesda, 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 Bethesda 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 Disease Clinical Trial in Bethesda, MD

About This Sickle Cell Disease Study in Bethesda

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Who Can Participate

Inclusion Criteria

RECIPIENT: Participants must fulfill one disease category (1 or 2) and 3

Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F): A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb B. Abnormal trans-cranial Doppler examination (\>=200 cm/s); OR C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR D. Sickle cell related renal insufficiency defined by a creatinine level \>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \<60mL/min/1.73m2 for patients \<16 years of age or \<50mL/min for patients \>16 years of age OR requiring peritoneal or hemodialysis; OR Age (Years): \<= 5 / Upper limit of normal serum creatinine (mg/dl): 0.8 Age (Years): 5 \< age \<= 10 / Upper limit of normal serum creatinine (mg/dl): 1.0 Age (Years): 10 \< age \<= 15 / Upper limit of normal serum creatinine (mg/dl): 1.2 Age (Years): \> 15 / Upper limit of normal serum creatinine (mg/dl): 1.3 E. Tricuspid regurgitant jet velocity (TRV) of \>=2.5 m/s in patients at least 3 weeks after a vaso-occlusive crisis; OR F. Recurrent severe priapism defined as at least two episodes of an erection lasting \>=4 hours requiring medical intervention (e.g. aspiration, injection of vasoconstrictor, prior penile surgery.); OR G. Sickle hepatopathy defined as EITHER ferritin \>1000mcg/L OR direct bilirubin \>0.4 mg/dL at baseline; OR H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment /medication; OR I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment /medication

Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:

Portal fibrosis by liver biopsy

Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week)

Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans

Non disease specific

Ages \>=4 years and less than 65 years old

Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10)

Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable

Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration.

Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant/injection from start of screening until immunosuppression is stopped.

Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped. DONOR:

Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study.

Donors age 4 or older and \>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study.

Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.

Exclusion Criteria

Karnofsky or Lanksy performance status of \<40

Diffusing capacity of carbon monoxide \<35% predicted: DLCO corrected for hemoglobin or KCO (corrected for lung volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing.

Baseline oxygen saturation of \<85% or PaO2 \<70

Left ventricular ejection fraction: \<35% estimated by ECHO

Transaminases \>5x upper limit of normal for age

Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen

Major anticipated illness or organ failure incompatible with survival from HCT

Pregnant or breastfeeding DONOR:

Pregnant or breastfeeding

Cognitively impaired subjects

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 Bethesda?

Yes, this clinical trial (NCT07599176) has an active research site in Bethesda, MD 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 Disease Treatment Options in Bethesda, MD

If you're searching for sickle cell disease treatment options in Bethesda, MD, this clinical trial (NCT07599176) 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 Bethesda research site is actively enrolling participants for this clinical trial. You'll receive care from experienced sickle cell disease 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 disease clinical trials near you to find additional studies recruiting in your area.

Sickle Cell Disease Clinical Trial in Bethesda, MD

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