Is NCT06442761 recruiting in Birmingham, AL?

Yes, NCT06442761 is currently recruiting participants at its Birmingham, AL location. This sickle cell disease clinical trial is actively enrolling qualified participants.

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

To join this clinical trial in Birmingham, 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 Birmingham 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 Birmingham, AL

About This Sickle Cell Disease Study in Birmingham

This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE * To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES * To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). * To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts * To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects

Sponsor: St. Jude Children's Research Hospital

Who Can Participate

Inclusion Criteria

Participants with severe sickle cell disease (SCD) who are ≥18 years of age and willing to donate autologous hematopoietic stem cells (HSCs) for advancing future gene therapy for SCD after collection of back-up product. Severe SCD, for the purpose of this study, will be defined as participants who are receiving chronic transfusion therapy due to SCD related complications or are eligible for or currently enrolled on an allogeneic transplant protocol.

Participant must have a documented diagnosis of SCD with documentation of SCD genotype by medical history

Participants should either have a central line in place, be able to undergo apheresis without the necessity of the insertion of a central venous catheter, or agree to have a central line placed if IV access is inadequate.

ECOG performance status/Karnofsky score/Lansky score \>80

White blood cell (WBC) count \>3.0 x 10\^9/L, absolute neutrophil count (ANC) \>1.0 x 10\^9/L, and platelet count \>150 x 10\^9/L, and hemoglobin \>7.0 gm/dL

Adequate renal function defined as serum/plasma creatinine \< 1.5 mg/dL and an estimated glomerular filtration rate (eGFR) of at least 60 mL/min/1.73 m\^2 based on the CKD-Epi equation or the St. Jude equation.

Adequate liver function defined as direct bilirubin \< 2.5 times the upper limit of normal range; aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 5 times the upper limit of normal range.

Participant's cardiac function (i.e., ejection fraction \>40%) and pulmonary status (i.e., no evidence of pulmonary hypertension) within the last 6 months must be sufficient to undergo apheresis, as assessed by the Principal Investigator or an independent physician evaluating the participant. If an assessment has not been done within the last 6 months, an echocardiogram will be performed.

Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II

Feasible manual or automated exchange transfusion plan to achieve hemoglobin S (HbS) near 30% within one week of mobilization

Female participants of childbearing age should have a negative pregnancy test.

Participants of childbearing potential should agree to use of a highly effective form of contraception during treatment and for at least 1 month after the last dose of motixafortide. Women of childbearing potential must agree to use 2 methods of effective contraception: One barrier method (e.g. diaphragm, or condom or sponge, each of which are to be combined with a spermicide) and one hormonal method, unless she uses a highly effective method. Highly effective methods of contraception include:

Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: oral, intravaginal, transdermal

Progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable, implantable

Intrauterine device (IUD)

Intrauterine hormone-releasing system (IUS)

Bilateral tubal occlusion

Vasectomised partner

Sexual abstinence.

Exclusion Criteria

Active and painful splenomegaly or splenomegaly (size greater than upper limit of normal on examination).

Participant who, by medical history, requires rare donor registry RBC units for transfusion, or is unable to receive routine transfusion. Eligible study participants must have undergone prior work-up for the presence of red cell alloantibodies and confirmation of available compatible blood product support

Known allergy to or contraindication for motixafortide administration, or medications routinely administered during apheresis

Participant who has had a prior autologous or allogeneic transplantation, inclusive of gene therapy

Active viral, bacterial, fungal, or parasitic infection.

History of cancer, excluding squamous carcinoma of the skin and cervical carcinoma in situ.

Participant who has received experimental therapy within 4 weeks prior to providing informed consent

Poorly controlled diabetes mellitus, as assessed by the Investigator

Concomitant treatment with alternative investigational agent unable to be held for 30 days

Unwillingness to use a highly effective method of contraception for 1 month after motixafortide

Inability or unwillingness of research participant or legal guardian/ representative to give written informed consent.

Inability or unwillingness of research participant to hold hydroxyurea for 30 days prior to first dose of study drug

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 Birmingham?

Yes, this clinical trial (NCT06442761) has an active research site in Birmingham, AL 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 Birmingham, AL

If you're searching for sickle cell disease treatment options in Birmingham, AL, this clinical trial (NCT06442761) 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 Birmingham 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 Birmingham, AL

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