Could a higher drug dose help sickle cell patients get stem cell transplants?
NCT ID NCT07341022
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tests whether a high dose of plerixafor can safely collect enough stem cells from people with sickle cell disease for a future transplant. Twelve patients aged 18-25 will receive the drug and have their stem cells collected through a process called apheresis. The goal is to see if this approach works better than standard methods, while monitoring for side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Plerixafor (high-dose)
- What this could lead to
- If successful, this could improve stem cell collection for sickle cell patients, making bone marrow transplants safer and more accessible.
- What could go wrong
- This is a very small early-phase trial (12 people) testing a higher dose than usual, so safety and effectiveness are not yet proven. The drug may cause side effects or fail to mobilize enough cells.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
-
Jun 2026
An estimate. Start dates often move.
- Expected to finish
-
Jun 2028
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 25 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: Patients with sickle cell disease 18 to 25 years old At least one allogeneic HSCT indication followin the Brazilian Bone Marrow Transplant Society (SIMÕES et al., \[s.d.\]) ECOG/ Karnofsky/Lansky scores \> 80 Hemoglobin \> 7 g/dL, WBC counts \> 3000/mm3, neutrophil counts \> 1500/mm3, platelet counts \> 150000/mm3 No evidence of severe hepatic disfunction, defined as aspartate aminotransferase and alanine aminotransferase \< 5 times ULN or bilirubin \< 2,5 times ULN No evidence of renal disfunction, defined as creatinine \<1,5 mg/dL ou GFR\> 60 mL/min LVEF \> 40% and no signals of pulmonary hypertension Negative serologies for HIV, HBV, HCV, syphilis, Chagas disease or HTLV Being able to undergo partial exchange transfusion to lower HbS \<30% within one week before CD34+ mobilization and collection No pregnancy or breastfeeding; acceptance to use two contraceptive methods during the study. Exclusion Criteria: Emergency room admission or hospitalization in the past 14 days prior to first dose of study drug Major surgery in the past 30 days prior to first dose of study drug 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 Pregnancy 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
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Sickle cell disease are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Sickle cell clues to diabetes risk hidden in DNA
- Can adding common pain drugs reduce morphine needs in sickle cell crises?
- Gene editing offers hope for a One-Time sickle cell cure
- Tiny biochip could reveal sickle cell severity
- Can a milder transplant cure sickle cell and thalassemia in adults?
- Can an antioxidant supplement calm sickle cell blood cells?