Health TrialsbyHealthPanel

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

ConditionsSickle Cell DiseaseBeta-thalassemia
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age4-65
SponsorNational Heart, Lung, and Blood Institute (NHLBI)

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • Total Body Irradiation

    Radiation

    Total Body Irradiation 400 cGy

  • Alemtuzumab

    Drug

    1 mg/kg

  • Abatacept

    Drug

    10 mg/kg x 6

  • Research blood draw

    Other intervention

    About 5 tablespoons of blood will be collected from donors for research purposes.

Treatment groups

90 Participants
are divided into 2 treatment groups
Group A: Matched related donorsOther 1 intervention
Group B: Transplant recipientsExperimental treatment 4 interventions

Trial outcomes

Primary outcomes

1

Donor myeloid chimerism

percentage of donor myeloid chimerism

Time frame
1 year

Secondary outcomes

1

Donor CD3 chimerism

percentage of donor myeloid chimerism

Time frame
1 year
2

Overall survival

percentage of patients surviving at 1 year

Time frame
1 year
3

Acute Graft versus Host Disease

percentage of patients with Grade 2-4 acute GVHD

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

National Heart, Lung, and Blood Institute (NHLBI)

Lead sponsor

Source ClinicalTrials.gov
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