Health TrialsbyHealthPanel

SCD Stem Cell Mobilization and Apheresis Using Motixafortide

ConditionSickle Cell Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorSt. Jude Children's Research Hospital

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • Motixafortide

    Drug

    Given Subcutaneously (under the skin).

Treatment groups

20 Participants
are divided into 2 treatment groups
Group A: Treatment-Arm A-Single DoseExperimental treatment 1 intervention
Group B: Treatment-Arm B-Two Daily DosesExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

To assess the safety and tolerability of motixafortide in participants with sickle cell disease (SCD) as determined by the incidence of adverse events.

Time frame
0 - 30 days

Secondary outcomes

1

To determine the yield of CD34+ cells/kg after a single or daily dose of motixafortide in participants with SCD.

Time frame
1 - 2 days
2

To determine the mobilization effects as determined by peripheral blood CD34+ counts/uL after a single or daily dose of motixafortide in participants with SCD.

Time frame
1 - 2 days
3

To recommend a phase 2 dosing strategy of one or two doses motixafortide in participants with SCD based on the incidence of adverse events

Time frame
through study completion, an average of 1 year
4

To recommend a phase 2 dosing strategy for timing of apheresis based on peripheral blood CD34+ counts/uL after motixafortide administration in participants with SCD

Time frame
through study completion, an average of 1 year

Other outcomes

Sponsors and contacts

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

St. Jude Children's Research Hospital

Lead sponsor

Ayrmid Ltd

Collaborator

Source ClinicalTrials.gov
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