Stem cell harvest trial aims to unlock gene therapy for rare blood disorders

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy * Age ≥ 18 years - 25 years * The following hematological parameters need to be met (regardless of transfusion or growth factor support) * Hb \> 8 g/dL * ANC \> 500/mm3 * Platelet \> 30,000/mm3 * Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis * Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed * Karnofsky score \>80 * Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II * Female participants of childbearing age should have a negative serum pregnancy test within one week of beginning Filgrastim and plerixafor administration Exclusion Criteria: * Participant with sickle cell disease * Participant who has had a prior autologous or allogeneic HSCT * Active viral, bacterial, fungal, or parasitic infection * Total bilirubin \>2.5x ULN or transaminases \>5x ULN * Moderate or severe renal failure defined as serum/plasma creatinine \>1.5 mg/dL and an estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73 m2 based on the CKD-Epi equation or the St. Jude equation * Diagnosis of MDS or other hematologic malignancy * History of malignancy * Known allergy to or contraindication for Filgrastim or plerixafor administration, or medications routinely administered during apheresis * Splenomegaly (size greater than upper limit of normal on examination) * Any disease or concomitant process that is not compatible with the study as per investigator opinion * Concomitant treatment with alternative investigational agent or participation in another clinical trial with an investigational drug within 5 half-lives of the investigational agent * Unwillingness to use a highly effective method of contraception for 1 month after plerixafor or GCSF * Pregnancy * Inability or unwillingness of research participant to give written informed consent.