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RecruitingPhase 1 / Phase 2

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years to 50 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Infusions

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests nula-cel, a treatment that changes genes in a person's own blood-forming stem cells, for severe sickle cell disease. It is for people ages 12 to 40. Researchers will check how safe it is and look for early signs that it works.

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You are 12 to 40 years old.
  • You have severe sickle cell disease despite proper care, with at least one of these: 4 or more severe pain crises in the past 2 years, or 2 or more episodes of acute chest syndrome (a serious lung problem) in the past 2 years, including at least 1 in the past year.
  • You score at least 80 on the Lansky or Karnofsky scale, which measures how well you can do daily activities.

Who can't join

  • You have a brother or sister who is available to donate stem cells and matches all 10 tested tissue markers.
  • You have had a blood-forming stem cell transplant or gene therapy.
  • You have or have had cancer, a disorder that makes too many blood cells, a major blood-clotting disorder, or a disorder that weakens your immune system.
  • You have an active infection from bacteria, viruses, fungi, or parasites that causes significant health concerns.
  • You are pregnant or breastfeeding after giving birth.
  • The study doctor finds a chromosome or gene change that may raise your risk of myelodysplastic syndrome (a bone marrow disorder) or acute myeloid leukemia (a blood cancer).

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * ≥12 to ≤ 40 years * Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures: * recurrent severe VOC (≥ 4 episodes in the preceding 2 years) * ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year) * Lansky/Karnofsky performance status of ≥ 80 Exclusion Criteria: * Available 10/10 HLA-matched sibling donor * Prior HSCT or gene therapy * Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder * Clinically significant and active bacterial, viral, fungal or parasitic infection * Pregnancy or breastfeeding in a postpartum female * Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment

Study sites (6)

CA

MO

NY

OH