TerminatedPhase 1

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

Stopped: The decision of early termination was made due to business reasons and was not a consequence of any safety concern.

United States4 participantsStarted 2020-08-25

Plain-language summary

This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Who can participate

Age range2 Years – 40 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female subjects age 2-40 years inclusive
✓. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others)
✓. Performance status \>70% (Karnofsky for subjects \>16 years of age and Lansky for subjects \<16 years of age)
✓. At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization
✓. Subjects, who have failed, not tolerated or refused hydroxyurea therapy.

Exclusion criteria

✕. Available matched related donor for HSCT
✕. Clinically significant active infection
✕. Seropositive for HIV or HTLV
✕. Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency
✕. Prior HSCT or gene therapy
✕. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis
✕. Protocol defined iron overload
✕. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya

What they're measuring

Number of participants with adverse events and serious adverse events

Timeframe: up to 24 months

Fetal hemoglobin (HbF) expression 6 months after hematopoietic stem cell transplant (HSCT)

Timeframe: at 6 months

Time to reach absolute neutrophil count (ANC) ≥500/μL for 3 consecutive days

Timeframe: up to 24 months

Trial details

NCT IDNCT04443907

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-01-06

View on ClinicalTrials.gov More Sickle Cell Disease trials

Who can participate

Age range2 Years – 40 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female subjects age 2-40 years inclusive
✓. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others)
✓. Performance status \>70% (Karnofsky for subjects \>16 years of age and Lansky for subjects \<16 years of age)
✓. At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization
✓. Subjects, who have failed, not tolerated or refused hydroxyurea therapy.

Exclusion criteria

✕. Available matched related donor for HSCT
✕. Clinically significant active infection
✕. Seropositive for HIV or HTLV
✕. Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency
✕. Prior HSCT or gene therapy
✕. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis
✕. Protocol defined iron overload
✕. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya

What they're measuring

Number of participants with adverse events and serious adverse events

Timeframe: up to 24 months

Fetal hemoglobin (HbF) expression 6 months after hematopoietic stem cell transplant (HSCT)

Timeframe: at 6 months

Time to reach absolute neutrophil count (ANC) ≥500/μL for 3 consecutive days

Timeframe: up to 24 months