A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

About the study

This study will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products – OTQ923 and HIX763 – each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Study point of contact

Novartis Pharmaceuticals
+41613241111
Novartis Pharmaceuticals
1-888-669-6682
novartis.email@novartis.com

Locations

3 United States sites

1 Italy site

Age

2 to 40 Years

Genotypes

HbSS, HbSC

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Biological

Compensation

Unknown

participation requirements

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.

participation restrictions

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
Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya

Other protocol defined inclusion/exclusion criteria may apply

Locations

  • Chicago, Illinois, United States, University of Chicago SC - 2, 60637 [Recruiting]
  • New York, New York, United States, Memorial Sloan Kettering Cancer Center, 10065 [Recruiting]
  • Memphis, Tennessee, United States, St Jude Children's Research Hospital, 38105-3678 [Recruiting]
  • Milano, MI, Italy, Novartis Investigative Site, 20132 [Recruiting]
Last updated 2022-05-06