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

Study point of contact

Novartis Pharmaceuticals
+41613241111
Novartis Pharmaceuticals
1-888-669-6682
[email protected]

Locations

1 United States site

Age

2 to 40 Years

Genotypes

HbSS, HbSC

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Biological

Compensation

Unknown

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.

participation requirements

1. Male or female subjects age 2-40 years inclusive

2. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC,
HbS/β0-thalassemia or others)

3. Performance status >70% (Karnofsky for subjects >16 years of age and Lansky for
subjects <16 years of age) 4. 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 5. Subjects, who have failed, not tolerated or refused hydroxyurea therapy.

participation restrictions

1. Available matched related donor for HSCT

2. Clinically significant active infection

3. Seropositive for HIV or HTLV

4. Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency

5. Prior HSCT or gene therapy

6. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis

7. Protocol defined iron overload

8. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya

9. Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya

Other protocol defined inclusion/exclusion criteria may apply

Locations

  • Memphis, Tennessee, United States, Novartis Investigative Site, 38105-3678 [Recruiting]

More info

View on ClinicalTrials.gov
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