Recent Updates
Recently added Catalysts

OTQ923

Phase 1

Sickle Cell Disease | Monoclonal antibody | Hematology |Novartis AG|Last Updated: Jul 28, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment4

FDA Designations

No designations recorded

Clinical trial landscape

OTQ923 · 1 trial · 1 indication

Phase 1 1
NCT06155500Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)Sickle Cell Disease
ACTIVE NOT_RECRUITING4 Analytics
PHASE1ACTIVE NOT_RECRUITING
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
Sickle Cell DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
Up to 15 years

Number of participants with delayed adverse events including new secondary malignancies, new incidence or exacerbation of a prior autoimmune disorder, new incidence or exacerbation of a prior rheumatologic disorder, new hematologic disorder, and other adverse events considered to be related to OTQ923 therapy.

Secondary Endpoints

Persistence of fetal hemoglobin expression
Up to 15 years
WBC chimerism in peripheral blood
5 years
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
OTQ923EXPERIMENTALPatients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment.

Interventions

NameTypeDescription
OTQ923BIOLOGICALThere is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101). 2. Patients must provide informed consent prior to their entry into this study. Exclusion Criteria: 1\. Completion of less than 1 year of safety follow-up in the treatment...

Countries:United States
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

LOWJul 28, 2026NCT06155500lastUpdatePostDate: changed
LOWJul 28, 2026NCT06155500lastUpdatePostDate: changed

Frequently asked questions about OTQ923

What is OTQ923 used for in Sickle Cell Disease?

OTQ923 is an investigational therapy being studied for the treatment of Sickle Cell Disease. It is currently in Phase 1 clinical development, with an active long-term follow-up trial enrolling adult patients in the United States. The therapy is not yet approved and remains under investigation.

Who is developing OTQ923?

OTQ923 is being developed by Novartis AG, a global pharmaceutical company traded on the New York Stock Exchange under the ticker NVS. The company is conducting a Phase 1 clinical trial to evaluate the therapy in patients with Sickle Cell Disease.

What phase is OTQ923 in?

OTQ923 is in Phase 1 clinical development. It is an investigational therapy for Sickle Cell Disease and has not been approved by regulatory authorities. The ongoing Phase 1 trial is active but not recruiting participants, with a small enrollment of four patients.

What clinical trials is OTQ923 in?

OTQ923 is being studied in a single Phase 1 clinical trial registered as NCT06155500. This trial is a long-term follow-up study of patients treated with genome-edited autologous hematopoietic stem and progenitor cells, conducted in the United States for adults with Sickle Cell Disease.

Is OTQ923 a monoclonal antibody?

OTQ923 is classified as a monoclonal antibody modality. It is being investigated for Sickle Cell Disease, a hematologic condition. The therapy is in Phase 1 clinical trials, and its mechanism of action has not been detailed in available information.