Sickle Cell DiseasePhase 1NCT06155500
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
A Phase 1 study testing OTQ923 in people with Sickle Cell Disease, run by Novartis Pharmaceuticals at 3 sites in United States.
Trial summary
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- OTQ923
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 3 sitesUnited States
- Study dates
- 16 Apr 2024to 11 Jan 2039
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101).
- Patients must provide informed consent prior to their entry into this study.
Who cannot join
- 1. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101).
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Chicago
Chicago, Illinois, 60637
St Jude Childrens Research Hospital
Memphis, Tennessee, 38105
Memorial Sloan Kettering Cancer Ctr
New York, New York, 10065
About this study
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
Read the detailed description
This study is monitoring patients treated with OTQ923, an investigational drug product of ex vivo genome-edited autologous hematopoietic stem and progenitor cells (HSPCs) that induces fetal hemoglobin (HbF) production, for a total of 15 years following infusion to monitor long-term safety and efficacy.
- Conditions
- Sickle Cell Disease
- Being tested
- OTQ923
- Keywords
- Gene therapy, genome-edited hematopoietic stem and progenitor cellular therapy, sickle cell, autologous transplant, BCL11A
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OTQ923Biological
There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)
| Study group | Role | What they receive |
|---|---|---|
| OTQ923 | Experimental | Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment. Receives: Biological: OTQ923 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
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.
Measured: Up to 15 years
Secondary outcomes (2)
Persistence of fetal hemoglobin expression
Assessment of fetal hemoglobin (HbF) expression persistence in peripheral blood will be done by assessing hemoglobin fractionation
Measured: Up to 15 years
WBC chimerism in peripheral blood
Concern for loss of engraftment by monitoring the WBC chimerism annually. Modified white blood cells (WBCs) measured by droplet digital PCR (ddPCR) and NGS will be performed annually through year 5 with subsequent samples stored and run annually in the event of concern for loss of engraftment
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2023
- Study start16 Apr 2024
- Primary completion11 Jan 2039 (estimated)
- Study completion11 Jan 2039 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06155500
- Sponsor's protocol ID
- CADPT03A12001
- Status last verified
- Jul 2026
- First submitted
- 24 Nov 2023
- Last updated
- 28 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
- ClinicalTrials.gov ID
- NCT06155500
- Acronym / sponsor ID
- CADPT03A12001
- Conditions
- Sickle Cell Disease
- Keywords
- Gene therapy, genome-edited hematopoietic stem and progenitor cellular therapy, sickle cell, autologous transplant, BCL11A
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 16 Apr 2024 (actual)
- Primary completion
- 11 Jan 2039 (estimated)
- Study completion
- 11 Jan 2039 (estimated)
- First posted
- 4 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
Detailed description
This study is monitoring patients treated with OTQ923, an investigational drug product of ex vivo genome-edited autologous hematopoietic stem and progenitor cells (HSPCs) that induces fetal hemoglobin (HbF) production, for a total of 15 years following infusion to monitor long-term safety and efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OTQ923 | Experimental | Biological: OTQ923Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101).
- Patients must provide informed consent prior to their entry into this study.
Key exclusion criteria (1 listed)
- 1. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OTQ923There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907) | Biological | OTQ923 |
Primary outcome
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
Time frame: 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.
2 secondary outcomes
Persistence of fetal hemoglobin expression
Time frame: Up to 15 years
WBC chimerism in peripheral blood
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OTQ923 in people with Sickle Cell Disease, run by Novartis Pharmaceuticals at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 28 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy (time frame: Up to 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jul 2026.