Anemia, Sickle CellPhase 1/2NCT02186418
Gene Transfer for Patients With Sickle Cell Disease
A Phase 1/2 study testing ARU-1801 in people with Anemia, Sickle Cell, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- ARU-1801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2014to 31 Oct 2022
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Signed informed consent form.
- Has confirmed diagnosis of sickle cell disease (SCD)
- Has severe sickle cell disease, defined as one or more of the following:
- Minimum of two episodes of clinically diagnosed acute chest syndrome (ACS) requiring hospital admission, or one life threatening episode of ACS requiring intensive care unit (ICU) admission for exchange transfusion and/or intubation, or frequent ACS episodes which necessitate treatment with chronic transfusion therapy.
- Frequent painful vaso-occlusive episodes (VOEs) which significantly interfere with normal life activities, defined as a history of 2 or more severe acute sickle pain events per year requiring additional treatment at a medical facility outside of home pain management over the preceding 2-year period prior to study enrollment, or that necessitate chronic transfusion therapy.
- Subjects on chronic transfusion therapy for severe disease symptoms other than those listed above, and which interfere with normal life activities.
- Has failed hydroxyurea therapy, was unable to tolerate hydroxyurea therapy, or has actively made the choice to not take the recommended daily hydroxyurea advised for severe disease (Note: must be off hydroxyurea therapy for 2 months prior to stem cell collection). If refusing hydroxyurea, the subject must document that they have been educated about the benefits and continue to refuse the treatment. Patients placed on chronic transfusion therapy instead of hydroxyurea for severe disease are eligible. Subjects unable to take hydroxyurea due to financial or safety monitoring constraints are eligible.
- Has adequate functional status and organ function as determined at Screening.
Who cannot join
- Female subjects who are pregnant or lactating/breastfeeding.
- Female subjects who are not surgically sterile, postmenopausal or who refuse to practice effective method of birth control as determined by the Investigator for one year after receiving the study drug. Women must also agree not to breastfeed for 1 year after receiving the study drug.
- Any participant of reproductive potential who refuses to agree to use an appropriate contraceptive method determined by the Investigator, for 1 year after receiving the study drug.
- Patients with an active malignant disease or receiving treatment for any type of cancer (except squamous cell carcinoma, basal cell carcinoma, or carcinoma in situ of the skin).
- Current diagnosis or history of hepatitis B, hepatitis C, or HIV.
- Has received another study drug within 30 days, or 5 half-lives of the last dose (whichever is longer), prior to screening.
Show all 14Show fewer
- Has severe obstruction, restriction or diffusion defect on pulmonary function tests.
- Has uncontrolled bacterial, viral or fungal infections within 1 month prior to starting the conditioning part of the study. Subjects with fever should wait for symptoms to resolve before starting the conditioning part of the study.
- Has a history of stroke or is at moderate to high risk of primary stroke (eg receiving chronic transfusions or hydroxyurea for primary prevention of stroke; has severe cerebral vasculopathy defined as moderate stenosis in >2 arterial segments; and/or has sever stenosis/occlusion in ≤2 segments in the polygon of Willis or presence of Moyamoya-like disease).
- Patients with alpha thalassemia sickle cell disease.
- Has previous liver biopsy showing cirrhosis, bridging hepatic fibrosis, or active hepatitis; or has received chronic transfusions and has previous evidence of iron overload and evidence of liver fibrosis by noninvasive liver imaging.
- Has a matched sibling donor, unless the subject has declined this option or this option is not feasible. Documentation must be included as part of the informed consent process for subjects who decline this option.
- Has a known hypersensitivity to any study treatments (e.g. melphalan, plerixafor).
- Other protocol-defined inclusion-exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Gene Transfer For Patients With Sickle Cell Disease Using A Gamma Globin Lentivirus Vector: An Open-Label Phase 1 / 2 Pilot Study
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease
Read the detailed description
This study will assess the feasibility, safety and efficacy of gene transfer using ARU-1801 (CD34+ cells transduced with the gamma-globin lentiviral vector). Gene transfer will occur ex-vivo into CD34+ enriched human bone marrow or plerixafor-mobilized peripheral blood hematopoietic stem cells (HSC) collected from subjects with severe sickle cell disease (SCD). Subjects will undergo reduced intensity chemotherapy conditioning with single-dose melphalan to facilitate engraftment of ex-vivo ARU-1801 via IV infusion. Subjects will return to the study site at regular intervals for follow-up for 2 years after the ARU-1801 infusion. It is anticipated that a separate long-term follow-up (LTFU) clinical study will be initiated, in which all subjects completing the 2 year study visit will be asked to consent and enroll, and will followed for a further 13 years.
- Conditions
- Anemia, Sickle Cell
- Being tested
- ARU-1801
- Keywords
- sickle cell disease, sickle cell anemia, hemoglobinopathies, hematopoietic stem cell, gene transfer, gene therapy
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARU-1801Genetic
Autologous CD34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector
- Subjects with sickle cell anemia will undergo hematopoietic stem cell procurement by bone marrow harvest or apheresis after mobilization with plerixafor
- Reduced intensity chemotherapy conditioning with single dose melphalan will be used to facilitate engraftment of ex-vivo transduced cells.
| Study group | Role | What they receive |
|---|---|---|
| ARU-1801 | Experimental | Autologous CD34+ hematopoietic stem cells transduced ex-vivo with gamma-globin lentiviral vector. Administered via IV infusion. Receives: Genetic: ARU-1801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Grade 3 allergic reaction
Incidence of Grade 3 allergic reaction associated with infusion of transduced cell product
Measured: From infusion (Day 0) to 15 years
Incidence of Grade 4 infection
Incidence of Grade 4 infection following infusion of transduced cell product uncontrolled for ≥14 days
Measured: From infusion (Day 0) to 15 years
Incidence of Grade 4 neutropenia
Incidence of Grade 4 neutropenia lasting >1 month following melphalan
Measured: From date of chemotherapy clearance visit to 15 years post-infusion of transduced cells
Incidence of Grade 3 or 4 organ toxicity
Incidence of Grade 3 or 4 organ toxicity attributable to study procedures
Measured: From screening to 15 years post-infusion of transduced cells
Incidence of Adverse Events (AEs)
Measured: From screening to 15 years post-infusion of transduced cells
Incidence of Serious Adverse Events (SAEs)
Measured: From screening to 15 years post-infusion of transduced cells
Incidence of death due to study procedures
Measured: From screening to 15 years post-infusion of transduced cells
Incidence of hematological malignancy
Incidence of hematological malignancy due to vector insertion
Measured: From infusion (Day 0) to 15 years
Incidence of hematological cancer
Incidence of hematological cancer related to investigational product or study medications/procedures
Measured: From screening to 15 years post-infusion of transduced cells
Time to neutrophil recovery
Number of days from melphalan-induced nadir to the first of 3 consecutive absolute neutrophil counts ≥500 cells/µL
Measured: From ≥36 hours before Day 0 to 2 years post-infusion of transduced cells
Time to platelet recovery
Number of days from melphalan-induced nadir to the first of 3 consecutive platelet counts >50,000 cells/µL and independent of platelet transfusion for ≥7 days consecutive days.
Measured: From ≥36 hours before Day 0 to 2 years post-infusion of transduced cells
≥8x10⁶kg viable CD34+ cells
Number of subjects with a total number of CD34+ cells recovered from all collections combined (mobilized peripheral blood and bone marrow) of at least ≥8x10⁶kg viable CD34+ cells
Measured: Up to Year 2
≥4x10⁶ CD34+ cells/kg body weight transduced
Proportion of subjects for which a minimum of 4x10⁵ CD34+ cells/kg body weight from all collections combined have been successfully transduced
Measured: Up to Year 2
Bone marrow aspirates with ≥1% gene-marked cells
Number of subjects with bone marrow aspirates at 1-year post-infusion with ≥1% gene-marked cells
Measured: Infusion (Day 0) to 1 year
Secondary outcomes (10)
Quantity of Hb (hemoglobin) subtypes
Quantification of HbF^G16D and other Hb subtypes, including HbF (endogenous), HbS, adult Hb (HbA), HbA2 and, if applicable, HbC and HbE
Measured: Months 6, 12, 18, 24 and year 3, 4, 5
Change in proportion of antisickling/sickling hemoglobin
Change in the proportion of antisickling/sickling hemoglobin ([HbF+HbF^G16D+HbA2]/HbS) in months 6-12 post-transplantation compared to baseline
Measured: Baseline to Month 6 through 12
Percentage of F-RBC (fetal hemoglobin content in red blood cells)
Measured by flow cytometry
Measured: Months 6, 12, 18, 24, 36
Percentage of F-retics (fetal hemoglobin content in reticulocytes)
Measured by flow cytometry
Measured: Months 6, 12, 18, 24, 36
Presence of vector copies in white blood cell fraction
Measured: Days 30, 60, 90, Months 4, 5, 6, 9, 12, 18, 21, 24
Presence of vector copies in bone marrow
Measured by qPCR and DNA
Measured: Prior to ARU-1801 infusion, month 6, 12, 18, 24 and 36
Presence of gene-marked colony-forming unit cells (CFU-c) in bone marrow (BM) indicting gene transfer
Measured by CFU-c assay by qPCR on individual CFU-c
Measured: Prior to ARU-1801 infusion, month 6, 12, 18, 24 and 36
Number of annualized vaso-occlusive episodes (VOEs) pre-transplant versus post-transplant
Change in disease severity
Measured: Baseline to year 15
Frequency of opioid use pre-transplant versus post-transplant
Change in disease severity
Measured: Baseline to year 15
Change in QoL (Quality of Life)
Measured by adult sickle cell quality of life measurement (ASCQ-Me®)
Measured: Baseline, month 4, 5, 6, 12 and 24 and year 3, 4, 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2014 (estimated)
- Study startJul 2014
- Primary completion31 Oct 2022
- Study completion31 Oct 2022
Why it stopped: Sponsor decision due to funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Anemia, Sickle CellStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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 1/2
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Gamma Globin Lentivirus Vector
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Stella M. Davies, MB BS, PhD, MRCP, Study chair, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02186418
- Sponsor's protocol ID
- ARU-1801_Ph1_01
- Status last verified
- Jul 2023
- First submitted
- 30 Jun 2014
- Last updated
- 12 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Grimley M, Davies SM, Shrestha A, Shova A, Asnani M, Kent M, Sayani F, Quinn CT, Niss O, Lutzko C, Mehta PA, Khandelwal P, Little C, Chandra S, Felker S, Chi M, Kalfa TA, Knight-Madden J, Arumugam PI, Ramos KN, Witting S, Latham T, Bushman FD, Malik P. Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial. Nat Med. 2025 Jul;31(7):2204-2212. doi: 10.1038/s41591-025-03662-2. Epub 2025 May 26.
PubMed 40419809
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gene Transfer For Patients With Sickle Cell Disease Using A Gamma Globin Lentivirus Vector: An Open-Label Phase 1 / 2 Pilot Study
- ClinicalTrials.gov ID
- NCT02186418
- Acronym / sponsor ID
- ARU-1801_Ph1_01
- Conditions
- Anemia, Sickle Cell
- Keywords
- sickle cell disease, sickle cell anemia, hemoglobinopathies, hematopoietic stem cell, gene transfer, gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision due to funding
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- Jul 2014 (actual)
- Primary completion
- 31 Oct 2022 (actual)
- Study completion
- 31 Oct 2022 (actual)
- First posted
- 10 Jul 2014 (estimated)
- 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.
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease
Detailed description
This study will assess the feasibility, safety and efficacy of gene transfer using ARU-1801 (CD34+ cells transduced with the gamma-globin lentiviral vector). Gene transfer will occur ex-vivo into CD34+ enriched human bone marrow or plerixafor-mobilized peripheral blood hematopoietic stem cells (HSC) collected from subjects with severe sickle cell disease (SCD). Subjects will undergo reduced intensity chemotherapy conditioning with single-dose melphalan to facilitate engraftment of ex-vivo ARU-1801 via IV infusion. Subjects will return to the study site at regular intervals for follow-up for 2 years after the ARU-1801 infusion. It is anticipated that a separate long-term follow-up (LTFU) clinical study will be initiated, in which all subjects completing the 2 year study visit will be asked to consent and enroll, and will followed for a further 13 years.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Gamma Globin Lentivirus Vector
| Arm | Type | Receives |
|---|---|---|
| ARU-1801 | Experimental | Genetic: ARU-1801Autologous CD34+ hematopoietic stem cells transduced ex-vivo with gamma-globin lentiviral vector. Administered via IV infusion. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Signed informed consent form.
- Has confirmed diagnosis of sickle cell disease (SCD)
- Has severe sickle cell disease, defined as one or more of the following:
- Has failed hydroxyurea therapy, was unable to tolerate hydroxyurea therapy, or has actively made the choice to not take the recommended daily hydroxyurea advised for severe disease (Note: must be off hydroxyurea therapy for 2 months prior to stem cell collection). If refusing hydroxyurea, the subject must document that they have been educated about the benefits and continue to refuse the treatment. Patients placed on chronic transfusion therapy instead of hydroxyurea for severe disease are eligible. Subjects unable to take hydroxyurea due to financial or safety monitoring constraints are eligible.
- Has adequate functional status and organ function as determined at Screening.
Key exclusion criteria (14 listed)
- Female subjects who are pregnant or lactating/breastfeeding.
- Female subjects who are not surgically sterile, postmenopausal or who refuse to practice effective method of birth control as determined by the Investigator for one year after receiving the study drug. Women must also agree not to breastfeed for 1 year after receiving the study drug.
- Any participant of reproductive potential who refuses to agree to use an appropriate contraceptive method determined by the Investigator, for 1 year after receiving the study drug.
- Patients with an active malignant disease or receiving treatment for any type of cancer (except squamous cell carcinoma, basal cell carcinoma, or carcinoma in situ of the skin).
- Current diagnosis or history of hepatitis B, hepatitis C, or HIV.
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 |
|---|---|---|
| ARU-1801Autologous CD34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector * Subjects with sickle cell anemia will undergo hematopoietic stem cell procurement by bone marrow harvest or apheresis after mobilization with plerixafor * Reduced intensity chemotherapy conditioning with single dose melphalan will be used to facilitate engraftment of ex-vivo transduced cells. | Genetic | ARU-1801 |
Primary outcome
Incidence of Grade 3 allergic reaction
Time frame: From infusion (Day 0) to 15 years
Incidence of Grade 3 allergic reaction associated with infusion of transduced cell product
Incidence of Grade 4 infection
Time frame: From infusion (Day 0) to 15 years
Incidence of Grade 4 infection following infusion of transduced cell product uncontrolled for ≥14 days
Incidence of Grade 4 neutropenia
Time frame: From date of chemotherapy clearance visit to 15 years post-infusion of transduced cells
Incidence of Grade 4 neutropenia lasting >1 month following melphalan
Incidence of Grade 3 or 4 organ toxicity
Time frame: From screening to 15 years post-infusion of transduced cells
Incidence of Grade 3 or 4 organ toxicity attributable to study procedures
Incidence of Adverse Events (AEs)
Time frame: From screening to 15 years post-infusion of transduced cells
Incidence of Serious Adverse Events (SAEs)
Time frame: From screening to 15 years post-infusion of transduced cells
Incidence of death due to study procedures
Time frame: From screening to 15 years post-infusion of transduced cells
Incidence of hematological malignancy
Time frame: From infusion (Day 0) to 15 years
Incidence of hematological malignancy due to vector insertion
Incidence of hematological cancer
Time frame: From screening to 15 years post-infusion of transduced cells
Incidence of hematological cancer related to investigational product or study medications/procedures
Time to neutrophil recovery
Time frame: From ≥36 hours before Day 0 to 2 years post-infusion of transduced cells
Number of days from melphalan-induced nadir to the first of 3 consecutive absolute neutrophil counts ≥500 cells/µL
Time to platelet recovery
Time frame: From ≥36 hours before Day 0 to 2 years post-infusion of transduced cells
Number of days from melphalan-induced nadir to the first of 3 consecutive platelet counts >50,000 cells/µL and independent of platelet transfusion for ≥7 days consecutive days.
≥8x10⁶kg viable CD34+ cells
Time frame: Up to Year 2
Number of subjects with a total number of CD34+ cells recovered from all collections combined (mobilized peripheral blood and bone marrow) of at least ≥8x10⁶kg viable CD34+ cells
≥4x10⁶ CD34+ cells/kg body weight transduced
Time frame: Up to Year 2
Proportion of subjects for which a minimum of 4x10⁵ CD34+ cells/kg body weight from all collections combined have been successfully transduced
Bone marrow aspirates with ≥1% gene-marked cells
Time frame: Infusion (Day 0) to 1 year
Number of subjects with bone marrow aspirates at 1-year post-infusion with ≥1% gene-marked cells
10 secondary outcomes
Quantity of Hb (hemoglobin) subtypes
Time frame: Months 6, 12, 18, 24 and year 3, 4, 5
Change in proportion of antisickling/sickling hemoglobin
Time frame: Baseline to Month 6 through 12
Percentage of F-RBC (fetal hemoglobin content in red blood cells)
Time frame: Months 6, 12, 18, 24, 36
Percentage of F-retics (fetal hemoglobin content in reticulocytes)
Time frame: Months 6, 12, 18, 24, 36
Presence of vector copies in white blood cell fraction
Time frame: Days 30, 60, 90, Months 4, 5, 6, 9, 12, 18, 21, 24
Presence of vector copies in bone marrow
Time frame: Prior to ARU-1801 infusion, month 6, 12, 18, 24 and 36
Presence of gene-marked colony-forming unit cells (CFU-c) in bone marrow (BM) indicting gene transfer
Time frame: Prior to ARU-1801 infusion, month 6, 12, 18, 24 and 36
Number of annualized vaso-occlusive episodes (VOEs) pre-transplant versus post-transplant
Time frame: Baseline to year 15
Frequency of opioid use pre-transplant versus post-transplant
Time frame: Baseline to year 15
Change in QoL (Quality of Life)
Time frame: Baseline, month 4, 5, 6, 12 and 24 and year 3, 4, 5
Results and safety
The study was stopped early (reason given: Sponsor decision due to funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2024.
- Derived from this study: PubMed 40419809Grimley M, Davies SM, Shrestha A, Shova A, Asnani M, Kent M, Sayani F, Quinn CT, Niss O, Lutzko C, Mehta PA, Khandelwal P, Little C, Chandra S, Felker S, Chi M, Kalfa TA, Knight-Madden J, Arumugam PI, Ramos KN, Witting S, Latham T, Bushman FD, Malik P. Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial. Nat Med. 2025 Jul;31(7):2204-2212. doi: 10.1038/s41591-025-03662-2. Epub 2025 May 26.
- 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.
Stella M. Davies, MB BS, PhD, MRCP study chair
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ARU-1801 in people with Anemia, Sickle Cell, run by Children's Hospital Medical Center, Cincinnati at 1 site 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 8 inclusion and 14 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: stopped early, as of the sponsor's last update on 12 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision due to funding). Results are not available in the source record.
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?
Incidence of Grade 3 allergic reaction (time frame: From infusion (Day 0) to 15 years). The study lists 14 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02186418, which also lists 1 linked publication.
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 12 Apr 2024.