β-thalassemia MajorPhase 1/2NCT01745120
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
A Phase 1/2 study testing LentiGlobin BB305 Drug Product in people with β-thalassemia Major, run by Genetix Biotherapeutics Inc. at 6 sites in 3 countries.
Trial summary
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- β-thalassemia Major
- What is being tested?
- LentiGlobin BB305 Drug Product
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 6 sitesin 3 countries
- Study dates
- Aug 2013to 21 Feb 2018
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants between 12 and 35 years of age, inclusive, at the time of consent/assent, and able to provide written consent/assent, if applicable.
- Diagnosis of β-thalassemia major and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years.
- Eligible for allogeneic bone marrow transplant.
- Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
Who cannot join
- Positive for presence of human immunodeficiency virus type 1 or 2 (HIV 1 and HIV 2).
- A white blood cell (WBC) count <3 × 10^9/L, and / or platelet count <100 × 10^9/L if not due to hypersplenism.
- Uncorrected bleeding disorder.
- Any prior or current malignancy or myeloproliferative or immunodeficiency disorder.
- Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis).
- Receipt of an allogeneic transplant.
Show all 16Show fewer
- Advanced liver disease, including persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 × the upper limit of normal, liver biopsy demonstrating cirrhosis, extensive bridging fibrosis, or active hepatitis.
- Kidney disease with a calculated creatinine clearance <30% normal value.
- Uncontrolled seizure disorder.
- Diffusion capacity of carbon monoxide (DLco) <50% of predicted (corrected for hemoglobin).
- A cardiac T2* <10 ms by magnetic resonance imaging (MRI).
- Any other evidence of severe iron overload that, in the Investigator's opinion, warrants exclusion.
- Clinically significant pulmonary hypertension, as defined by the requirement for ongoing pharmacologic treatment or the consistent or intermittent use of supplemental home oxygen.
- Participation in another clinical study with an investigational drug within 30 days of Screening.
- Any prior or current malignancy or myeloproliferative disorder.
- Prior receipt of gene therapy.
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
United States4 sites
Study site
Chicago, Illinois
Study site
Los Angeles, California
Study site
Oakland, California
Study site
Philadelphia, Pennsylvania
About this study
Official title: A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product [autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene].
Read the detailed description
Subject participation for this study will be 2 years. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant.
- Conditions
- β-thalassemia Major
- Being tested
- LentiGlobin BB305 Drug Product
- Keywords
- gene therapy, β thalassemia, hemoglobin, anemia, CD34
- Medical subject headings
- Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LentiGlobin BB305 Drug ProductGenetic
Transplant of autologous hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector.
| Study group | Role | What they receive |
|---|---|---|
| LentiGlobin BB305 Drug Product | Experimental | Not described Receives: Genetic: LentiGlobin BB305 Drug Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Sustained Production of >=2.0 Grams Per Deciliter (g/dL) of Hemoglobin A (HbA) Containing βA-T87Q-globin (HbAT87Q) for the Six Months Between Month 18 and Month 24
Percentage of participants with sustained production of >=2.0 grams per deciliter (g/dL) of hemoglobin A (HbA) containing βA-T87Q-globin (HbAT87Q) for 6 months (Month 18 to Month 24) was reported.
Measured: Month 18 to Month 24
Percentage of Participants Who Achieved Transfusion Independence (TI)
TI was defined as a weighted average hemoglobin (Hb) >= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of >=12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Percentage of participants who achieved TI from time of drug product infusion up to 24 months was reported.
Measured: From time of drug product infusion up to 24 months
Secondary outcomes (17)
Percentage of Participants Who Achieved Transfusion Independence (TI) at Month 18 and Month 24
TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion.
Measured: Month 18, Month 24
Duration of Transfusion Independence (TI)
TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Time period of TI will start when participants achieve a Hb >= 9 g/dL with no transfusions in the preceding 60 days. Duration of TI was calculated as the time from the start of TI (i.e. first Hb >= 9 g/dL with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met.
Measured: From time of drug product infusion up to 24 months
Time From LentiGlobin BB305 Drug Product Infusion to Last pRBC Transfusion Prior to Achieving Transfusion Independence (TI)
TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Time From LentiGlobin BB305 Drug Product Infusion to last pRBC transfusion prior to achieving TI was reported.
Measured: From time of drug product infusion up to 24 months
Time From LentiGlobin BB305 Drug Product Infusion to Achieving Transfusion Independence (TI)
TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Time from drug product infusion to initial achievement of TI was calculated as the time from drug product infusion to the first Hb at which a participant can be declared as TI.
Measured: From time of drug product infusion up to 24 months
Weighted Average Hemoglobin (Hb) During Period of Transfusion Independence (TI)
The weighted average Hb is an average area under the curve during the period of TI, from the start of TI when the Hb is first >= 9 g/dL with no transfusions in the preceding 60 days to the last available Hb at which the TI criteria are still met. TI was defined as a weighted average Hb >= 9 g/dL without any pRBC transfusions for a continuous period of >= 12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Weighted average Hb during the period of TI was reported.
Measured: From time of drug product infusion up to 24 months
Percentage Change From Baseline in Annualized Number of Packed Red Blood Cells (pRBC) Transfusions at Month 24
The annualized number of pRBC transfusions over the 2 year period prior to drug product infusion was compared to the annualized number of pRBC transfusions during the Month 6 to Month 24 period post drug product infusion and the percentage change was reported.
Measured: Baseline, Month 24
Percentage Change From Baseline in Average Annual Packed Red Blood Cells (pRBC) Transfusion Volume at Month 24
The annualized volume of pRBC transfusions over the 2 year period prior to drug product infusion was compared to the annualized volume of pRBC transfusions in the Month 6 to Month 24 period post drug product Infusion and the percentage change from baseline was reported.
Measured: Baseline, Month 24
Weighted Average Nadir Hemoglobin (Hb)
Weighted average Hb nadir was defined as an average area under the curve where the Hb closest but within 3 days prior to a transfusion is used as the Hb nadir. If there is a period of more than 60 days without a pRBC transfusion, all Hb records between Day 61 and day of last visit or next transfusion (inclusive) were also considered as nadirs. The weighted average nadir Hb during the period of Month 6 to Month 24 was compared to the weighted average nadir Hb during the 2 years prior to enrollment.
Measured: Baseline, Month 6 to Month 24
Number of Participants With Successful Neutrophil Engraftment
Neutrophil engraftment was defined as achieving 3 consecutive absolute neutrophil count (ANC) >= 0.5 × 10^9/L on different days after a post-transplant value of < 0.5 × 10^9/L within 42 days after drug product infusion.
Measured: From time of drug product infusion up to 24 months
Time to Neutrophil Engraftment
Time to neutrophil engraftment was defined as the time to the first of 3 consecutive absolute neutrophil count (ANC) >= 0.5 × 10^9/L obtained on different days after a post-transplant value of < 0.5 × 10^9/L. The Day of neutrophil engraftment is the first day of the 3 consecutive measurements, where Day 1 is the day of drug product infusion.
Measured: From time of drug product infusion up to 24 months
Number of Participants With Successful Platelet Engraftment
Platelet engraftment was defined as achieving 3 consecutive platelet values >= 20 × 10^9/L on different days after a post-transplant value of < 20 × 10^9/L, while no platelet transfusions administered for 7 days immediately preceding and during the evaluation period.
Measured: From time of drug product infusion up to 24 months
Time to Platelet Engraftment
Time to platelet engraftment was defined as achieving of first 3 consecutive platelet values >= 20 × 10^9/L obtained on different days after a post-transplant value of < 20 × 10^9/L, while no platelet transfusions administered for 7 days immediately preceding and during the evaluation period. The day of platelet engraftment is the first day of the 3 consecutive measurements, where Day 1 is the day of drug product infusion.
Measured: From time of drug product infusion up to 24 months
Transplant-related Mortality
Transplant-related mortality was determined by the investigator (any deaths considered related to the transplant.)
Measured: Through 100 and 365 days post-LentiGlobin BB305 Drug Product infusion
Overall Survival
Overall survival was defined as time from date of LentiGlobin BB305 Drug Product infusion (Day 1) to date of death. Overall survival was censored at the date of last visit if the participant was still alive. Percentage of participants who survived throughout the study were reported.
Measured: From time of drug product infusion up to 24 months
Percentage of Participants Detected With Replication-competent Lentivirus (RCL)
Blood samples were analyzed for detection of RCL using RCL co-culture assay.
Measured: From time of drug product infusion up to 24 months
Number of Participants With Integration Site Analysis (ISA) With >30% Clonal Contribution
Linear amplification-mediated polymerase chain reaction (LAM-PCR) coupled with next generation sequencing and subsequent (semi-) automated data mining allowed high-throughput analysis of vector integration site (IS) in blood cells from treated participants at multiple time points. ISs detected in peripheral blood cells at early time points generally were due to the expansion of transduced short-term progenitor stem cell clones, and gradually shift to include sites detected due to expansion of transduced long-term stem cell clones. An efficient transduction procedure was anticipated to give rise to a polyclonal population in the participant, reflected by the detection of multiple IS. Additionally, ISA allowed monitoring of the relative contribution of individual clones over time. Number of participants who had IS that contributed to >=30% of the total clones at any time was used as a first step to investigating whether clonal dominance was achieved.
Measured: From time of drug product infusion up to 24 months
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was defined as any unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A SAE was any AE, occurring at any dose and regardless of causality that: results in death, was life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect.
Measured: From signing of informed consent to 24 months after the drug product infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2012 (estimated)
- Study startAug 2013
- Primary completion8 Feb 2018
- Study completion21 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for β-thalassemia MajorStudies of the same condition, open and under way.
- 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mohammed Asmal, MD, Study director, Genetix Biotherapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01745120
- Sponsor's protocol ID
- HGB-204
- Status last verified
- Apr 2019
- First submitted
- 6 Dec 2012
- Last updated
- 8 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarre C, Beuzard Y, Chretien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.
PubMed 29669226
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)
- ClinicalTrials.gov ID
- NCT01745120
- Acronym / sponsor ID
- HGB-204
- Conditions
- β-thalassemia Major
- Keywords
- gene therapy, β thalassemia, hemoglobin, anemia, CD34
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2013
- Primary completion
- 8 Feb 2018 (actual)
- Study completion
- 21 Feb 2018 (actual)
- First posted
- 7 Dec 2012 (estimated)
- Results
- Posted 8 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product [autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene].
Detailed description
Subject participation for this study will be 2 years. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LentiGlobin BB305 Drug Product | Experimental | Genetic: LentiGlobin BB305 Drug Product |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Participants between 12 and 35 years of age, inclusive, at the time of consent/assent, and able to provide written consent/assent, if applicable.
- Diagnosis of β-thalassemia major and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years.
- Eligible for allogeneic bone marrow transplant.
- Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
Key exclusion criteria (16 listed)
- Positive for presence of human immunodeficiency virus type 1 or 2 (HIV 1 and HIV 2).
- A white blood cell (WBC) count <3 × 10^9/L, and / or platelet count <100 × 10^9/L if not due to hypersplenism.
- Uncorrected bleeding disorder.
- Any prior or current malignancy or myeloproliferative or immunodeficiency disorder.
- Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis).
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 |
|---|---|---|
| LentiGlobin BB305 Drug ProductTransplant of autologous hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector. | Genetic | LentiGlobin BB305 Drug Product |
Primary outcome
Percentage of Participants With Sustained Production of >=2.0 Grams Per Deciliter (g/dL) of Hemoglobin A (HbA) Containing βA-T87Q-globin (HbAT87Q) for the Six Months Between Month 18 and Month 24
Time frame: Month 18 to Month 24
Percentage of participants with sustained production of >=2.0 grams per deciliter (g/dL) of hemoglobin A (HbA) containing βA-T87Q-globin (HbAT87Q) for 6 months (Month 18 to Month 24) was reported.
Percentage of Participants Who Achieved Transfusion Independence (TI)
Time frame: From time of drug product infusion up to 24 months
TI was defined as a weighted average hemoglobin (Hb) >= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of >=12 months at any time during the study after LentiGlobin BB305 Drug Product infusion. Percentage of participants who achieved TI from time of drug product infusion up to 24 months was reported.
17 secondary outcomes
Percentage of Participants Who Achieved Transfusion Independence (TI) at Month 18 and Month 24
Time frame: Month 18, Month 24
Duration of Transfusion Independence (TI)
Time frame: From time of drug product infusion up to 24 months
Time From LentiGlobin BB305 Drug Product Infusion to Last pRBC Transfusion Prior to Achieving Transfusion Independence (TI)
Time frame: From time of drug product infusion up to 24 months
Time From LentiGlobin BB305 Drug Product Infusion to Achieving Transfusion Independence (TI)
Time frame: From time of drug product infusion up to 24 months
Weighted Average Hemoglobin (Hb) During Period of Transfusion Independence (TI)
Time frame: From time of drug product infusion up to 24 months
Percentage Change From Baseline in Annualized Number of Packed Red Blood Cells (pRBC) Transfusions at Month 24
Time frame: Baseline, Month 24
Percentage Change From Baseline in Average Annual Packed Red Blood Cells (pRBC) Transfusion Volume at Month 24
Time frame: Baseline, Month 24
Weighted Average Nadir Hemoglobin (Hb)
Time frame: Baseline, Month 6 to Month 24
Number of Participants With Successful Neutrophil Engraftment
Time frame: From time of drug product infusion up to 24 months
Time to Neutrophil Engraftment
Time frame: From time of drug product infusion up to 24 months
Number of Participants With Successful Platelet Engraftment
Time frame: From time of drug product infusion up to 24 months
Time to Platelet Engraftment
Time frame: From time of drug product infusion up to 24 months
Transplant-related Mortality
Time frame: Through 100 and 365 days post-LentiGlobin BB305 Drug Product infusion
Overall Survival
Time frame: From time of drug product infusion up to 24 months
Percentage of Participants Detected With Replication-competent Lentivirus (RCL)
Time frame: From time of drug product infusion up to 24 months
Number of Participants With Integration Site Analysis (ISA) With >30% Clonal Contribution
Time frame: From time of drug product infusion up to 24 months
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From signing of informed consent to 24 months after the drug product infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 29669226Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarre C, Beuzard Y, Chretien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.
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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.
Mohammed Asmal, MD study director
Genetix Biotherapeutics Inc.
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LentiGlobin BB305 Drug Product in people with β-thalassemia Major, run by Genetix Biotherapeutics Inc. at 6 sites in 3 countries.
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 4 inclusion and 16 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: finished, as of the sponsor's last update on 8 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Australia, Thailand. 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?
Yes. Results are posted in the registry (first posted 8 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With Sustained Production of >=2.0 Grams Per Deciliter (g/dL) of Hemoglobin A (HbA) Containing βA-T87Q-globin (HbAT87Q) for the Six Months Between Month 18 and Month 24 (time frame: Month 18 to Month 24). The study lists 2 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 NCT01745120, 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 8 May 2019.