ThalassemiaEarly Phase 1NCT03728322
iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
A Early Phase 1 study testing iHSCs treatment group in people with Thalassemia, run by Allife Medical Science and Technology Co., Ltd..
Trial summary
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9. Read more about this study
- Who is it for?
- Ages 2 to 60 years
- Conditions the study is for
- Thalassemia
- What is being tested?
- iHSCs treatment group
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- Jan 2019to Jan 2021
- Phase
- Early Phase 1
- 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 2 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects ≥ 2 and ≤ 60 years of age
- Subjects was confirmed the transfusion-dependent β-thalassemia
- Adequate organ function, as defined by:
- Serum creatinine ≤ 1.5 mg/dl ； Serum ALT/AST）≤2.5×ULN；ALB≥25g/L； Serum total bilirubin < 1.5x ULN Left ventricular ejection fraction≥50%
- Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases
- Subjects survival was expected≥6 months
- Adult patients were willing to use reliable contraceptives (such as condoms) and not to donate sperm throughout the study period and within three months of discharge
- Subjects and the guardians able to undergo post-physical therapy/rehabilitation
Who cannot join
- Subjects allergic to macromolecular biological agents such as antibodies or cytokines
- Subjects receipt of any investigational clinical trials within 3 months.
- Subjects previous treatment with any hematopoietic stem cell transplantation or other organ transplantation
- Uncontrolled bleeding symptoms
- Severe cardiovascular disease is known, including any of the following:
- Myocardial infarction or thrombosis has occurred in the past six months Subjects with unstable angina pectoris Subjects with Class III/IV cardiovascular disability according to the New York Heart Association Classification
Show all 13Show fewer
- Subjects have one kinds of tumors within 5 years
- Active hepatitis B (HBV DNA>1000copy/mL), hepatitis C or HIV infection.
- Subjects have an infectious diseases that cannot be controlled within 4 weeks
- subjects have severe central nervous system disease or epilepsy
- Subjects are Suffering from mental illness; Patients with alcohol dependence, drug abuse, drug addiction, and medical, psychological or social conditions that may interfere with research or have an impact on the evaluation of research results
- Women in pregnancy (positive urine/blood pregnancy test) or lactation
- Subjects who have other conditions that were not appropriate for the group determined by the researchers.
About this study
Official title: A Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
Read the detailed description
The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
- Conditions
- Thalassemia
- Being tested
- iHSCs treatment group
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iHSCs treatment groupBiological
iHSCs intravenous injection
| Study group | Role | What they receive |
|---|---|---|
| iHSCs treatment group | Experimental | Not described Receives: Biological: iHSCs treatment group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of treatment related adverse events as assessed by CTCAE v4.0
Defined as >= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2018
- Study startJan 2019 (estimated)
- Primary completionJan 2020 (estimated)
- Study completionJan 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThalassemiaStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Allife Medical Science and Technology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03728322
- Sponsor's protocol ID
- HBB HSC-01
- Status last verified
- Oct 2018
- First submitted
- 31 Oct 2018
- Last updated
- 5 Nov 2018
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations
- ClinicalTrials.gov ID
- NCT03728322
- Acronym / sponsor ID
- HBB HSC-01
- Conditions
- Thalassemia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Allife Medical Science and Technology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jan 2019 (estimated)
- Primary completion
- Jan 2020 (estimated)
- Study completion
- Jan 2021 (estimated)
- First posted
- 2 Nov 2018 (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.
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
Detailed description
The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
Study design
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| iHSCs treatment group | Experimental | Biological: iHSCs treatment group |
Who may qualify
- Ages
- 2 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Subjects ≥ 2 and ≤ 60 years of age
- Subjects was confirmed the transfusion-dependent β-thalassemia
- Adequate organ function, as defined by:
- Serum creatinine ≤ 1.5 mg/dl ； Serum ALT/AST）≤2.5×ULN；ALB≥25g/L； Serum total bilirubin < 1.5x ULN Left ventricular ejection fraction≥50%
- Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases
Key exclusion criteria (13 listed)
- Subjects allergic to macromolecular biological agents such as antibodies or cytokines
- Subjects receipt of any investigational clinical trials within 3 months.
- Subjects previous treatment with any hematopoietic stem cell transplantation or other organ transplantation
- Uncontrolled bleeding symptoms
- Severe cardiovascular disease is known, including any of the following:
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 |
|---|---|---|
| iHSCs treatment groupiHSCs intravenous injection | Biological | iHSCs treatment group |
Primary outcome
Occurrence of treatment related adverse events as assessed by CTCAE v4.0
Time frame: 1 year
Defined as >= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2018.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing iHSCs treatment group in people with Thalassemia, run by Allife Medical Science and Technology Co., Ltd..
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 13 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: status not confirmed, as of the sponsor's last update on 5 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
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?
Occurrence of treatment related adverse events as assessed by CTCAE v4.0 (time frame: 1 year). 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 5 Nov 2018.