Sickle Cell DiseasePhase 1/2NCT02504619
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
A Phase 1/2 study testing CordIn in people with Sickle Cell Disease and Thalassemia, run by Gamida Cell ltd at 3 sites in 2 countries.
Trial summary
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™. Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- Sickle Cell Disease, Thalassemia
- What is being tested?
- CordIn
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 4 Apr 2016to Dec 2017
- 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 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient is a candidate for allogeneic SCT for treatment of SCD or thalassemia
- Patients must have one partially HLA-matched CBUs
- Adequate Karnofsky Performance score or Lansky Play-Performance scale
- Sufficient physiological reserves
- Signed written informed consent
Who cannot join
- Prior allogeneic HSCT
- Evidence of HIV infection or HIV positive serology
- Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
- Active or uncontrolled infection
- Pregnancy or lactation
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
UCSF Benioff Children's Hospital
Oakland, California, 94609
Children's National
Washington D.C., District of Columbia, 20010
About this study
Official title: Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
Read the detailed description
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
The trial ends when the last patient completes their last visit. The overall study objectives are to evaluate the safety and efficacy of CordIn™: single ex-vivo expanded cord blood unit transplantation in patients with hemoglobinopathies (sickle cell disease or thalassemia major) following a preparative therapy.
- Conditions
- Sickle Cell Disease, Thalassemia
- Being tested
- CordIn
- Medical subject headings
- Anemia, Sickle Cell, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CordInBiological
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
| Study group | Role | What they receive |
|---|---|---|
| CordIn | Experimental | Transplantation of CordIn Receives: Biological: CordIn |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.
The number of patients with grade 4 or 5 toxicity were estimated together with 95% confidence limits based on the binomial distribution. The proportion with toxicity grades 1, 2, and 3 was also estimated.
Measured: 24 hours
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
Measured: 42 days
Secondary outcomes (1)
Percentage of Overall Survival at 365 Days After Transplantation
The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method.
Measured: 365 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2015 (estimated)
- Study start4 Apr 2016
- Primary completionAug 2016
- Study completionDec 2017
Why it stopped: lack of recruitment
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.
- Other trials for Sickle Cell DiseaseStudies 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gamida Cell ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jean-Hugues Dalle, MD, Principal investigator, Robert Debre, Paris, FranceFranco Locatelli, MD, Principal investigator, IRCCS Ospedale Pediatrico Bambino Gesu, Rome, ItalyAllistair Abraham, MD, Principal investigator, Children's National, Washington DC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02504619
- Sponsor's protocol ID
- GC P#01.01.030
- Status last verified
- Jun 2019
- First submitted
- 21 Jan 2015
- Last updated
- 26 Jun 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
- ClinicalTrials.gov ID
- NCT02504619
- Acronym / sponsor ID
- GC P#01.01.030
- Conditions
- Sickle Cell Disease, Thalassemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of recruitment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Gamida Cell ltdIndustry (pharma, biotech, devices)
- Study start
- 4 Apr 2016 (actual)
- Primary completion
- Aug 2016 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 22 Jul 2015 (estimated)
- Results
- Posted 26 Jun 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
Detailed description
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
The trial ends when the last patient completes their last visit. The overall study objectives are to evaluate the safety and efficacy of CordIn™: single ex-vivo expanded cord blood unit transplantation in patients with hemoglobinopathies (sickle cell disease or thalassemia major) following a preparative therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CordIn | Experimental | Biological: CordInTransplantation of CordIn |
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patient is a candidate for allogeneic SCT for treatment of SCD or thalassemia
- Patients must have one partially HLA-matched CBUs
- Adequate Karnofsky Performance score or Lansky Play-Performance scale
- Sufficient physiological reserves
- Signed written informed consent
Key exclusion criteria (5 listed)
- Prior allogeneic HSCT
- Evidence of HIV infection or HIV positive serology
- Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
- Active or uncontrolled infection
- Pregnancy or lactation
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 |
|---|---|---|
| CordInCordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. | Biological | CordIn |
Primary outcome
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.
Time frame: 24 hours
The number of patients with grade 4 or 5 toxicity were estimated together with 95% confidence limits based on the binomial distribution. The proportion with toxicity grades 1, 2, and 3 was also estimated.
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
Time frame: 42 days
One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
1 secondary outcome
Percentage of Overall Survival at 365 Days After Transplantation
Time frame: 365 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Jean-Hugues Dalle, MD principal investigator
Robert Debre, Paris, France
Franco Locatelli, MD principal investigator
IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy
Allistair Abraham, MD principal investigator
Children's National, Washington DC
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CordIn in people with Sickle Cell Disease and Thalassemia, run by Gamida Cell ltd at 3 sites in 2 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 5 inclusion and 5 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 26 Jun 2019. 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, France. 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 26 Jun 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?
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion. (time frame: 24 hours). 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?
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 26 Jun 2019.