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Author Topic: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials  (Read 78423 times)
Sharmin
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Little A


« Reply #120 on: February 23, 2014, 04:14:29 PM »

Thanks Andy,
You're right - by the time lil A is old enough - likely 10 patients will have been through the procedure and we will have a good idea of how things are going.  Things sound really positive already - I can't wait to hear more updates about the patients in June!

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Sharmin
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« Reply #121 on: March 06, 2014, 03:04:51 PM »

Memorial Sloan-Kettering Cancer Center has updated the clinicaltrials.gov website on March 4, 2014.

Following are the highlights:

1)   The primary outcome measure data collection is still July 2014 (this is good given it was postponed in the past).
2)   The safety issue should be addressed by July 2014 (removing the main hurdle)

Excerpts from the site as follows:

Further study details as provided by Memorial Sloan-Kettering Cancer Center:

Primary Outcome Measures:
•safety [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
of transplanted autologous CD34+ hematopoietic cells that are transduced ex vivo with TNS9.3.55 and transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen.

a.The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts and leukocyte clonality using FACS analysis, qPCR for vector. copy number, LAM-PCR and/or 454 sequencing;
b.The generation of a replication-competent lentivirus (RCL).
c.The safety of a low dose non-myeloablative conditioning regimen

•tolerability [ Time Frame: 2 years ] [ Designated as safety issue: No ]
of transplanted autologous CD34+ hematopoietic cells that are transduced ex vivo with TNS9.3.55 & transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen. monitoring the following: 1. The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts & leukocyte clonality using FACS analysis, qPCR for vector. copy number, LAM-PCR and/or 454 sequencing; 2.The generation of a replication-competent lentivirus (RCL). 3.The safety of a low dose non-myeloablative conditioning regimen

Secondary Outcome Measures:

•the level of engraftment [ Time Frame: 2 years ] [ Designated as safety issue: No ]
of the genetically modified autologous CD34+ cells and expression of the transduced ß-globin gene as measured by: Blood for measurement of vector copy number and transgene expression in blood cells at months 1, 2 and 3.
- Bone Marrow Samples for evaluation of CFU, BFU-E gene+ colonies and Q-PCR will be tested at month 1.

•The frequency of post transplant palliative transfusions [ Time Frame: 2 years ] [ Designated as safety issue: No ]
The number of red blood cell transfusions after engraftment will be quantitatively evaluated for efficacy purposes.


Estimated Enrollment:   10
Study Start Date:   July 2012
Estimated Study Completion Date:   July 2014
Estimated Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
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Rajiv
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« Reply #122 on: March 07, 2014, 09:17:11 AM »

Dear Canadian family,
thanks a lot for the update.
Valuable info.  Always pray to GOD to give us a permanent solution to this problem.
May GOD bless all patients and friends by giving this cure.
Best regards,
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Sharmin
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Little A


« Reply #123 on: March 07, 2014, 06:08:08 PM »

Canadian Family,

Thank you kindly for summarizing the information and sharing it with us. 

Best,

Sharmin
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Sharmin
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« Reply #124 on: April 22, 2014, 03:25:41 AM »

Any inside news on these trials...please share.

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