Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: CrazyPharm on November 04, 2015, 08:23:43 PM



Title: Phase 3 Luspatercept Trial
Post by: CrazyPharm on November 04, 2015, 08:23:43 PM
Hi everyone! I didn't see any threads about the new Luspatercept trial so I figured let me pass some info. along and get a discussion going since I am so excited. Recently Acceleron and Celgene announced they are ready to progress their Luspatercept program into Phase 3 Clinical trials. This means once this trial is over and the results are good they can submit to the FDA for drug approval.

Recap: Luspatercept is a subcutaneous injection given once every 3 weeks to help reduce the blood transfusion burden in thalassemia patients. Phase 2 results have been excellent with minimal side effects and all evaluable patients showing greater than 40% reduction in blood transfusion requirement (transfusion dependant patients).

The phase 3 trial is titled the "BELIEVE" trial and is scheduled to start by the end of this year, 2015. Patients who are regularly transfused (8-20 units per 24 weeks) will be eligible to participate. 200 patients will receive luspatercept 1 mg/kg SubQ while 100 patients will receive placebo (non-active injection). Neither patient nor investigator will know who is receiving drug or placebo. The trial is therefore random and double-blinded.

No details yet on where the trial will be offered but I have been told it is a global trial. Stay alert for more updates. If any one has any further to add please do so :)


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on November 07, 2015, 12:25:30 PM
I can't see why a double blind trial is being used. It's not like a placebo effect is going to raise the Hb level.


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on November 07, 2015, 03:17:57 PM
I can't see why a double blind trial is being used. It's not like a placebo effect is going to raise the Hb level.

I absolutely agree with you. I think it complicates things and adds undue harm to the placebo recipient since they will wait for hgb to drop to a specific level before transfusing most likely 1 unit at a time. There will be at least weekly cbc to monitor hgb. For 48 weeks for a patient to get placebo and undergo all this is tortourous (being a patient myself).

The only logic I can see in a placebo trial is to compare side effects. If placebe thalassemia patients complain of headache and bone pain more so than the general population it may help to discredit these side effects being attributed to the drug itself. I can't imagine why a patient would stick it out for 48 weeks when one would realize by week 5-6 that obviously the drug isn't being administered since blood requirement would remain the same and quality of life worse than before.


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on November 07, 2015, 05:52:59 PM
All good points. And something expressed to me is that not knowing if they're getting the drug is keeping patients from joining trials.


Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on November 08, 2015, 12:09:33 PM
I have been waiting to enrol lil A in this trial for a long time.  I understand why it may be helpful to make this trial double blind, but for my son - who has an antibody which is active some times and not others - his participation in the study may skew the results of the study is conducted double blind.  His results can only be compared to himself - how he did in a given year with and without luspatercept. 


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on November 11, 2015, 10:53:04 PM
I have been waiting to enrol lil A in this trial for a long time.  I understand why it may be helpful to make this trial double blind, but for my son - who has an antibody which is active some times and not others - his participation in the study may skew the results of the study is conducted double blind.  His results can only be compared to himself - how he did in a given year with and without luspatercept. 

Hi Sharmin, how old is your son? I hope the trial is flexible and gets under way quickly.


Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on November 12, 2015, 12:15:38 AM
Hello CrazyPharm,

I agree, this medication, if it turns out to be what it seems will improve the quality of life of thals. 

My son is 17. I think participants are required to be 18+ years of age.


Title: Re: Phase 3 Luspatercept Trial
Post by: Canadian_Family on November 12, 2015, 07:35:23 AM
Where are these trials conducted.

Sharmin,

Little A is now 17, amazing, how fast they grow. I remember talking about him on the forum when he was six (I believe). You certainly have done an excellent job.



Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on November 15, 2015, 12:14:18 AM
The trials will be conducted at multiple centres Canadian Family.  Oakland may be involved in the study.  I will keep you posted.  By next year all of our kids may have the option to be on this medication which may eliminate or greatly reduce their transfusion requirement. 

Time has passed by so quickly.  Lil A is preparing for university.  I hope lil miss A is doing well also.

Hoping for the best,

Sharmin


Title: Re: Phase 3 Luspatercept Trial
Post by: Canadian_Family on November 15, 2015, 05:33:49 PM
Thanks for the information. Good Luck to young man, wishing him best of luck in everything.

Little Miss A is 12 now and in grade 7. She is a happy kid and interested in arts.



Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on November 18, 2015, 09:15:07 PM
Glad to hear that Canadian Family!


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on December 09, 2015, 07:24:45 AM
Celgene and Acceleron Announce New Results from an Investigational Study with Luspatercept in Beta-Thalassemia Presented at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition

SUMMIT, N.J. & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced preliminary results from two Phase 2 clinical trials of luspatercept in patients with beta-thalassemia were presented at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition. Results highlighted in an oral presentation showed that luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life measures and had beneficial effects on liver iron concentration in patients with beta-thalassemia. Celgene and Acceleron are jointly developing luspatercept.

"These luspatercept results are very exciting as they show positive effects across a range of clinically challenging complications of beta-thalassemia," said Professor Antonio Piga, M.D., Ph.D., Director of Pediatrics at San Luigi Gonzaga University Hospital in Torino, Italy and coordinating principal investigator of the study. "A therapy that could potentially treat the anemia, complications of beta-thalassemia, such as iron overload, and improve measures of health-related quality of life would be a huge advance to help address the substantial unmet need of patients with beta-thalassemia."

Luspatercept Data Presented at ASH

Luspatercept was evaluated in a Phase 2, multicenter, open-label study in adults with non-transfusion-dependent (NTD) and transfusion-dependent (TD) beta-thalassemia patients. The primary objectives were to assess the proportion of NTD patients that achieved a hemoglobin increase ≥ 1.5 g/dL from baseline and the proportion of TD patients that achieved at least a 20% reduction in transfusion burden. A total of 64 patients, of which 59 were efficacy evaluable (31 NTD and 28 TD), were enrolled in the dose escalation and expansion stages of the Phase 2 clinical trial. In this study, patients received up to 5 doses via subcutaneous injection once every 3 weeks. 51 of the 64 patients from this 3-month study enrolled in the long-term Phase 2 extension trial in which these patients may receive up to two years of treatment with luspatercept. Data was presented from both the 3-month study and the long-term extension study.

Improvement of anemia and transfusion burden:

Of the 17 NTD patients that received at least 5 cycles of luspatercept at dose levels of 0.8 mg/kg or higher
65% (11/17) increased hemoglobin levels ≥ 1.0 g/dL over a 12-week period
47% (8/17) increased hemoglobin levels ≥ 1.5 g/dL over a 12-week period
Data presented showed sustained increases in hemoglobin with the longest-treated patients having received nearly six months of luspatercept
Of the 28 TD patients
79% (22/28) patients had ≥ 20% reduction of transfusion burden
75% (21/28) patients had ≥ 33% reduction of transfusion burden
57% (16/28) patients had ≥ 50% reduction of transfusion burden
Changes in iron overload:

Liver iron concentration (LIC), a measure of iron overload, was maintained or reduced in both non-transfusion dependent and transfusion-dependent patients
50% (4/8) TD patients with baseline LIC ≥ 5 mg/g dry weight (dw) had decrease in LIC ≥ 2 mg/g dw
100% (14/14) TD patients with baseline LIC &#60 5 mg/g dw maintained LIC &#60 5 mg/g dw
36% (5/14) NTD patients with baseline LIC ≥ 5 mg/g dw had decrease in LIC ≥ 2 mg/g dw
100% (14/14) NTD patients with baseline LIC &#60 5 mg/g dw maintained LIC &#60 5 mg/g dw
Improvement in health-related quality of life (QoL) measures in NTD patients:

Improved health-related QoL (FACT-An anemia subscore) correlated with increases in hemoglobin in NTD Patients
Safety:

The most common related adverse events were bone pain, myalgia, headache, arthralgia, musculoskeletal pain, asthenia, injection site pain, back pain and pain in jaw
Related grade 3 adverse events included headache, bone pain, asthenia, and myalgia
There were no drug-related serious adverse events
Celgene and Acceleron are in the process of initiating a global Phase 3 study in regularly transfused beta-thalassemia patients.

Luspatercept is an investigational product that is not approved for any use in any country.


Title: Re: Phase 3 Luspatercept Trial
Post by: catchR on December 14, 2015, 06:38:44 AM
thanks Parin for the update!!

these are very good results...hope this becomes available to patients soon...


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on December 30, 2015, 08:13:51 AM
Dec 30th and still not started... tsk tsk :wah


Title: Re: Phase 3 Luspatercept Trial
Post by: nice friend on January 05, 2016, 05:39:50 PM
wat is the procedure and requirement to be a part of this trial ??


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on January 05, 2016, 06:22:32 PM
There's still no update about when or where the trials will start to recruit patients.

https://clinicaltrials.gov/ct2/show/NCT02604433?term=luspatercept&rank=1


Title: Re: Phase 3 Luspatercept Trial
Post by: nice friend on January 14, 2016, 04:06:20 AM
Thanx for the link , Andy .... here is something what the site say regarding recruitment :
Quote
This study is not yet open for participant recruitment.


Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on January 14, 2016, 08:31:46 AM
My understanding is that trials may start this spring.  The trials will be double blind.  2/3 of the patients will reveive Luspatercept and 1/3 a placebo.  Patients who are selected will be ones who live in close proximity to the clinic from which the trials are being run because they will be required to attend the centre every 3 weeks for repeat doses and assessment. 


Title: Re: Phase 3 Luspatercept Trial
Post by: nice friend on January 14, 2016, 09:05:28 AM
Sharmin,
it mean's no chance for me to participate in this do or die attempt :D ...


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on January 14, 2016, 11:10:13 AM
We have to wait and see where trials are run, but it is expected that this drug will not take a long time to reach the market once trials are concluded.


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on January 20, 2016, 08:30:41 AM
Do you think the trial will last 3 years or will they conclude after the 48 weeks period? I thought this trial was only going to be for a year. It says trial completion 2019 :wah


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on January 20, 2016, 11:02:31 PM
It probably means trials in more than one location starting at different times. There would be no need for an individual trial longer than one year.


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on January 29, 2016, 09:56:41 AM
so that means we have to wait til 2019 for approval? :-\

btw.. whats with the delay?? they said trial would start "end year 2015" :huh


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on January 30, 2016, 12:07:30 PM
I have emailed the Associate Director of the Clinical Trial for Luspatercept. I will report if I receive any reply.


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on February 01, 2016, 12:03:15 AM
Thank you Andy for follow up....



Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on February 01, 2016, 10:30:05 AM
Thank you for looking into that Andy, this study is of great interest to everyone.  If it works as well as the potential it has shown thus far - it will revolutionize the treatment of thalassemia.  Reducing transfusions, depleting iron stores - it is the answer to so many problems.

Sharmin


Title: Re: Phase 3 Luspatercept Trial
Post by: catchR on February 03, 2016, 06:06:39 AM
thanks Andy for following up! this one has great potential for all of thals...


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on March 02, 2016, 10:49:10 AM
Trial locations have been updated massive trial! Looks like it is ready to start soon! Good news


Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on March 02, 2016, 07:51:37 PM
That's wonderful CrazyPharm!  I'm happy for all thals.  It's amazing that this treatment not only has the potential to reduce transfusions - utilizing iron stores but it also reverses so many of the negative effects of thalassemia.   Let's hope that by this time next year we know that the drug works like a charm and that more thals are preparing to transition to this treatment. 



Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on April 02, 2016, 10:56:10 AM
Update:

Trial has started in Childrens hospital Los Angeles. Excellent news I hope the trial goes underway in FULL very soon. We need this drug ASAP


Title: Re: Phase 3 Luspatercept Trial
Post by: Sharmin on April 03, 2016, 05:57:12 PM
Thank you for sharing CrazyPharm,

I'm very happy to hear that.  Praying for the best.  


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on April 04, 2016, 01:26:46 AM
Thank you Sharin....its a great news..



Title: Re: Phase 3 Luspatercept Trial
Post by: Lokkhi maa on April 04, 2016, 04:34:44 AM

Thanks Sharmin.

All of we are eagerly waiting for the drug.. :grouphug


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on June 10, 2016, 04:02:03 PM
Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Myelodysplastic Syndromes at the 21st Congress of the European Hematology Association

http://investor.acceleronpharma.com/releasedetail.cfm?ReleaseID=975231

June 10, 2016
Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Myelodysplastic Syndromes at the 21st Congress of the European Hematology Association
- Preliminary results show that treatment with investigational drug luspatercept results in clinically meaningful increases in hemoglobin and durable transfusion independence in patients with lower risk myelodysplastic syndromes -

- Acceleron to host conference call and live webcast today at 8:00 a.m. EDT (2:00 p.m. CEST) -

CAMBRIDGE, Mass. & SUMMIT, N.J.--(BUSINESS WIRE)-- Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation (NASDAQ: CELG), today announced preliminary results from an ongoing long-term Phase 2 extension study with luspatercept in patients with lower risk myelodysplastic syndromes (MDS) at the 21st Congress of the European Hematology Association (EHA) in Copenhagen, Denmark. Results highlighted in an oral presentation showed that 51% of patients with lower risk MDS treated with luspatercept (n=49) achieved increased hemoglobin levels and 35% of patients achieved transfusion independence in the 3-month base study. In the ongoing extension study, 81% (26/32) of patients had increased hemoglobin levels and of the patients eligible for transfusion independence (TI), 50% achieved TI with luspatercept treatment. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

"The results for luspatercept in lower risk MDS patients are increasingly encouraging as we gain longer term safety and efficacy experience with this agent," said Uwe Platzbecker, M.D., Professor of Hematology and Head of the MDS program at the University Hospital in Dresden, Germany. "There is a significant unmet need for new therapies that reduce the number of or eliminate the need for blood transfusions."

Highlights of the Luspatercept MDS Phase 2 Data Presented at EHA

Study Design

Data from two Phase 2 studies were presented at the conference: the completed dose-escalation study in which patients received treatment with luspatercept for three months and the ongoing long-term extension study in which patients receive treatment with luspatercept for an additional 24 months. In both the 3-month base study and the long-term extension study, high transfusion burden patients (≥ 4 units RBC / 8 weeks) and low transfusion burden patients ( < 4 units RBC / 8 weeks) were enrolled and treated with open-label luspatercept, dosed subcutaneously once every 3 weeks. The primary outcome measure for the 3 month study was the proportion of patients who had an erythroid response. Erythroid response was defined as hemoglobin ≥ 1.5 g/dL from baseline for ≥ 14 days in non-transfusion dependent patients or a reduction of either ≥ 4 units or ≥ 50% of units of RBCs transfused compared to pretreatment in transfusion-dependent patients. The primary outcome for the long-term extension study is to evaluate the long-term safety and tolerability of luspatercept with low or intermediate-1 risk MDS who were previously enrolled in the 3-month study.

Efficacy

              
        Response rate (% of patients)   
    
3-month base study
(n=49, higher dose
levels)
    
Long-term extension
study
(n=32)
International Working Group Hematologic
Improvement-Erythroid (IWG HI-E)
Response Rate (reduction of ≥4 units RBC / 8
weeks or a hemoglobin increase ≥1.5 g/dL ≥ 8
weeks)
    51% (25/49)       81% (26/32)   
RBC Transfusion Independence (RBC-TI)
Response Rate (Transfusion free ≥ 8 weeks for
patients with ≥ 2 units RBC / 8 weeks prior to
treatment)
    35% (14/40)       50% (11/22)   
Duration of RBC-TI               Range: 9-80+ weeks   
       
For reference, results presented six months ago at the American Society of Hematology (ASH) annual meeting in December 2015 were as follows:

IWG HI-E response rate was 69% (22/32)
RBC-TI response rate was 50% (11/22)
Duration of RBC-TI ranged from 9 to 50+ weeks
Safety

There were three grade 3 adverse events possibly/probably related to study drug (blast cell count increase, myalgia and worsening of general condition).
Adverse events at least possibly related to study drug that occurred in at least 2 patients during studies were fatigue, bone pain, diarrhea, myalgia, headache, hypertension and injection site erythema.
Luspatercept is an investigational product that is not approved for use in any country.

The MEDALIST Trial, a global Phase 3 study in patients with very low, low, or intermediate risk, MDS with ring sideroblasts who require red blood cell transfusions, is currently enrolling.

The slides from this oral presentation are available on Acceleron's website (www.acceleronpharma.com) under the Science tab.


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on June 11, 2016, 11:59:21 AM
The Phase 3 trials are currently recruiting. More info can be found at https://clinicaltrials.gov/ct2/show/NCT02604433?term=luspatercept&rank=1


Title: Re: Phase 3 Luspatercept Trial
Post by: BabyRiya on July 30, 2016, 08:24:04 PM
Friends
If everything comes out good, will it open doors for kids/infants? Or it's limited to adult only?


Title: Re: Phase 3 Luspatercept Trial
Post by: sofear on July 30, 2016, 10:43:40 PM
Is it really going to take as long as the 2020's or something until Luspatercept will be available? That's a damn long time.


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on July 31, 2016, 06:19:19 PM
The phase 3 trials will answer a lot of questions. I think it will be available to children once approved.


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on December 07, 2016, 12:31:46 AM
Acceleron and Celgene Announce Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia Presented at the 58th Annual Meeting of the American Society of Hematology

"Beta-thalassemia is a severe, chronic disease with no pharmaceutical treatment options to correct or improve the underlying anemia in patients," said Michael Pehl, President, Hematology and Oncology for Celgene. "These longer term luspatercept Phase 2 data are encouraging, and we are continuing to enroll patients in the Phase 3 BELIEVE study in transfusion dependent beta-thalassemia patients."

Luspatercept Beta-Thalassemia Data Presented at ASH

Results in Transfusion Dependent (TD) Beta-Thalassemia Patients

    
RBC transfusion reduction
over any 12 weeks versus 12
weeks pre-treatment
        Response rate (% of patients)
            3-month base study
(n=31)
        Long-term extension study
(n=24)
    ≥ 20%           81% (25/31)           96% (23/24)
    ≥ 33%           71% (22/31)           83% (20/24)
    ≥ 50%           55% (17/31)           71% (17/24)
               
Results in Non-Transfusion Dependent (NTD) Beta-Thalassemia Patients

    
Hemoglobin (Hb) response over
any 12 weeks versus 12 weeks
pre-treatment
        Response rate (% of patients)
in patients treated with ≥ 0.6 mg/kg
            3-month base study
(n=21)
        Long-term extension study
(n=27)
    Increase in mean Hb ≥ 1.0 g/dL           62% (14/21)           78% (21/27)
    Increase in mean Hb ≥ 1.5 g/dL           33% (7/21)           52% (14/27)
               
In the long-term extension study, the median duration of a hemoglobin increase ≥ 1.0 g/dL maintained for at least 12 weeks in responders is 13.5 months (N=21) with treatment still ongoing.


Title: Re: Phase 3 Luspatercept Trial
Post by: Hafiz Akhtar on December 08, 2016, 01:25:31 AM
for how long phase 3 wi
ll continue. Andy Sir what will u suggest the tentative time for its approval.


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on December 08, 2016, 10:43:11 AM
Trials will be taking place in many different areas over a period of time, as patients are recruited. My educated guess is that we will see this drug on the market within 5 years.


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on December 16, 2016, 05:04:38 AM
I have tried to enroll into the trials but was rejected the day it opened in my city because quota is already full. unbelievable... :huh.. i've been watching this drug ready to jump on the trial since its early phase 2 days. bummer i cant be a part i hope for a fast approval :wah


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on December 16, 2016, 12:01:47 PM
Check the recent post by Parin.

Quote
bluebird bio is also moving forward with plans to initiate Northstar-3 (HGB-212), a Phase 3 trial of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia with the β0/β0 genotype. This study will also be conducted under the new manufacturing process, and is expected to begin enrolling patients in 2017. The primary endpoint of this planned study is transfusion reduction.


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on December 16, 2016, 05:30:36 PM
Thank you Andy for valuable information.


Title: Re: Phase 3 Luspatercept Trial
Post by: sofear on January 14, 2017, 06:22:09 AM
Short question that came up: why are they never releasing detailed blood test results? Do they only mention the 'transfusion reduction' because that is also the current goal for their studies? I think it would be very helpful to see which effects the drug has on many other blood values and therefore on the body as a whole.


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on January 14, 2017, 06:24:22 PM
The goal is raising total Hb, so that's what gets reported. They may be following other values, as well, but in the context of trials, the goal is specific. I am curious if this would raise MCV levels. I imagine it would.


Title: Re: Phase 3 Luspatercept Trial
Post by: sofear on March 06, 2017, 02:30:53 PM
I am curious if this would raise MCV levels. I imagine it would.

I feel like that probably won't be the case. I just had a look at some older power points of Luspatercept (like this one (http://"https://celerion.com/wordpress/wp-content/uploads/2012/12/CelerionAcceleron_ACE-536-Increases-Hemoglobin-in-Healthy_ASH_124121.pdf)) and it seems that the increase in hemoglobin always happens with quite a large increase in the RBC count. Why is no one talking about the MCV that always seems to be low in thalassemia patients? We have multiple options to increase the RBC count, that is nothing new.



Title: Re: Phase 3 Luspatercept Trial
Post by: sofear on May 03, 2017, 01:01:25 PM
I'm not sure whether it has yet been posted, but I recently stumbled upon a nice animation of the working mechanism of Luspatercept:

https://www.youtube.com/watch?v=oPfig2aOIYw

(I'm not sure how accurate it is, though, but it's pretty cool to watch.)


Title: Re: Phase 3 Luspatercept Trial
Post by: Manal on May 03, 2017, 01:45:44 PM
I met Dr. Piga last November in Jordan ( pan middle east conference ) and he said that he expects Laucpatercept to be available in the market in 3 years and he added that in few months they will start the pediatric clinical trails.

Manal


Title: Re: Phase 3 Luspatercept Trial
Post by: sofear on May 05, 2017, 06:42:46 AM
After I saw the animation of the mechanism of action of Luspatercept, I tried to understand it a bit better and went through a lot of videos and articles about basic biological topics but also thalassemia-specific ones.

Although I'm not able to judge the quality of the content, I later found a site with articles about thalassemias of which some were really interesting to read:

http://cursoenarm.net/UPTODATE/contents/search.htm?search=thalassemia&menu=0&submitType=Search

One part of an article (http://cursoenarm.net/UPTODATE/contents/mobipreview.htm?29/32/30208) had the following passage:

Quote
Normal hemoglobin biosynthesis requires an intact structural gene and the structural and spatial integrity of silencers, enhancers, promoters, and LCR sequences. Despite considerable effort, we do not fully understand how the sequences and factors regulating globin gene expression interact with one another to ensure high levels of expression of the proper globins at the proper developmental stage and during the proper steps of erythroid differentiation.

This information has been last updated in 2011, so I'm not sure how up-to-date it still is, but the question that came to my mind was:

How are the processes of red blood cell differentiation and maturation, which Luspatercept interacts with, actually linked to the expression of the globin genes?



Title: Re: Phase 3 Luspatercept Trial
Post by: Hafiz Akhtar on May 10, 2017, 12:44:10 AM
Andy Sir what is ur opinion in this regard. can v say that it will b approved & commercialized within 3 years and our children 2 some extent will be free from transfusion. i mean to say burden of transfusion will b reduced. perhaps our dream will come true.


Title: Re: Phase 3 Luspatercept Trial
Post by: anshul on May 15, 2018, 12:05:39 PM
Is there any update on luspatercept trials?


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on May 15, 2018, 07:03:36 PM
Only comments from patients in the trials. Some are doing very well. I don't anticipate any official findings until trials conclude.


Title: Re: Phase 3 Luspatercept Trial
Post by: CrazyPharm on July 11, 2018, 12:31:44 AM
LUSPATERCEPT is a success! Phase 3 primary and secondary outcomes were positive as reported on July 9. FDA submission will be during first half of 2019 so we could potentially be using Luspatercept in Summer/Early Fall 2019.  :biggrin good news! Congrats and best wishes to all


Title: Re: Phase 3 Luspatercept Trial
Post by: Parin on July 11, 2018, 01:03:31 AM
Great News..thanks for sharing


Title: Re: Phase 3 Luspatercept Trial
Post by: Andy Battaglia on July 14, 2018, 03:28:28 PM
We have gotten two very positive reports about Luspatercept in the past two weeks. The fact that they are seeking  approval demonstrates that they believe it works well enough to be a significant factor in treating thalassemia.


Title: Re: Phase 3 Luspatercept Trial
Post by: Lokkhi maa on July 17, 2018, 11:43:22 AM
 :thumbsup