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Post by Deleted on Nov 10, 2017 8:13:03 GMT -5
I thought this was posted but could not locate it. If this is a duplication then delete. -To assess the safety and tolerability of Afrezza in children ages 4 to 17 years with type 1 diabetes mellitus (T1DM). Estimated Enrollment: 46 Detailed Description: The patients are expected to participate in the study for approximately 6 to 8 weeks from Screening to final follow-up visit. Patients who completed 4 weeks of Afrezza treatment and have shown to be safe and well controlled with Afrezza + basal insulin will have the option to continue the extension treatment up to 1 year. clinicaltrials.gov/ct2/show/NCT02527265?term=Afrezza&recrs=ab&draw=1&rank=2 |
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Post by Deleted on Jun 9, 2018 17:31:05 GMT -5
Rising pointed out something interesting.
Up until March 26, it said:
"The patients are expected to participate in the study for approximately 6 to 8 weeks from Screening to final follow-up visit.
Patients who completed 4 weeks of Afrezza treatment and have shown to be safe and well controlled with Afrezza + basal insulin will have the option to continue the extension treatment up to 1 year."
But as of May 18, it says only:
"The patients are expected to participate in the study for approximately 6 to 8 weeks from Screening to final follow-up visit."
Why would they remove the option to extend the treatment if the patient wanted it?
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Is it possible the trial will be completed in less than a year?
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Post by Deleted on Jun 10, 2018 9:53:43 GMT -5
This is a phase 2 trial, when completed and if primary and secondary endpoints are met, is a phase 3 trial required?
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Post by agedhippie on Jun 10, 2018 10:08:50 GMT -5
This is a phase 2 trial, when completed and if primary and secondary endpoints are met, is a phase 3 trial required? I am not sure, but the role of Phase 2 trials is to see if the drug works and has any side effects on the target population, pediatric diabetics in this case. Phase 1 is usually done on a healthy group. If you look at the trial the primary endpoints are adverse events and hypoglycemia, but not effectiveness which is phase 3. In this case since there has been an adult phase 3 trial the pediatric phase 3 may be unnecessary. |
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Post by akemp3000 on Jun 10, 2018 10:38:15 GMT -5
In very general terms, Phase 1 is about safety, Phase 2 is about efficacy and Phase 3 is about a larger population study. It does seem like they could waive Phase 3 given the importance of this for pediatrics. It's good to now have Dr. Kendall making this case. Let's hope.
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Post by sportsrancho on Jun 10, 2018 10:45:26 GMT -5
In very general terms, Phase 1 is about safety, Phase 2 is about efficacy and Phase 3 is about a larger population study. It does seem like they could waive Phase 3 given the importance of this for pediatrics. It's good to now have Dr. Kendall making this case. Let's hope. If that’s the case what’s the timeframe on that? I have parents asking me, and also a woman who is trying to get her son into the trial right now. |
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Post by Deleted on Jun 10, 2018 12:01:30 GMT -5
Statistics Some 1.25 million Americans are living with T1D, including about 200,000 youth (less than 20 years old) and more than 1 million adults (20 years old and older). 40,000 people are diagnosed each year in the U.S. 5 million people in the U.S. are expected to have T1D by 2050, including nearly 600,000 youth.
www.jdrf.org/about/what-is-t1d/facts/-------------------------------------------- While there are no repercussions with denials for adults I suspect it willnot be the case for children. In my opinion Afrezza's greatest potential lies with children. |
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Post by akemp3000 on Jun 10, 2018 14:04:05 GMT -5
In very general terms, Phase 1 is about safety, Phase 2 is about efficacy and Phase 3 is about a larger population study. It does seem like they could waive Phase 3 given the importance of this for pediatrics. It's good to now have Dr. Kendall making this case. Let's hope. If that’s the case what’s the timeframe on that? I have parents asking me, and also a woman who is trying to get her son into the trial right now. The following shows the averages for all drugs, not specific to pediatrics...and it's probably important to consider there's nothing "average" about MNKD  Expenses: Phase I: $15.2 million Phase II:: $23.4 million Phase III: $86.5 million Time: Phase I: 21.6 months Phase II: 25.7 months Phase III: 30.5 months Overall probability of success: Phase I: 30% Phase II: 14% Phase III: 9% Conditional probability of success: Phase I: 75% Phase II: 48% Phase III: 64% “Overall probability of success” is the unconditional probability of reaching a given stage. For example, 30 percent of drugs make it to phase I testing. “Conditional probability of success” shows the probability of advancing to the next stage of the process conditional on reaching a given stage. For example, the probability of advancing to Phase III testing conditional on starting Phase II testing is 48 percent. |
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Post by Deleted on Jun 10, 2018 14:36:33 GMT -5
akemp3000 those statistics apply for drugs without approval. Given Afrezza is approved for adults and contains an identical human hormone, the probability of success for pediatrics is 100%.
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Post by peppy on Jun 10, 2018 14:46:17 GMT -5
Rising pointed out something interesting. Up until March 26, it said: "The patients are expected to participate in the study for approximately 6 to 8 weeks from Screening to final follow-up visit. Patients who completed 4 weeks of Afrezza treatment and have shown to be safe and well controlled with Afrezza + basal insulin will have the option to continue the extension treatment up to 1 year." But as of May 18, it says only: "The patients are expected to participate in the study for approximately 6 to 8 weeks from Screening to final follow-up visit." Why would they remove the option to extend the treatment if the patient wanted it? ----------------------------------- Is it possible the trial will be completed in less than a year?  I had to turn on the exhaust fan. Oh, that smell. |
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Post by Deleted on Jun 11, 2018 0:30:03 GMT -5
Statistics Some 1.25 million Americans are living with T1D, including about 200,000 youth (less than 20 years old) and more than 1 million adults (20 years old and older). 40,000 people are diagnosed each year in the U.S. 5 million people in the U.S. are expected to have T1D by 2050, including nearly 600,000 youth.
www.jdrf.org/about/what-is-t1d/facts/-------------------------------------------- While there are no repercussions with denials for adults I suspect it willnot be the case for children. In my opinion Afrezza's greatest potential lies with children. Yes children are the best target rich environment because they have no allegiance to anyone other than their parents and the parents want an easy effective way of managing their child's BG Levels. Afrezza is the answer. |
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Post by Deleted on Jun 11, 2018 0:40:22 GMT -5
If the above theories are true it will be VERY TOUGH to predict WHEN and IF the PEDS TRIAL will be approved. Unless Mike updates at the next investors conference.
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Post by peppy on Jun 11, 2018 8:02:05 GMT -5
If the above theories are true it will be VERY TOUGH to predict WHEN and IF the PEDS TRIAL will be approved. Unless Mike updates at the next investors conference. the MNKD chart saids, the pediatric trial has started. www.screencast.com/t/Grz7ASiiyWhat is going on in this thread? this thread started with the words, "I thought" |
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Post by boca1girl on Jun 11, 2018 8:11:29 GMT -5
If the above theories are true it will be VERY TOUGH to predict WHEN and IF the PEDS TRIAL will be approved. Unless Mike updates at the next investors conference. the MNKD chart saids, the pediatric trial has started. www.screencast.com/t/Grz7ASiiyWhat is going on in this thread? this thread started with the words, "I thought" The question was if the trials could END earlier than expected. |
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Post by mnholdem on Jun 11, 2018 8:25:46 GMT -5
ClinicalTrials.gov Identifier: NCT02527265
Recruitment Status: Recruiting
First Posted: August 18, 2015
Last Update Posted: May 16, 2018
Study Design
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 46 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label, Single-arm, Multiple-dose Safety, Titration, and Pharmacokinetic Trial of Afrezza® in Pediatric Patients Ages 4 to 17 Years With Type 1 Diabetes Mellitus
Actual Study Start Date: September 28, 2017
Estimated Primary Completion Date: July 2020
Estimated Study Completion Date: January 2021
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This study design was posted in 2015 when MannKind had a different CEO and Chief Medical Officer working with the FDA. With Michael Castagna PharmD promoted to CEO in May-2017, followed by the hiring of David Kendall MD as the company's new CMO, the trial design and estimated completion dates may have changed.
Even though the ClinicalTrials.gov site for this trial was last updated May-2018, MannKind may not have revised the estimated completion dates on this filing, even though the graphics presented at the ASM indicate that the trial may be completed way ahead of schedule.
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