Pediatric Trials Started?? - NM - has already been mentioned

[boytroy88]

Saw this on ST - clinicaltrials.gov/ct2/show/NCT02527265?term=sar439065&rank=2

Says that they are enrolling patients.  I haven't read up on it but I'll probably get things wrong since I don't have a medical background...does this give us at least some hint what SNY will or will not do in January??

EDIT:  Sorry...I guess this has already been mentioned....

[peppy]

scroll down
Estimated Enrollment: 46
Study Start Date: October 2015
Estimated Study Completion Date: August 2017

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/study/NCT02527265?term=sar439065&rank=2

Phase 1
Primary Outcome Measures: •Number of patients with adverse events [ Time Frame: up to 1 week after last treatment ] [ Designated as safety issue: Yes ]

•Number of patients with hypoglycemic events [ Time Frame: up to 1 week after last treatment ] [ Designated as safety issue: Yes ]



Secondary Outcome Measures: •insulin maximum observed concentration (Cmax) [ Time Frame: 1 day ] [ Designated as safety issue: No ]

•insulin time to reach Cmax (tmax) [ Time Frame: 1 day ] [ Designated as safety issue: No ]

•insulin area under concentration time curve (AUC) [ Time Frame: 1 day ] [ Designated as safety issue: No ]

•insulin apparent clearance (CL/F) [ Time Frame: 1 day ] [ Designated as safety issue: No ]

•insulin apparent volume of distribution (Vss/F) [ Time Frame: 1 day ] [ Designated as safety issue: No ]

•Assessment of fumaryl diketopiperazine (FDKP) elimination half-life (t1/2) [ Time Frame: 1 day ] [ Designated as safety issue: No ]

•Number of patients adhered to dose titration rules [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]

•Number of patients completed the scheduled treatment period [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]

•Assessment of patient's ability to use Afrezza inhalation device [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]

•Measurement of anti-insulin antibodies [ Time Frame: Up to 13 months ] [ Designated as safety issue: Yes ]

[lakers]

Primary Objective:

-To assess the safety and tolerability of Afrezza in children ages 4 to 17 years with type 1 diabetes mellitus (T1DM).

Secondary Objectives:

To assess the ability to titrate the prandial and supplemental doses of Afrezza at each meal.

[This coincides w/ meeting in SD last week w/ early adopters on titration and suppl doses, Dr Steve Edelman's Sanofi sponsored TCOY/CME traing tour
Incl titration guidance, and Ultra RA patent on split doses]

mnkd.proboards.com/thread/4459/hilton-santa-clara-ultra-afrezza

To assess pharmacokinetics (PK) following a prandial dose of Afrezza in children ages 4 to 17 years with T1DM.

The Pediatric Trial from beginning to end takes 8 weeks. All measurement except one can be completed after 30 days. Only one long pole measurement is anti-insulin antibodies which can be up to 13 mos. So if all go well, the Phase 1 trial can finish in much less than a year.

MNholdem said:
The FDA wants a 2-part pediatric trial, with the Part 1 for type 1 diabetes and Part 2 for types 1 or 2. Part 2 of the trial includes a 52-week randomized intervention phase.

Here is the FDA description of the post-market requirement:

An open-label pharmacokinetic (PK), and multiple-dose safety and tolerability dose-titration trial of Afrezza in pediatric patients ages 4 to 17 years (inclusive) with type 1 diabetes (Part 1), followed by a prospective, multicenter, open-label, randomized, controlled trial comparing the efficacy and safety of prandial Afrezza to prandial subcutaneous insulin aspart used in combination with subcutaneous basal insulin in pediatric patients 4 to 17 years old (inclusive) with type 1 or type 2 diabetes (Part 2). Part 2 of the trial should include a 4-week run-in phase and a 52-week randomized intervention phase.

Sanofi has not published nor have they begun recruitment of a trial that studies the FDA requirements for Part 2 studies, but it looks to me that in order to meet your estimate of completing the pediatric trial(s) by August 2017, they would have to recruit and start the Part 2 pediatric trial by May/June 2016 at the latest.

Read more: mnkd.proboards.com/thread/3511/afrezza-pediatrics-trial-recruiting?page=3#ixzz3uEe4medY

[dudley]

We can only hope that these trials are structured to allow Afrezza to be used as it SHOULD - i.e. after the meal begins, and NOT well before the meal as injections require and as the Affinity trials were forced to do. If structured properly this trial alone should show clear superiority over subcutaneous : Lower hypo events, better sugar control etc. I can't see anywhere how the dosages are supposed to be taken. If it is just a repeat of the same protocols required in the Affinity trials the outcome will be the same - non-inferiority. It is not clear how the dosages will be administered. Hopefully Sanofi has some pull and can get the trial structured to clearly show superiority, as it will if dosages are administered properly. That would grease the skids toward a label change trial. Fingers crossed.

[peppy]

Dec 13, 2015 19:57:51 GMT -5 dudley said:

We can only hope that these trials are structured to allow Afrezza to be used as it SHOULD - i.e. after the meal begins, and NOT well before the meal as injections require and as the Affinity trials were forced to do. If structured properly this trial alone should show clear superiority over subcutaneous : Lower hypo events, better sugar control etc. I can't see anywhere how the dosages are supposed to be taken. If it is just a repeat of the same protocols required in the Affinity trials the outcome will be the same - non-inferiority. It is not clear how the dosages will be administered. Hopefully Sanofi has some pull and can get the trial structured to clearly show superiority, as it will if dosages are administered properly. That would grease the skids toward a label change trial. Fingers crossed.

trial 171 dosing times

screencast.com/t/zy3GnRAw

I am glad you are posting again.

In the pediatric trial, all 46 trial participants are wearing CGMs.

[dudley]

Hi Peppy - thanks for posting this.  Do you mind sharing how you found it and if you are sure this is for the Afrezza trial?  If so that is very good news because these dosing times seem to be much closer to the "optimum performance window" than the Affinity trials were and thus much better measurements should be demonstrated by the patients in this trial.  Many thanks.

[compound26]

Apparently the trial has started (probably sometime in October, Since Sam's message was dated 3 November).

[peppy]

Dec 14, 2015 12:59:55 GMT -5 dudley said:

Hi Peppy - thanks for posting this.  Do you mind sharing how you found it and if you are sure this is for the Afrezza trial?  If so that is very good news because these dosing times seem to be much closer to the "optimum performance window" than the Affinity trials were and thus much better measurements should be demonstrated by the patients in this trial.  Many thanks.

Tripoley posted the  study dosing times a couple of months ago . I snagged it.

Dosing time study 171 repost.

screencast.com/t/zy3GnRAw