Insmed Inc (INSM)

[rod5247]

Why would the MMD study design change? The original design was 0.5 mg for 8 weeks, 1.0 mg for 8 weeks, 2.0 mg for 8 weeks, then 24 weeks at optimal dose. The abstract to be presented May 4 used 0.5 mg for 8 weeks then 1.0 mg for 16 weeks. There are 5 more patients in this leg of the study whose data will be added upon completion. Is it probable that these remaining 5 will follow original dose escalation? If not, will they require another 24 week study for dose escalation to 2 mg? Did the researchers determine that 1.0 mg dose was optimal without further need to even test 2.0 mg? The number of patients in each study does not add up. Study design called for 15 in each leg. Update from January, 07 stated 2/3 had been identified which would represent 20 or more. Only 8 are announced in first study.

I guess the main question is, does the deviation in doseing and numbers indicate the study is progressing better than expected, worse than expected or nothing at all? It is hard for my layman mind to conclude that it means nothing. After 18 months there is 8 months of data on 3 patients. At this rate, how long will it take for UR to complete phase 2, large-scale, multicenter trial.

http://www.mdausa.org/research/view_ctrial.aspx?id=154

"STUDY DETAILS:
There will be two study phases, each involving 15 patients with type 1 MMD.

The investigators prefer patients who live in the greater Rochester, N.Y., area and have participated in the National Registry of Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Patients and Family Members: www.urmc.rochester.edu/nihregistry/

As of January 2007, approximately two-thirds of the participants had been identified.

For additional information, see www.clinicaltrials.gov, maintained by the National Institutes of Health, and enter SomatoKine in the search box.

Dose Escalation Phase

The first phase will be a 24-week study of SomatoKine/iPlex at three dosage levels: 0.5, 1 and 2 milligrams per kilogram of body weight, with each dose given daily for eight weeks.

Participants will be observed for side effects and effectiveness of the study compound at each dose so that investigators can identify an optimal dose.

Measures of effectiveness, taken at the start of the study and every eight weeks thereafter, include a dual energy X-ray absorptiometry (DEXA) scan, muscle strength testing and testing for myotonia (inability to relax muscles).

Five inpatient stays at the University of Rochester (N.Y.) Medical Center are required. The first three are during the six months in which the study drug will be administered. Each visit involves staying at the center for four nights and three days. Then, when the drug is stopped, participants must return for two more visits, each lasting two days and one night.

Outpatient visits are required every two weeks for routine blood tests and an assessment of side effects and injection sites.

These visits will take place at the University of Rochester for participants within driving distance, or at a local health care provider’s office for participants who live outside the Rochester area.

The results of the initial dose escalation study will guide the selection of the optimal dose.


Optimal Dose SomatoKine/iPlex Study

After determination of the optimal dose, there will be a 24-week trial of SomatoKine/iPlex at the optimal dosage level to test the safety and tolerability of the drug as a daily treatment for six months.

The Optimal Dose SomatoKine/iPlex study is designed to be long enough to provide an initial impression about its potential beneficial effects on muscle mass and strength, although results will not be definitive.

The study visit requirements are the same as in the first phase.

The findings will influence the design and identification of the most appropriate methods to assess safety and effectiveness in a future, phase 2, large-scale, multicenter trial of SomatoKine/iPlex.



OPENING/CLOSING DATES: 11/01/2005 - To Be Determined



TARGET NUMBER OF PARTICIPANTS: 30-40


RECRUITMENT STATUS: Open"

Hope a lot more clarity will come early May but history on Insmed's full disclosure is not good to date.

Rod