This Day On The Street
Continue to site right-arrow
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

Raptor Pharmaceutical To Enroll Five Additional Patients In Pivotal Phase 3 Clinical Trial Of DR Cysteamine For The Potential Treatment Of Nephropathic Cystinosis

Stocks in this article: RPTP

NOVATO, Calif., Feb. 15, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced it has reopened enrollment in its Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis"). The pivotal Phase 3 clinical trial is designed as a study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of DR Cysteamine compared with immediate-release cysteamine bitartrate.

The design of the pivotal Phase 3 clinical trial is a result of discussions with the FDA through which the FDA provided significant guidance on trial protocol design, clinical endpoints, and a statistical analysis plan ("SAP"). The primary endpoint of the trial is the steady-state white blood cell ("WBC") cystine levels of patients taking DR Cysteamine, dosed every twelve hours, compared to immediate-release cysteamine bitartrate, which requires dosing every six hours. At defined intervals during the clinical study, WBC cystine measurements are taken from each patient, one per day, over three day periods. The spread of these daily measurements is used to calculate a statistical value called the intra-patient variance and this value, as defined in the SAP, is used to determine the total number of patients required in the study.

As defined in the SAP, the total number of patients required to complete the study, is determined by an interim statistical analysis of intra-patient variance of the first 20 patients once they had completed the study. Last month, the Company reported completion of enrollment based upon an intra-patient variance report received by the Company, which determined that a total of 30 patients would be required to complete the study. As of February 7, 2011, a total of 31 patients successfully completed the study. Upon final quality control and quality assurance review of the clinical data set, but prior to database lock, it was reported to the Company that the calculation used to determine the intra-patient variance value contained a mathematical error. A repeat of the 20-patient interim analysis of the intra-patient variance using the corrected data resulted in an increase in the required patient number to 36 patients, rather than 30, as previously reported. Therefore, as required in the SAP, the Company is obligated to enroll at least 36 patients in the trial.

1 of 3

Select the service that is right for you!

Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!


DOW 17,778.15 +421.28 2.43%
S&P 500 2,061.23 +48.34 2.40%
NASDAQ 4,748.3960 +104.0840 2.24%

Brokerage Partners

Rates from

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs