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A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 1
18 Years
Open (Enrolling)
Paroxysmal Nocturnal Hemoglobinuria (PNH)

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Trial Information

A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is an open-label, single-dose, dose-escalation study to assess the safety,
tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC
injection in subjects with PNH or evidence of circulating PNH cells.

Any cohort may be expanded by up to 3 subjects for purposes of safety evaluation based on
specific criteria defined below, and a further 6 subjects may be added to a cohort to obtain
additional safety and PK data. All subjects will sign informed consent and be screened for
eligibility within 28 days of receiving the single dose of TT30. Once a subject is
determined to be eligible for the study, the subject will be vaccinated with meningococcal
vaccine at least two weeks prior to dosing (if not previously vaccinated; if revaccination
is recommended per the manufacturer's guideline, or if vaccinated greater than 3 years ago;
or if subjects have been previously vaccinated, but there is no adequate documentation to
verify prior vaccination).

On Study day 1, the day of TT30 dosing, the subject will come to the GCRC or clinic for the
required testing prior to dosing. Once the testing is complete the subject will receive the
single dose of TT30 as an IV infusion over 60 minutes or as an SC injection. The subject
will be observed for any untoward effects and the required study evaluations will be
completed for the remainder of Study day 1. Subjects may leave the clinic after all
required evaluations are complete and they have been observed for at least 8 hours following
the completion of the IV infusion of TT30.

Subjects will return for the required study evaluations over the next 59 days.

Inclusion Criteria:

- Individuals at least 18 years of age with a diagnosis of PNH & vaccination against

Exclusion Criteria:

- Abnormal renal or liver function

- History of meningococcal disease

- History of Guillain-Barre syndrome

- Known infection with HIV or Hepatitis B or C

- History of thrombotic events

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Assess the safety and tolerability of a single dose of TT30.

Outcome Description:

Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.

Outcome Time Frame:

One year

Safety Issue:



United States: Food and Drug Administration

Study ID:




Start Date:

December 2010

Completion Date:

February 2013

Related Keywords:

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • PNH
  • Hemoglobinuria
  • Hemoglobinuria, Paroxysmal



USC/Norris Comprehensive Cancer CenterLos Angeles, California  90033-0800