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Acute Intermittent Porphyria clinical trials at UCSF
2 in progress, 2 open to new patients

  • A Phase 1 Study of ALN-AS1 in Patients With Acute Intermittent Porphyria (AIP)

    open to eligible people ages 18–65

    The purpose of this study is to evaluate the safety and tolerability of ALN-AS1 in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.

    San Francisco, California and other locations

  • Clinical Diagnosis of Acute Porphyria

    open to eligible people ages 15 years and up

    The purpose of this study is to test whether a focused questionnaire and laboratory tests can better define risk factors associated with possible genetic porphyria. The investigators hypothesize that the genetic carrier state of acute porphyria is distinctive enough that the Genetic Carrier Profile the investigators devise through this study will be useful in identifying carriers of genetic porphyria among the large population with undiagnosed abdominal pain.

    San Francisco, California and other locations