Friday, September 25, 2015

Clinical Trials for Acute Porphyrias we still need you!

We still need Medical Heroes for Acute Porphyrias. Part 2

Check out this one.  Please contact the APF:  1-866-APF-3635 ask for Jessica or Natalia for more Information


Trial record 3 of 24 for:    alnylam

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2015 by Alnylam Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02452372
First received: May 19, 2015
Last updated: May 21, 2015
Last verified: May 2015
  Purpose
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.

ConditionInterventionPhase
Acute Intermittent PorphyriaDrug: ALN-AS1
Drug: Sterile Normal Saline (0.9% NaCl)
Phase 1

Study Type:Interventional
Study Design:Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title:A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The safety of ALN-AS1 evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation [ Time Frame: Part A (SAD phase): through day 42; Part B (MAD) phase: through Day 70; Part C (MD) phase: through Day 126 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Profile of Pharmacokinetics (PK) of ALN-AS1 [ Time Frame: Part A (SAD) phase: predose - 42 days post-dose; Part B (MAD) phase: predose - 70 days post-dose; Part C (MD) phase: predose - 126 days post-dose ] [ Designated as safety issue: No ]
    Cmax, tmax, AUC, t1/2
  • The change in delta-aminolevulinic acid (ALA) from baseline [ Time Frame: Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 126 days post-dose ] [ Designated as safety issue: No ]
  • The change in Porphobilinogen (PBG) from baseline [ Time Frame: Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 126 days post-dose ] [ Designated as safety issue: No ]

Estimated Enrollment:48
Study Start Date:May 2015
Estimated Study Completion Date:July 2016
Estimated Primary Completion Date:May 2016 (Final data collection date for primary outcome measure)
ArmsAssigned Interventions
Active Comparator: ALN-AS1Drug: ALN-AS1
Single or multiple doses of ALN-AS1 by subcutaneous (sc) injection
Placebo Comparator: Sterile Normal Saline (0.9% NaCl)Drug: Sterile Normal Saline (0.9% NaCl)
calculated volume to match active comparator

  Eligibility

Ages Eligible for Study:  18 Years to 65 Years
Genders Eligible for Study:  Both
Accepts Healthy Volunteers:  No
Criteria
Parts A and B
Inclusion Criteria:
  • Diagnosis of AIP
  • Urine PBG at Screening indicating patient is a high excreter
  • No clinically significant health concerns
  • Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception
  • Willing to provide written informed consent and willing to comply with study requirements.
Exclusion Criteria:
  • Porphyria attack within 6 months of screening
  • Started a new prescription medication within 3 months of screening
  • Clinically significant abnormal laboratory results
  • Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
  • History of multiple drug allergies or intolerance to subcutaneous injection
Part C
Inclusion Criteria:
  • Diagnosis of AIP
  • Patient experienced a porphyria attack or was taking medication to prevent attacks recently
  • No clinically significant health concerns
  • Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception
  • Willing to provide written informed consent and willing to comply with study requirements.
Exclusion Criteria:
  • Stared a new prescription medication within 3 months of screening
  • Clinically significant abnormal laboratory results
  • Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
  • History of multiple drug allergies or intolerance to subcutaneous injection
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02452372

No comments:

Post a Comment

Acute Intermittent Porphyria strikes Claire Sadowniczak

Claire Sadowniczak Type of Porphyria:  Acute Intermittent Porphyria (AIP) Claire Sadowniczak of Orlando, Florida, is a member of th...