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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02949830
Other study ID # ALN-AS1-002
Secondary ID 2016-002638-54
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date October 2016
Est. completion date November 5, 2021

Study information

Verified date March 2024
Source Alnylam Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).


Recruitment information / eligibility

Status Completed
Enrollment 16
Est. completion date November 5, 2021
Est. primary completion date November 5, 2021
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Completed participation in Part C of study ALN-AS1-001 (NCT02452372) - Not on a scheduled regimen of hemin - Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception - Willing and able to comply with the study requirements and to provide written informed consent Exclusion Criteria: - Clinically significant abnormal laboratory results - Received an investigational agent (other than ALN-AS1) 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

Study Design


Intervention

Drug:
Givosiran
Givosiran by subcutaneous (SC) injection.

Locations

Country Name City State
Sweden Clinical Trial Site Stockholm
United Kingdom Clinical Trial Site London
United States Clinical Trial Site Galveston Texas
United States Clinical Trial Site New York New York
United States Clinical Trial Site San Francisco California

Sponsors (1)

Lead Sponsor Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  Sweden,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of Participants With Adverse Events (AEs) An AE is any untoward medical occurrence in a participant or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. Through Month 49
Secondary The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels. Baseline; Month 48
Secondary The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels. Baseline; Month 48
Secondary Annualized Rate of Composite Porphyria Attacks Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. Composite porphyria attacks included porphyria attacks that required hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home. The annualized attack rate (AAR) was calculated as the number of composite porphyria attacks/total person-years. Through Month 48
Secondary Annualized Rate of Hemin Administration The annualized rate of hemin administration was evaluated by annualized days of hemin use, which is calculated as the number of doses of hemin administered/total person-years. Through Month 49
See also
  Status Clinical Trial Phase
Terminated NCT02922413 - Panhematin for Prevention of Acute Attacks of Porphyria Phase 2
Completed NCT02943213 - Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride Phase 1
Completed NCT02082860 - Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria Phase 1
Completed NCT03338816 - ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) Phase 3
Completed NCT02452372 - A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) Phase 1
Completed NCT00418795 - Porphozym in the Treatment of Acute Attacks in AIP Phase 2/Phase 3
Active, not recruiting NCT01617642 - Dental Health, Diet, Inflammation and Biomarkers in Patients With Acute Intermittent Porphyria(AIP)
Completed NCT02076763 - Observational Study of Acute Intermittent Porphyria Patients N/A
Active, not recruiting NCT02935400 - Acute Porphyria Biomarkers for Disease Activity