IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. 
A Phase 1 Study of ALN-AS1 in Patients With Acute Intermittent Porphyria (AIP)
This study is currently recruiting participants.
Verified June 2017 by Alnylam Pharmaceuticals
Sponsor:
Alnylam Pharmaceuticals
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02452372
First received: May 19, 2015
Last updated: June 2, 2017
Last verified: June 2017
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
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.
| Condition | Intervention | Phase |
|---|---|---|
| Acute Intermittent Porphyria | Drug: ALN-AS1 Drug: Sterile Normal Saline (0.9% NaCl) | Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Participant 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:
Genetics Home Reference related topics:
porphyria
MedlinePlus related topics:
Porphyria
Genetic and Rare Diseases Information Center resources:
Porphyria
Congenital Erythropoietic Porphyria
Acute Intermittent Porphyria
Porphyria Cutanea Tarda
U.S. FDA Resources
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 168 ]
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 - 168 days post-dose ]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 - 168 days post-dose ]
- 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 - 168 days post-dose ]
| Estimated Enrollment: | 72 |
| Study Start Date: | May 2015 |
| Estimated Study Completion Date: | August 2017 |
| Primary Completion Date: | May 2017 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Active Comparator: ALN-AS1 |
Drug: 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 (Adult) |
| Sexes Eligible for Study: | All |
| 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT02452372
Contacts
| Contact: Alnylam Clinical Trials Hotline Call for Complete Site List | 617-575-7400 | ||
| Contact: Alnylam Clinical Trials Hotline Call for Complete Site List | 1-866-330-0326 |
Locations
| United States, Alabama | |
| Clinical Trial Site | Recruiting |
| Birmingham, Alabama, United States | |
| United States, California | |
| Clinical Trial Site | Recruiting |
| San Francisco, California, United States | |
| United States, New York | |
| Clinical Trial Site | Recruiting |
| New York, New York, United States | |
| United States, Texas | |
| Clinical Trial Site | Recruiting |
| Galveston, Texas, United States | |
| Sweden | |
| Clinical Trial Site | Recruiting |
| Stockholm, Sweden | |
| United Kingdom | |
| Clinical Trial Site | Recruiting |
| London, United Kingdom | |
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
| Study Director: | Amy Simon, MD | Alnylam Pharmaceuticals |
More Information
| Responsible Party: | Alnylam Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT02452372 History of Changes |
| Other Study ID Numbers: |
ALN-AS1-001 |
| Study First Received: | May 19, 2015 |
| Last Updated: | June 2, 2017 |
Keywords provided by Alnylam Pharmaceuticals:
|
RNAi therapeutic Porphyria AIP |
Additional relevant MeSH terms:
|
Porphyrias Porphyria, Erythropoietic Porphyria, Acute Intermittent Metabolic Diseases Skin Diseases, Genetic |
Genetic Diseases, Inborn Skin Diseases Porphyrias, Hepatic Liver Diseases Digestive System Diseases |
ClinicalTrials.gov processed this record on July 14, 2017