Acute Hepatic PorphyriaNCT04056481
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
A study testing Givosiran in people with Acute Hepatic Porphyria, run by Alnylam Pharmaceuticals.
Trial summary
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Acute Hepatic Porphyria
- What is being tested?
- Givosiran
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)
- Have adequate venous access for program sample collections as judged by the Investigator for study sample collections
Who cannot join
- Previously or currently participating in a givosiran clinical trial
- Has any of the following laboratory parameter assessments at Screening:
- Alanine aminotransferase (ALT) >2×ULN
- Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is <2×ULN
- Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2
- On an active liver transplantation waiting list
About this study
Official title: Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
Read the detailed description
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the link provided in More Information.
- Conditions
- Acute Hepatic Porphyria
- Being tested
- Givosiran
- Keywords
- Acute Hepatic Porphyria (AHP), Acute Intermittent Porphyria (AIP), Porphyria, Acute Intermittent, Acute Porphyria, Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP), Givosiran, Expanded Access, EAP
- Medical subject headings
- Coproporphyria, Hereditary, Porphyria, Acute Intermittent, Porphyrias, Porphyria, Variegate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GivosiranDrug
Also called ALN-AS1
givosiran (ALN-AS1) administered as a subcutaneous (SC) injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Hepatic PorphyriaStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04056481
- Sponsor's protocol ID
- ALN-AS1-005
- Status last verified
- May 2024
- First submitted
- 12 Aug 2019
- Last updated
- 20 May 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)
- ClinicalTrials.gov ID
- NCT04056481
- Acronym / sponsor ID
- ALN-AS1-005
- Conditions
- Acute Hepatic Porphyria
- Keywords
- Acute Hepatic Porphyria (AHP), Acute Intermittent Porphyria (AIP), Porphyria, Acute Intermittent, Acute Porphyria, Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP)
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 14 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
Detailed description
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the link provided in More Information.
Study design
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)
- Have adequate venous access for program sample collections as judged by the Investigator for study sample collections
Key exclusion criteria (6 listed)
- Previously or currently participating in a givosiran clinical trial
- Has any of the following laboratory parameter assessments at Screening:
- On an active liver transplantation waiting list
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GivosiranAlso: ALN-AS1givosiran (ALN-AS1) administered as a subcutaneous (SC) injection | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Alnylam Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A study testing Givosiran in people with Acute Hepatic Porphyria, run by Alnylam Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 May 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2024.