Trial · NCT02949830
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
NCT02949830, Phase 1, Phase 2, Completed, Alnylam Pharmaceuticals; results first posted 2024-03-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP). Trials, record NCT02949830. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02949830
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
- NCT number
- NCT02949830
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 16 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Acute Intermittent Porphyria
- Interventions
- Givosiran
- First posted
- 2016-10-31
- Last updated
- 2024-03-12
- Other registries' IDs
- eudract:2016-002638-54
- Results first posted
- 2024-03-12
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Adverse Events (AEs) | Through Month 49 | 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. |
| Secondary | The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline | Baseline; Month 48 | The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels. |
| Secondary | The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline | Baseline; Month 48 | The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels. |
| Secondary | Annualized Rate of Composite Porphyria Attacks | Through Month 48 | 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. |
| Secondary | Annualized Rate of Hemin Administration | Through Month 49 | 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. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Adverse Events (AEs) | Through Month 49 | SAS: All patients who received any amount of study drug. | percentage of participants | NUMBER |
| SECONDARY | The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline | Baseline; Month 48 | Patients from the PD Analysis Set (all patients who received any amount of study drug and who had at least 1 post-dose blood sample for PD), who were treated with givosiran 2.5 mg/kg SC injection QM. Values that occurred during a porphyria attack were excluded as a means of controlling for potential confounding by hemin. Overall number of participants analyzed is the number of participants available at the given time point. | percent decrease | MEAN |
| SECONDARY | The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline | Baseline; Month 48 | Patients from the PD Analysis Set (all patients who received any amount of study drug and who had at least 1 post-dose blood sample for PD), who were treated with givosiran 2.5 mg/kg SC injection QM. Values that occurred during a porphyria attack were excluded as a means of controlling for potential confounding by hemin. Overall number of participants analyzed is the number of participants available at the given time point. | percent decrease | MEAN |
| SECONDARY | Annualized Rate of Composite Porphyria Attacks | Through Month 48 | SAS: All patients who received any amount of study drug. | composite attacks/total person-years | NUMBER |
| SECONDARY | Annualized Rate of Hemin Administration | Through Month 49 | SAS: All patients who received any amount of study drug. | hemin doses/total person-years | NUMBER |
Source ClinicalTrials.gov
Results by group5
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Adverse Events (AEs) | Givosiran | 100 | percentage of participants | ||
| The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline | Givosiran | 92.531 | Standard Error 1.879 | percent decrease | |
| The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline | Givosiran | 94.194 | Standard Error 2.617 | percent decrease | |
| Annualized Rate of Composite Porphyria Attacks | Givosiran | 0.4 | composite attacks/total person-years | ||
| Annualized Rate of Hemin Administration | Givosiran | 0.9 | hemin doses/total person-years |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Givosiran | 16 | |
| Overall Study | COMPLETED | Givosiran | 14 | |
| Overall Study | NOT COMPLETED | Givosiran | 2 |
Source ClinicalTrials.gov
Adverse events160
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neutrophilia | Blood and lymphatic system disorders | Givosiran | 1 | 16 | |
| other | Palpitations | Cardiac disorders | Givosiran | 1 | 16 | |
| other | Cerumen impaction | Ear and labyrinth disorders | Givosiran | 1 | 16 | |
| other | Ear haemorrhage | Ear and labyrinth disorders | Givosiran | 1 | 16 | |
| other | Tinnitus | Ear and labyrinth disorders | Givosiran | 1 | 16 | |
| other | Conjunctival haemorrhage | Eye disorders | Givosiran | 2 | 16 | |
| other | Eye pain | Eye disorders | Givosiran | 1 | 16 | |
| other | Eye pruritus | Eye disorders | Givosiran | 1 | 16 | |
| other | Swelling of eyelid | Eye disorders | Givosiran | 1 | 16 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Givosiran | 1 | 16 |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Givosiran | 0 | 16 |
| Total, other adverse events | Givosiran | 16 | 16 |
| Total, serious adverse events | Givosiran | 7 | 16 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39363243 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02949830
GET https://conductscience.org/api/trials/v1/NCT02949830/events?page=2