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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002638-54 | EudraCT Number |
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Givosiran | Experimental | At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly(QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Givosiran | Drug | Givosiran by subcutaneous (SC) injection. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | San Francisco | California | United States | |||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39363243 | Derived | Sardh E, Balwani M, Rees DC, Anderson KE, Jia G, Sweetser MT, Wang B. Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study. Orphanet J Rare Dis. 2024 Oct 3;19(1):365. doi: 10.1186/s13023-024-03284-w. |
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Patients who completed parent study ALN-AS1-001 (NCT02452372) and met all eligibility criteria for this study (ALN-AS1-002) were enrolled.
Patients with acute intermittent porphyria (AIP) were enrolled at five sites in Sweden, United Kingdom and the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | Givosiran | At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly (QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 29, 2021 | Nov 5, 2022 |
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The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels. |
| Baseline; Month 48 |
| 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 |
| 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 |
| New York |
| New York |
| United States |
| Clinical Trial Site | Galveston | Texas | United States |
| Clinical Trial Site | Stockholm | Sweden |
| Clinical Trial Site | London | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety Analysis Set (SAS): All patients who received any amount of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Givosiran | At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly (QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | SAS: All patients who received any amount of study drug. | Posted | Number | percentage of participants | 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. | 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. | Posted | Mean | Standard Error | percent decrease | 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. | 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. | Posted | Mean | Standard Error | percent decrease | 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. | SAS: All patients who received any amount of study drug. | Posted | Number | composite 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. | SAS: All patients who received any amount of study drug. | Posted | Number | hemin doses/total person-years | Through Month 49 |
|
|
Treatment-emergent adverse events (AEs) are any AE with onset after the first administration of study drug through the end of exposure, or any event that was present at Baseline but worsened in severity or was subsequently considered drug-related by the Investigator (up to approximately 52 months).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Givosiran | At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly (QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM. | 0 | 16 | 7 | 16 | 16 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Forearm fracture | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA23.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA23.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutrophilia | Blood and lymphatic system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA23.0 | Systematic Assessment |
| |
| Cerumen impaction | Ear and labyrinth disorders | MedDRA23.0 | Systematic Assessment |
| |
| Ear haemorrhage | Ear and labyrinth disorders | MedDRA23.0 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA23.0 | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA23.0 | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA23.0 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA23.0 | Systematic Assessment |
| |
| Swelling of eyelid | Eye disorders | MedDRA23.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Discomfort | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Facial pain | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site discolouration | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site dryness | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site indentation | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site rash | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Injection site urticaria | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA23.0 | Systematic Assessment |
| |
| Allergy to animal | Immune system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Oral allergy syndrome | Immune system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA23.0 | Systematic Assessment |
| |
| Arthropod sting | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Venomous sting | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Albumin urine present | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Anticoagulation drug level below therapeutic | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Bilirubin conjugated increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Blood homocysteine increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Blood sodium decreased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Carbon dioxide increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Coronavirus test positive | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Creatinine urine increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Glomerular filtration rate decreased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Liver function test increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Protein urine present | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Prothrombin level increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Urine ketone body present | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Urine output decreased | Investigations | MedDRA23.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA23.0 | Systematic Assessment |
| |
| Gluten sensitivity | Metabolism and nutrition disorders | MedDRA23.0 | Systematic Assessment |
| |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | MedDRA23.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Costochondritis | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Dysaesthesia | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA23.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA23.0 | Systematic Assessment |
| |
| Attention deficit hyperactivity disorder | Psychiatric disorders | MedDRA23.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA23.0 | Systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA23.0 | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA23.0 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA23.0 | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA23.0 | Systematic Assessment |
| |
| Metrorrhagia | Reproductive system and breast disorders | MedDRA23.0 | Systematic Assessment |
| |
| Oligomenorrhoea | Reproductive system and breast disorders | MedDRA23.0 | Systematic Assessment |
| |
| Allergic bronchitis | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pharyngeal erythema | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA23.0 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Palmar erythema | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA23.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA23.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA23.0 | Systematic Assessment |
| |
| Superficial vein prominence | Vascular disorders | MedDRA23.0 | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA23.0 | Systematic Assessment |
|
It is intended that after completion of the study, the data are to be submitted for publication in a scientific journal and/or for reporting at a scientific meeting. A copy of any proposed manuscript must be provided and confirmed received at the Sponsor at least 30 days before its submission, and according to any additional publication details in the Investigator Agreement.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Alnylam Pharmaceuticals Inc. | 866-330-0326 | clinicaltrials@alnylam.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 17, 2019 | Nov 5, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D017118 | Porphyria, Acute Intermittent |
| D011164 | Porphyrias |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000630124 | givosiran |
Not provided
Not provided
Not provided
| White |
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| United States of America |
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