Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ended
Participants planned
76
Countries
7
Sites
13
Familial Chylomicronemia Syndrome (FCS)
The objectives of the study are to evaluate the efficacy and safety of ARO-APOC3 in adults with FCS
Key facts
- Sponsor
- Arrowhead Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Phenomena and Processes [G] - Genetic Phenomena [G05]
- Trial duration
- 24 Mar 2022 → 21 Apr 2026
- Decision date (initial)
- 2024-11-11
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Arrowhead Pharmaceuticals, Inc.
External identifiers
- EU CT number
- 2024-514336-24-00
- EudraCT number
- 2021-003680-10
- ClinicalTrials.gov
- NCT05089084
- ISRCTN
- ISRCTN12904794
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic, Efficacy, Safety
The objectives of the study are to evaluate the efficacy and safety of ARO-APOC3 in adults with FCS
Conditions and MedDRA coding
Familial Chylomicronemia Syndrome (FCS)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 24.1 | LLT | 10085051 | Familial chylomicronemia syndrome | 100000004848 |
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-003420-PIP01-23
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Men and nonpregnant women, ≥18 years of age (or ≥19 years of age, where applicable according to the local regulation)
- Fasting TG ≥10 mmol/L (≥880 mg/dL) that is refractory to standard lipid-lowering therapy
- Diagnosis of FCS
Exclusion criteria 5
- Recent use of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule
- Active pancreatitis
- Elevated ALT or AST (≥3×ULN)
- Elevated total bilirubin (≥1.5×ULN)
- Glycated hemoglobin (HbA1c) ≥9.0% (or ≥75 mmol/mol International Federation of Clinical Chemistry [IFCC] units) or glomerular filtration rate <30 mL/min/1.73 m2
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Percent change from baseline at Month 10 in fasting TG
Secondary endpoints 3
- Percent change from baseline at Months 10 and 12 (averaged) in fasting TG
- Percent change from baseline at Month 10 in fasting APOC3
- Percent change from baseline at Month 12 in fasting APOC3
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
PRD11241612 · Product
- Active substance
- Synthetic Double-Stranded Sirna Oligonucleotide Directed Against Apolipoprotein C-Iii Mrna and Covalently Linked to a Ligand Containing Three N-Acetylgalactosamine Residues
- Pharmaceutical form
- SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 200 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- ARROWHEAD PHARMACEUTICALS INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2459
PRD9077320 · Product
- Active substance
- Synthetic Double-Stranded Sirna Oligonucleotide Directed Against Apolipoprotein C-Iii Mrna and Covalently Linked to a Ligand Containing Three N-Acetylgalactosamine Residues
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 200 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- ARROWHEAD PHARMACEUTICALS INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2459
Placebo 1
0.9% Normal Saline for injection
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Arrowhead Pharmaceuticals Inc.
- Sponsor organisation
- Arrowhead Pharmaceuticals Inc.
- Address
- 177 East Colorado Boulevard Suite 700
- City
- Pasadena
- Postcode
- 91105-1976
- Country
- United States
Scientific contact point
- Organisation
- Arrowhead Pharmaceuticals Inc.
- Contact name
- Jesse Ho
Public contact point
- Organisation
- Arrowhead Pharmaceuticals Inc.
- Contact name
- Jesse Ho
Third parties 11
| Organisation | City, country | Duties |
|---|---|---|
| Europese Organisatie Voor Onderzoek En Behandeling Van Kanker Organisation Europeenne Pour La Recherche Et Le Traitement Du Cancer European Organi ORG-100010848
|
Sint-Lambrechts-Woluwe, Belgium | Other, E-data capture |
| Fortrea Inc. ORG-100012602
|
Durham, United States | Code 8 |
| 4g Clinical LLC ORG-100042775
|
Wellesley, United States | Interactive response technologies (IRT) |
| Keystone Bioanalytical Inc. ORG-100048363
|
North Wales, United States | Laboratory analysis |
| Medpace Finland Oy ORG-100009147
|
Helsinki, Finland | On site monitoring, Code 12, Other, Code 2, Laboratory analysis, Code 5, E-data capture |
| PCI Pharma Services Germany GmbH ORG-100031981
|
Großbeeren, Germany | Code 14 |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | Data management |
| Charles River Laboratories Montreal ULC ORG-100041009
|
Senneville, Canada | Laboratory analysis |
| Kaleidoscope Data Privacy Consultants Limited ORG-100050361
|
Dublin 7, Ireland | Other |
| The University Of Western Ontario ORG-100044596
|
London, Canada | Laboratory analysis |
| Stichting EuroQol Research Foundation ORG-100048809
|
Rotterdam, Netherlands | Other, E-data capture |
Locations
7 EU/EEA countries · 13 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ended | 1 | 1 |
| Belgium | Ended | 9 | 3 |
| Croatia | Ended | 2 | 1 |
| France | Ended | 4 | 2 |
| Ireland | Ended | 1 | 1 |
| Poland | Ended | 2 | 1 |
| Spain | Ended | 2 | 4 |
| Rest of world
Australia, Korea, Republic of, Japan, Argentina, United States, Oman, Mexico, Serbia, Singapore, Turkey, Israel, Canada, New Zealand
|
— | 55 | — |
Investigational sites
Medical University Of Graz
Klin. Abteilung für Endokrinologie, Neue Stiftingtalstrasse 6, 8010, Graz
Centre hospitalier universitaire de Liege
Endocrinology, Avenue De L'hopital 1, 4000, Liege
Universitair Ziekenhuis Gent
Endocrinology, Corneel Heymanslaan 10, 9000, Gent
UZ Leuven
Endocrinology, Herestraat 49, 3000, Leuven
University Hospital Centre Zagreb
Institute for Metabolic Diseases, Ulica Mije Kispatica 12, 10000, Zagreb
Assistance Publique Hopitaux De Paris
Endocrinology, Metabolism and Prevention of Cardiovascular Diseases Department, Num Voie 47 A 83, 47 Boulevard De L Hopital, Paris
Assistance Publique Hopitaux de Marseille (AP-HM) - Hôpital La Conception
Department of Nutrition, Metabolic diseases and Endocrinology, 147, boulevard Baille, Marseille
Instytut Centrum Zdrowia Matki Polki
Klinika Kardiologii i Wad Wrodzonych Dorosłych, Ul. Rzgowska 281/289, 93-338, Lodz
Complexo Hospitalario Universitario De Santiago
Endocrinology, Calle Choupana Da S/n, 15706, Santiago De Compostela
Hospital General Universitario Gregorio Maranon
internal medicine, Calle Del Doctor Esquerdo 46, 28009, Madrid
Area Sanitaria Da Coruna E Cee
internal medicine, Lugar Jubias De Arriba Num 84, 15006, A Coruna
Hospital Universitario Virgen De Las Nieves
internal medicine, Avenida De Las Fuerzas Armadas 2, 18014, Granada
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Austria | 2022-03-24 | 2025-05-20 | 2022-04-26 | 2022-06-29 | |
| Belgium | 2022-08-31 | 2026-03-31 | 2022-10-11 | 2023-03-30 | |
| Croatia | 2023-03-17 | 2026-01-27 | 2023-03-23 | 2023-04-17 | |
| France | 2022-10-18 | 2026-03-31 | 2022-10-26 | 2023-04-03 | |
| Ireland | 2023-03-16 | 2024-10-21 | 2023-03-29 | 2023-03-29 | |
| Poland | 2022-04-14 | 2025-06-16 | 2022-05-10 | 2022-05-10 | |
| Spain | 2023-03-31 | 2026-01-21 | 2023-03-31 | 2023-04-04 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 87 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-514336-24_Arrowhead_redacted | 8.0 |
| Protocol (for publication) | D1_Protocol_2024-514336-24_redacted | 5.0 |
| Protocol (for publication) | D4_Patient facing documents_EORTC QLQ-C30_English_2024-514336-24-00_Arrowhead | 1 |
| Protocol (for publication) | D4_Patient facing documents_EORTC QLQ-PAN26_2024-514336-24-00_Arrowhead | 1 |
| Protocol (for publication) | D4_Patient facing documents_EQ-5D-5L_2024-514336-24-00_Arrowhead | 1 |
| Recruitment arrangements (for publication) | 2024-514336-24_RECRUTEMENT_AROAPOC3-3001_Blank | N/A |
| Recruitment arrangements (for publication) | 2024-514336-24-00_DOCUMENT_Doc Additionel_AROAPOC3-3001 | N/A |
| Recruitment arrangements (for publication) | 2024-514336-24-00_DOCUMENT_Recruitment and Informed Consent procedure_AROAPOC3-3001_Blank | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_AUT_ Arrowhead | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_BEL_Arrowhead_blank | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_IE_Arrowhead_blank | n/a |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_MS_Arrowhead_blank | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_PL_Arrowhead_blank | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_SP_Arrowhead_blank | N/A |
| Recruitment arrangements (for publication) | K2_Recruitment material_Description_Arrowhead_blank | N/A |
| Subject information and informed consent form (for publication) | 2024-514336-24_NIFC_Addendum_AROAPOC3-3001 | 2.0 |
| Subject information and informed consent form (for publication) | 2024-514336-24_NIFC_Futur Research_AROAPOC3-3001_redacted | 1.1 |
| Subject information and informed consent form (for publication) | 2024-514336-24_NIFC_Home study visit_AROAPOC3-3001_redacted | 1.1 |
| Subject information and informed consent form (for publication) | 2024-514336-24_NIFC_Main_AROAPOC3-3001_Clean | 6.0 |
| Subject information and informed consent form (for publication) | 2024-514336-24_NIFC_PK_AROAPOC3-3001_redacted | 1.1 |
| Subject information and informed consent form (for publication) | 2024-514336-24_NIFC_Pregnancy_AROAPOC3-3001_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_ HomeHealthcare_Arrowhead_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_ Instructions for self-injection_ Arrowhead | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future Research ICF_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future Research ICF_PL_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future Research_Arrowhead_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future Research_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home Healthcare ICF_Arrowhead | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home healthcare ICF_DU_Arrowhead | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home healthcare ICF_EN_Arrowhead | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home healthcare ICF_FR_Arrowhead | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home healthcare ICF_PL_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home Healthcare_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home Study Visit ICF_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Addendum ICF_Arrowhead | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Addendum_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_Arrowhead | 5.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_Arrowhead_redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_DU_Arrowhead | 7.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_EN_Arrowhead | 7.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_FR_Arrowhead | 7.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_PL_Arrowhead_redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Arrowhead_redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Arrowhead_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_New Info Addendum ICF_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_New Information Addendum ICF_DU_Arrowhead | N/A |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_New Information Addendum ICF_EN_Arrowhead | N/A |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_New Information Addendum ICF_FR_Arrowhead | N/A |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_New Information Addendum ICF_PL_Arrowhead | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Collection ICF_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Pharmacokinetic ICF_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PK ICF_Arrowhead Pharmaceuticals | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PK ICF_PL_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PK_Arrowhead_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PK_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Follow Up ICF_DU_Arrowhead | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Follow Up ICF_EN_Arrowhead | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Follow Up ICF_FR_Arrowhead | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_Arrowhead_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant ICF_PL_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Arrowhead_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner ICF_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner ICF_PL_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Patient or Partner ICF_Arrowhead | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PregnantParticipant ICF_Arrowhead_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_Instructions for Self-Administration_Arrowhead | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Description_Arrowhead_blank | N/A |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_DU_2024-514336-24_arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_FR_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_protocol lay synopsis_FR_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_GER_2014-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_PL_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_Spanish_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_Croatian_2024-514336-24-00_Arrowhead Pharmaceuticals | 5 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_DU_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_Dutch_2024-514336-24-00_Arrowhead | 5 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_ENG_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_French_2024-514336-24_Arrowhead_Clean | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_French_2024-514336-24-00_Arrowhead | 5 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_French_2024-514336-24-00_Arrowhead | 5.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_GER_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_German_2024-514336-24-00_Arrowhead | 5 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_German_2024-514336-24-00_Arrowhead | 5.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_Polish_2024-514336-24_Arrowhead | 8.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_Spanish_2024-514336-24-00_Arrowhead | 1 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-09 | Spain | Acceptable with conditions 2024-09-26
|
2024-09-26 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-02-12 | Spain | Acceptable 2025-05-09
|
2025-05-09 |
| 3 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-07-11 | Spain | Acceptable 2025-08-19
|
2025-08-20 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-10-23 | Spain | Acceptable 2025-08-19
|
2025-10-23 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-04-24 | Spain | Acceptable 2025-08-19
|
2026-04-24 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2026-05-07 | Acceptable 2025-08-19
|
2026-05-07 |