Clinical trials: sponsored by Ionis Pharmaceuticals, Inc.
Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Trial name
- NCT07298447
- Testing
- Donidalorsen
- Where
- 18 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Dravet Syndrome
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
- Trial name
- NCT07531745
- Testing
- ION337
- Where
- 7 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Pelizaeus-Merzbacher Disease
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to…
- Trial name
- NCT05659901
- Where
- 10 sites in United States, France and 5 more
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
- Trial name
- NCT07727538
- Testing
- Olezarsen
- Where
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Angelman Syndrome
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.
- Trial name
- NCT07782827
- Testing
- obudanersen
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Trial name
- NCT06430385
- Testing
- ION440
- Where
- 12 sites in United States, Austria and 3 more
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alexander Disease
Zilganersen Expanded Access Program for Individuals With Alexander Disease
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
- Trial name
- NCT07487389
- Testing
- zilganersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Angelman Syndrome
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
The purpose of this study is to evaluate the efficacy and safety of obudanersen in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
- Trial name
- NCT06914609
- Testing
- obudanersen
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 156 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alexander Disease
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
- Trial name
- NCT04849741
- Testing
- zilganersen
- Where
- 13 sites in United States, Australia and 6 more
- Volunteers
- 54 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Angelman Syndrome
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
- Trial name
- NCT05127226
- Testing
- ION582
- Where
- 11 sites in United States, Australia and 4 more
- Volunteers
- 71 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis
FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
- Trial name
- NCT04768972
- Testing
- ION363
- Where
- 25 sites in United States, Belgium and 14 more
- Volunteers
- 89 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) in cerebrospinal fluid (CSF) and blood; characterize longitudinal…
- Trial name
- NCT06014541
- Where
- 5 sites in United States
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05392114
- Testing
- Donidalorsen
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 154 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Trial name
- NCT06150716
- Testing
- ION356
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05139810
- Testing
- Donidalorsen
- Where
- 51 sites in United States, Belgium and 12 more
- Volunteers
- 91 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Crohn's Disease
Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease
ISIS 2302 is an antisense oligonucleotide drug that reduces the production of a specific protein called intercellular adhesion molecule (ICAM-1), a substance that plays a significant role in the increase of inflammation.
- Trial name
- NCT00048113
- Testing
- Alicaforsen
- Where
- 30 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Lafora Disease
Natural History and Functional Status Study of Patients With Lafora Disease
A natural history and functional status study to characterize the clinical disease course in Lafora disease patients using standardized, quantitative evaluations and to identify useful biomarkers and clinical outcome measures for…
- Trial name
- NCT03876522
- Where
- 4 sites in United States, Italy and 1 more
- Volunteers
- 33 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Crohn's Disease
Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease
ISIS 2302 is an antisense oligonucleotide drug that reduces the production of a specific protein called intercellular adhesion molecule (ICAM-1), a substance that plays a significant role in the increase of inflammation.
- Trial name
- NCT00048295
- Testing
- Alicaforsen
- Where
- 15 sites in Belgium, Czechia and 4 more
- Volunteers
- 150 people
- Sponsor
- Ionis Pharmaceuticals, Inc.