Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02519036 | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease | PHASE1 | COMPLETED | 46 | — | — | Aug 6, 2015 | Nov 8, 2017 | May 31, 2019 | 9 | Canada, Germany +1 |
An adverse event (AE) was any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. An AE was to be regarded as a TEAE if it was present prior to receiving the first dose of Study Drug and subsequently worsened or was not present prior to receiving the first dose of Study Drug but subsequently appeared.
| Arm | Type | Description |
|---|---|---|
| ISIS 443139 10 mg | EXPERIMENTAL | Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 30 mg | EXPERIMENTAL | Participants received ISIS 443139, 30 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 60 mg | EXPERIMENTAL | Participants received ISIS 443139, 60 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 90 mg | EXPERIMENTAL | Participants received ISIS 443139, 90 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 120 mg | EXPERIMENTAL | Participants received ISIS 443139, 120 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| Placebo | PLACEBO_COMPARATOR | Participants received placebo, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| Name | Type | Description |
|---|---|---|
| ISIS 443139 10 mg | DRUG | ISIS 443139, 10 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 30 mg | DRUG | ISIS 443139, 30 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 60 mg | DRUG | ISIS 443139, 60 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 90 mg | DRUG | ISIS 443139, 90 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 120 mg | DRUG | ISIS 443139, 120 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| Placebo | OTHER | Placebo was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
Key Inclusion Criteria: * Diagnosed with early manifest Huntington's disease * Male or female, aged 25 to 65 years, inclusive, at the time of informed consent * Able and willing to meet all study requirements, including travel to Study Center and participation in all procedures and measurements at ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 2 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |