An extension study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of the study drug, RO7234292 (ISIS 443139), in patients who participated in prior investigational studies of RO7234292

Phase 1

• Conditions: Early Manifest Huntington's Disease; MedDRA version: 20.0Level: PTClassification code 10070668Term: Huntington's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-002471-25-DE
• Lead Sponsor: F.Hoffmann La-Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-08-07
• Last Update Posted: 13 October 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 46

Inclusion Criteria

1. Must be capable of giving informed consent (in the opinion of the Investigator)
2. Must have completed the Treatment Period of Study ISIS 443139-CS1
3. Able and willing to meet all study requirements in the opinion of the Investigator,
4. Females must be non-pregnant, non-lactating
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 43
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion Criteria

1. Treatment with an investigational drug (other than ISIS 443139 in Study ISIS 443139-CS1), biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer. Concurrent or planned concurrent participation in any clinical study (including observational and non-interventional studies) without approval of the Sponsor Medical Monitor
2. Antiplatelet or anticoagulant therapy within the 14 days prior to first lumbar puncture in the study or anticipated use during the study, including but not limited to aspirin (unless = 81 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban and apixaban
3. Prior treatment with an antisense oligonucleotide including siRNA (other than ISIS 443139 in Study ISIS 443139-CS1)
4. Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter
5. Clinically-relevant hematological, hepatic, cardiac or renal disease or event. Clinically-significant abnormal hepatic, renal or hematology lab
tests at Screening must be discussed with the Sponsor MedicalMonitor
6. Malignancy within 5 years of Screening, except for basal or squamouscell carcinoma of the skin or carcinoma in situ of the cervix that has
been successfully treated
7. Any condition that significantly increases risk of meningitis unlesspatient is receiving appropriate prophylactic treatment
8. History of bleeding diathesis or coagulopathy, platelet count < LLN unless stable and assessed by the Investigator and Sponsor Medical Monitor to be not clinically significant
9. Have any other condition which, in the opinion of the Investigator or Sponsor, would make the patient unsuitable for inclusion or could
interfere with the patient participating in or completing the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified