A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)

Roche

Status and phase: Completed; Phase 2

Conditions: Huntington's Disease

Treatments: Drug: RO7234292 (RG6042)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03342053

BN40697

Details and patient eligibility

About

This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.

Enrollment: 46 patients

Sex: All

Ages: 25+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Must have completed dosing in ISIS 443139-CS1

Key Exclusion Criteria:

• Any new condition or worsening of existing condition that could make the patient unsuitable for participation or interfere with the patient participating in and/or completing the study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

46 participants in 2 patient groups

RO7234292 Monthly

Experimental group

Description:

RO7234292 is administered every 28 days intrathecally for 14 months.

Treatment:

Drug: RO7234292 (RG6042)

RO7234292 Bimonthly

Experimental group

Description:

RO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose.

Treatment:

Drug: RO7234292 (RG6042)