CompletedPhase 2

A Study in Subjects With Late Prodromal & Early Manifest HD to Assess the Safety, Tolerability, pk, and Efficacy of Pepi

United States, Canada301 participantsStarted 2015-07

Plain-language summary

The purpose of this study is to evaluate the safety, tolerability, PK, and efficacy of Pepinemab in subjects with late prodromal and early manifest Huntington's disease.

Who can participate

Age range

21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Male or female and are at least greater than or equal to 21 years of age at Screening.
. Must fulfill one of the following criteria at Screening:
. Late prodromal HD as defined by a CAP score of greater than 200 and DCL 2 or 3.
. Early manifest HD as defined by a TFC greater than or equal to 11. Subject must have been determined to have a clinical diagnosis of HD by the Site Investigator as defined by a DCL of 4.
. Must fulfill both of the following criteria at Screening:
. Have undergone genetic testing with a known CAG repeat greater than or equal to 36.
. No features of juvenile HD (Westphal variant).
. If female must be either surgically sterile, postmenopausal, or nonlactating and nonpregnant. Female subjects of childbearing potential must practice a highly effective method of contraception.

Exclusion criteria

. Have participated in an investigational drug or device study within 30 days of the Baseline Visit, or 180 days if previous investigational drug was a MAb therapeutic, or previously participated in SIGNAL Cohort A. This does not apply to Cohort B subjects who are being offered the option to participate in the extension of Cohort B.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Revisions to SAP were made prior to un-blinding, on June 30, 2020, upon consultation with FDA.

Timeframe: Prior to DBL/Study Completion

Safety and tolerability of monthly intravenous (IV) administration of pepinemab relative to placebo in subjects with early HD (Cohort B pooled, includes Cohort B1 Early Manifest and Cohort B2 Late Prodromal HD).

Timeframe: Up to 18 months

Efficacy of monthly IV administration of pepinemab relative to placebo in Early Manifest HD (Cohort B1)

Timeframe: Up to 18 months

Trial details

NCT IDNCT02481674

SponsorVaccinex Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-08

View on ClinicalTrials.gov More Huntington's Disease trials

Who can participate

Age range

21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Male or female and are at least greater than or equal to 21 years of age at Screening.
. Must fulfill one of the following criteria at Screening:
. Late prodromal HD as defined by a CAP score of greater than 200 and DCL 2 or 3.
. Early manifest HD as defined by a TFC greater than or equal to 11. Subject must have been determined to have a clinical diagnosis of HD by the Site Investigator as defined by a DCL of 4.
. Must fulfill both of the following criteria at Screening:
. Have undergone genetic testing with a known CAG repeat greater than or equal to 36.
. No features of juvenile HD (Westphal variant).
. If female must be either surgically sterile, postmenopausal, or nonlactating and nonpregnant. Female subjects of childbearing potential must practice a highly effective method of contraception.

Exclusion criteria

. Have participated in an investigational drug or device study within 30 days of the Baseline Visit, or 180 days if previous investigational drug was a MAb therapeutic, or previously participated in SIGNAL Cohort A. This does not apply to Cohort B subjects who are being offered the option to participate in the extension of Cohort B.

What they're measuring

Revisions to SAP were made prior to un-blinding, on June 30, 2020, upon consultation with FDA.

Timeframe: Prior to DBL/Study Completion

Timeframe: Up to 18 months

Efficacy of monthly IV administration of pepinemab relative to placebo in Early Manifest HD (Cohort B1)

Timeframe: Up to 18 months