CompletedPhase 2

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

United States301 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 range21 Years

SexALL

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.
✕. Have had previous neurosurgery for Huntington's disease or other movement disorders.
✕. Are a suicide risk, as determined by meeting any of the following criteria:
✕. suicide attempt within one year prior to Baseline.
✕. suicidal ideation as defined by a positive response to question 5 on the C-SSRS (Baseline visit form) suicidal ideation section, within 60 days of the Baseline Visit.
✕. significant risk of suicide, as judged by the site Investigator.
✕. Have marked cognitive impairment with a Montreal Cognitive Assessment (MoCA) Score less than or equal to 22.
✕. Have a presence of clinically significant psychosis and/or confusional states, in the opinion of the site Investigator.

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 range21 Years

SexALL

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.
✕. Have had previous neurosurgery for Huntington's disease or other movement disorders.
✕. Are a suicide risk, as determined by meeting any of the following criteria:
✕. suicide attempt within one year prior to Baseline.
✕. suicidal ideation as defined by a positive response to question 5 on the C-SSRS (Baseline visit form) suicidal ideation section, within 60 days of the Baseline Visit.
✕. significant risk of suicide, as judged by the site Investigator.
✕. Have marked cognitive impairment with a Montreal Cognitive Assessment (MoCA) Score less than or equal to 22.
✕. Have a presence of clinically significant psychosis and/or confusional states, in the opinion of the site Investigator.

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