A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120102 Administered Intrathecally in Patients with Huntington’s Disease
- Conditions
- Huntington’s DiseaseMedDRA version: 20.0 Level: PT Classification code 10070668 Term: Huntington's disease System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2016-005142-39-GB
- Lead Sponsor
- Wave Life Sciences UK Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 48
1. Documented ability to understand the written study ICF(s), and has provided signed written informed consent prior to any study procedures.
2. Ambulatory male or female.
3. Age =25 to =65 years old.
4. Body mass index (BMI) =30.
5. Documented CAG triplet repeats =36 in the Huntingtin gene.
6. Documented heterozygosity for SNP2.
7. Documented presence of the T variant of SNP2 on the same allele as the pathogenic CAG expansion
8. Has clinical diagnostic motor features of HD, defined as UHDRS Diagnostic Confidence Score = 4.
9. Stage I or Stage II HD, defined as UHDRS Total Functional Capacity scores =7 and =13.
10. In the opinion of the Investigator, the patient is able to tolerate all study procedures, and is willing to comply with all other protocol requirements.
11. Willingness to practice highly effective contraception for the duration of the study if patients or their partners are of childbearing potential. Non-childbearing potential and highly effective methods of contraception will be defined in the protocol.
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 48
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
2. Positive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
3. Known to be positive for human immunodeficiency virus (HIV).
4. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
5. Received an investigational drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer.
6. Implantable central nervous system (CNS) device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan.
7. Diagnostic and Statistical Manual of Mental Disorders 5th Edition (DSM-5) diagnosis at the Screening Visit of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening Visit.
8. Positive for opioids (unprescribed), cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit.
9. Started or changed dose for concomitant medication for the treatment of HD symptoms or psychiatric disorders within 30 days prior to the Screening Visit (concomitant medications that have been administered on a stable regimen for =30 days are permitted).
10. Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the course of the study.
11. Clinically significant laboratory abnormality at Screening, including, but not limited to:
a Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values at Screening or Baseline >3 times the upper limit of normal (ULN).
b Renal insufficiency, defined as either as serum creatinine >1.8 mg/dL or creatinine clearance <40 mL/min.
12. Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) =450 msec for males or =470 msec for females.
13. Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary,
gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in
the opinion of the Investigator, precludes the patient’s safe participation in the study or
would interfere with the study assessments.
14. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or
unsuccessful lumbar puncture.
15. Inability to undergo brain MRI (with or without sedation).
16. Deemed to be at significant risk for suicidal behavior based on:
a The opinion of the Investigator; or
b Answers yes” to Actual Suicide Attempts or Suicidal Behaviors in the Suicidal
Behaviors section of the Columbia Suicide Severity Rating Scale (C-SSRS) with
reference to a 2-year period prior to the Screening Visit; or
c Answers yes” on any items in the Suic
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method