The Effect of the Dose of PI-2301 on Efficacy, Safety, and Tolerability, in Subjects with the Relapsing Remitting Form of Multiple Sclerosis: A Phase 2 Randomized, double-blind, four–arm, parallel, placebo-controlled and active descriptive-comparator, 40 week trial

• Register: EUCTR
• Last refreshed on: 10 March 2025
• Main ID: EUCTR2009-012716-40-PL
• Date of registration: 11/01/2010
• Prospective Registration: Yes
• Primary sponsor: Peptimmune Inc.
• Public title: The Effect of the Dose of PI-2301 on Efficacy, Safety, and Tolerability, in Subjects with the Relapsing Remitting Form of Multiple Sclerosis: A Phase 2 Randomized, double-blind, four–arm, parallel, placebo-controlled and active descriptive-comparator, 40 week trial
• Scientific title: The Effect of the Dose of PI-2301 on Efficacy, Safety, and Tolerability, in Subjects with the Relapsing Remitting Form of Multiple Sclerosis: A Phase 2 Randomized, double-blind, four–arm, parallel, placebo-controlled and active descriptive-comparator, 40 week trial
• Date of first enrolment: 06/07/2010
• Target sample size: 350
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-012716-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: yes Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Bulgaria, Czech Republic, Estonia, France, Germany, Poland, Slovakia

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male or female, between the ages of 18 and 55 years (inclusive).
2. Subject is willing and able to comply with the protocol requirements, has gone through the consent process, and has signed an approved informed consent form.
3. Subject is able to learn and self-administer subcutaneous injections (a care-giver may be trained to inject the subject).
4. Patients must have a current diagnosis of RR-MS according to the 2005 revised McDonald MS diagnostic criteria.
5. Subject must have at least one (and may meet 2 or 3 of these criteria) of the following:
• Documented history of 2 MS relapses during the 2 years prior to Screening Visit 1.
OR
• Documented history of 1 MS relapse in the year prior to Screening Visit 1.
OR
• Evidence of at least one gadolinium-enhancing lesion on the Screening MRI (T1 weighted imaging with gadolinium contrast) using a 1.5 or 3.0 Tesla magnet. (MRI magnet strength should be the same for all subjects at a clinical site for all MRI visits and preferably the same MRI machine is used over the course of the trial.)
6. EDSS score of 0-5.5.
7. Female subjects of both childbearing potential and non-childbearing potential may be included, unless the local regulatory authority requires that only women of non-childbearing potential be included. All women of childbearing potential (WOCBP) must test negative for enrollment based on a serum pregnancy test and agree to use a highly reliable method of birth control (such as use of oral contraceptives or Norplant ®; a reliable barrier method i.e. diaphragms with contraceptive jelly; cervical caps with contraceptive jelly; condoms with contraceptive foam; intrauterine devices; partners with vasectomy or abstinence) beginning at least 14 days prior to Screening Visit 1 and continuing throughout the study to End of Study (EOS) Visit 14. WOCBP must not be planning to become pregnant while enrolled in this study.
Non-childbearing potential is defined as one of the following:
• Post-menopausal, defined as amenorrheic (complete cessation of menstruation) for at least 1 year.
• A documented hysterectomy, bilateral oophorectomy or bilateral tubal ligation at least 6 months prior to study initiation.
8. With the exception of signs and symptoms that directly relate to their MS, subjects must be in good general health, per Investigator opinion, without unstable medical conditions, significant physical examination findings, or clinically significant abnormal laboratory results.
9. Subjects must have a negative urine screen for alcohol and drugs of abuse at the Screening Visit 1, unless either opiates or cannaboids were prescribed or recommended by a physician.
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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Any MS categorized as secondary progressive (SP), primary progressive (PP), or progressive relapsing (PR).
2. Any relapse of MS within 30 days of Screening Visit 1 or Baseline Visit 2 or a relapse which has not stabilized.
3. Greater than 20 gadolinium-enhancing lesions on the Screening MRI scan.
4. Systemic glucocorticoid therapy, beta interferon-1a or beta interferon-1b therapy within 30 days of Screening Visit 1 or Baseline Visit 2.
5. Allergy to mannitol.
6. Treatment with azathioprine, cyclosporine or methotrexate within 3 months of Screening Visit 1.
7. Any prior administration of Copaxone® (glatiramer acetate), any biosimilar to Copaxone, Tysabri (natalizumab), Rituxan (rituximab) or history of total lymphoid irradiation.
8. Treatment with cladribine, Cellcept (mycophenolate mofetil), fingolimod, (FTY720), BG-12 (fumarate), Zenapax (daclizumab), laquinimod, teriflunomide, ustekinumab, mitoxantrone, cyclosphosphamide, Campath (alemtuzumab), dirucotide, BHT-3009 within one year of Screening Visit 1.
9. Known to be seropositive for Human Immunodeficiency Virus (HIV) or with known immunosuppression due to acquired immunodeficiency syndrome (AIDS) or other etiology of immunosuppression.
10. Positive serology result for Hepatitis B surface antigen (HbsAg) or anti-Hepatitis C antibody (anti-HCV).
11. History of alcoholism, or of abuse of alcohol.
12. Current or within past 5 years, history of malignancy other than successfully treated squamous or basal cell carcinoma of the skin, or successfully treated in situ cervical cancer.
13. History of clinically unstable gastrointestinal, renal, hepatic, endocrine, pulmonary or cardiovascular disease; or a history of tuberculosis, epilepsy, diabetes, psychosis, glaucoma; or any other condition, which in the opinion of the Investigator, would jeopardize the safety of the subject or impact the validity of the study results.
14. Clinically significant abnormality on screening or baseline electrocardiograms (ECGs) or clinical laboratory results.
15. Subject is pregnant or breastfeeding. Women must have a negative serum beta-human chorionic gonadotropin (ß-HCG) at screening (Visit 1) and a negative urine pregnancy test prior to randomization and the first dose of study medication at Visit 2 Week 1.
16. Contraindications for MRI, including but not limited to, a pacemaker, contrast allergy to gadolinium, impaired renal function contraindicating contrast administration, or inadequately treated claustrophobia making MRI examination unsuitable.
17. Participation in a previous investigational drug or device study within 30 days preceding Screening Visit 1.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Relapsing-remitting multiple sclerosis
MedDRA version: 9.1 Level: LLT Classification code 10063399 Term: Relapsing-remitting multiple sclerosis

Intervention(s)

Product Name: PI-2301
Product Code: PI-2301
Pharmaceutical Form: Solution for injection
CAS Number: 1026791-66-1
Current Sponsor code: PI-2301 acetate salt
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 6-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use

Product Name: PI-2301
Product Code: PI-2301
Pharmaceutical Form: Solution for injection
CAS Number: 1026791-66-1
Current Sponsor code: PI-2301 acetate salt
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use

Trade Name: Copaxone
Product Name: Copaxone
Pharmaceutical Form: Solution for injection
CAS Number: 147245-92-9
Other descriptive name: GLATIRAMER ACETATE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Primary end point(s): MRI: The sum of the total number of gadolinium-enhancing lesions on T1-weighted MRI brain scans at Weeks 24, 28, 32, 36 and 40.

Main Objective: The primary objective of this study is to evaluate the efficacy of PI-2301 by magnetic resonance imaging (MRI), as measured by the sum of the number of total gadolinium-enhancing lesions on serial T1-weighted MRI brain scans at Weeks 24 (Visit 9) through 40 (Visit 13) from 2 dose levels of PI-2301 (3 mg and 10 mg) as compared to placebo, in subjects with relapsing remitting multiple sclerosis (RR-MS) when administered study drug weekly for 40 weeks.

Secondary Objective: To evaluate the effect of two different doses (3 mg and 10 mg) of PI-2301 as compared to placebo on the following parameters:
- The sum of the number of new gadolinium-enhancing lesions on T1 weighted MRI brain scans at Weeks 24 through 40.
- Other MRI markers of lesion activity and burden
- Brain atrophy
- Annualized Relapse Rate
- Week 12 safety as per MRI scans
- Safety and tolerability of PI-2301

Secondary ID(s)

2009-012716-40-EE

CO-200-201

Ethics review

Status: Approved
Approval date: 22/02/2010
Contact: