Research study to determine whether an investigational drug, SHP647, is safe and effective long term in the treatment of moderate to severe Ulcerative Colitis or Crohn's Disease (AIDA)

• Register: EUCTR
• Last refreshed on: 19 February 2024
• Main ID: EUCTR2017-000574-11-IE
• Date of registration: 04/09/2017
• Prospective Registration: Yes
• Primary sponsor: Shire Human Genetic Therapies, Inc.
• Public title: Research study to determine whether an investigational drug, SHP647, is safe and effective long term in the treatment of moderate to severe Ulcerative Colitis or Crohn's Disease (AIDA)
• Scientific title: A Phase 3 Long-term Safety Extension Study of SHP647 in Subjects with Moderate to Severe Ulcerative Colitis or Crohn's Disease (AIDA) - AIDA
• Date of first enrolment: 13/03/2018
• Target sample size: 2453
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-000574-11
• 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: no Placebo: no Other: yes Other specify the comparator: 25 mg or 75 mg SHP647 Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Brazil, Bulgaria, Canada, Colombia, Croatia, Czech Republic, Czechia, Estonia, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Japan, Korea, Republic of, Lebanon, Lithuania, Mexico, Netherlands, New Zealand, Poland, Portugal, Romania, Russian Federation, Serbia, Slovakia, South Africa, Spain, Switzerland, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Holly Oakley
• Affiliation: Shire Human Genetic Therapies, Inc.

• Name: Holly Oakley
• Affiliation: Shire Human Genetic Therapies, Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
Subjects with UC

1. Subjects and/or their parent or legally authorized representative must have an understanding, ability, and willingness to fully comply with
study procedures and restrictions.
2. Subjects must be able to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent and/or assent, as applicable, to participate in the study.
3. Subjects must have been enrolled previously in Study SHP647-301 or SHP647-302 and are in the treatment period of Study SHP647-303, completed the ET or Week 52 visit in the maintenance study SHP647-303, had responded to ontamalimab treatment (in the induction and/or maintenance studies), and meet one of the following criteria:
o Subjects are on placebo at the maintenance study ET or Week 52 visit: they received ontamalimab in the induction studies and fulfilled the maintenance study response entry criteria, OR
o Subjects have received ontamalimab at the maintenance study ET or Week 52 visit:
?-Clinical composite score that has decreased by =2 points and =30%, with an accompanying decrease in the subscore for RB =1 point or a
subscore for RB =1, compared to the baseline value for induction studies, AND/OR
? -Composite score that has decreased by =30% and =3 points compared to the baseline value for induction studies.
4. Subjects receiving any treatment(s) for UC described in Section 5.2.1 are eligible provided they have been on a stable dose for the designated period of time.

Subjects with Crohn's Disease
1. Subjects and/or their parent or legally authorized representative must have an understanding, ability, and willingness to fully comply with
study procedures and restrictions.
2. Subjects must be able to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed
consent and/or assent, as applicable, to participate in the study.
3. Subjects must have been enrolled previously in Study SHP647-305 or SHP647-306 and are in the treatment period of Study SHP647-307,
completed the ET or Week 52 visit in maintenance study SHP647-307, had responded to ontamalimab treatment (in the induction and/or
maintenance studies), and meet one of the following criteria:
o Subjects are on placebo at the maintenance study ET or Week 52 visit:
they received ontamalimab in the induction study and fulfilled the maintenance study response criteria, OR
o Subjects have received ontamalimab at the maintenance study ET or Week 52 visit:
• CDAI score that has decreased by =100 points at the end of treatment visit compared to the baseline value for induction studies, AND/OR
• SES-CD that has decreased by =25% compared to the baseline value for induction studies.
4. Subjects receiving any treatment(s) for CD described in Section 5.3.1 are eligible provided they have been on a stable dose for the designated period of time.
Are the trial subjects under 18? yes
Number of subjects for this age range: 247
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2158
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 48

Exclusion criteria:
Subjects with UC:
1. Subjects who had major protocol deviation(s) (as determined by the sponsor) in Study SHP647-301, SHP647-302, or SHP647-303.
2. Subjects who permanently discontinued investigational product because of an adverse event (AE), regardless of relatedness to
investigational product, in Study SHP647-301, SHP647-302, or SHP647303.
3. Subjects who are likely to require major surgery for UC.
4. Subjects are females who became pregnant during Study SHP647-301, SHP647-302, or SHP647-303, females who are lactating, females who
are planning to become pregnant during the study period, or males or females of childbearing potential not agreeing to continue using appropriate contraception methods (ie, highly effective methods for female and medically appropriate methods for male study subjects) through the conclusion of study participation.
5. Subjects who do not agree to postpone donation of any organ or tissue, including male subjects who are planning to bank or donate sperm and female subjects who are planning to harvest or donate eggs, for the duration of the study and through 16 weeks after last dose of investigational product.
6. Subjects who, in the opinion of the investigator or the sponsor, will be uncooperative or unable to comply with study procedures.
7. Subjects who have a newly-diagnosed malignancy or recurrence of malignancy (other than resected cutaneous basal cell carcinoma, squamous cell carcinoma, or carcinoma in situ of the uterine cervix that has been treated with no evidence of recurrence).
8. Subjects who have developed any major illness/condition or evidence of an unstable clinical condition (eg, renal, hepatic, hematologic, gastrointestinal [GI] [except disease under study], endocrine, cardiovascular, pulmonary, immunologic [eg, Felty's syndrome], or local active infection/infectious illness) that, in the investigator's judgment, will substantially increase the risk to the subject if he or she participates in the study.
9. Subjects with any other severe acute or chronic medical or psychiatric condition or laboratory or electrocardiogram (ECG) abnormality that may
increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study
results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
10. Subjects with known exposure to Mycobacterium tuberculosis (TB) since testing at screening in Study SHP647-301 or SHP647-302 and who
have been advised to require treatment for latent or active disease, but who are without a generally accepted course of treatment.
11. Subjects who are investigational site staff members or relatives of those site staff members or subjects who are sponsor employees directly
involved in the conduct of the study.
12. Subjects who are participating in other investigational studies (other than SHP647-301, SHP647-302, or SHP647-303) or plan to participate in
other investigational studies during long-term extension study SHP647304.

Subjects with CD:
1. Subjects who had major protocol deviation(s) (as determined by the sponsor) in Study SHP647-305, SHP647-306, or SHP647-307.
2. Subjects who permanently discontinued investigational product because of an AE, regardless of relatedness to investigational product, in
Study SHP647-305, SHP647-306, or SHP647-307.
3. Subjects who are likely to require major surgery for CD or developed acute severe

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

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Ulcerative colitis or Crohn's Disease
MedDRA version: 20.1 Level: LLT Classification code 10045365 Term: Ulcerative colitis System Organ Class: 10017947 - Gastrointestinal disorders
MedDRA version: 20.0 Level: PT Classification code 10011401 Term: Crohn's disease System Organ Class: 10017947 - Gastrointestinal disorders

Intervention(s)

Product Code: SHP647
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ontamalimab
Current Sponsor code: SHP647
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 75-

Product Code: SHP647
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ontamalimab
Current Sponsor code: SHP647
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-

Primary Outcome(s)

Main Objective: The primary objective of the study is to evaluate the safety and tolerability of long-term treatment with ontamalimab in subjects with moderate to severe ulcerative colitis (UC) or Crohn's disease (CD).

Secondary Objective: Secondary - Subjects with Ulcerative Colitis:
- To evaluate the maintenance of response to long-term treatment with ontamalimab as measured by clinical composite score and biomarkers, with or without endoscopy.

Secondary - Subjects with Crohn's Disease:
- To evaluate the maintenance of response to long-term treatment with ontamalimab as measured by Crohn's Disease Activity Index (CDAI) score and biomarkers, with or without endoscopy.

Primary end point(s): The primary endpoint is safety assessment as is assessment of safety as measured by: incidence and severity of AEs; incidence and nature of serious infections; actual values and change from baseline, as well as the incidence of abnormalities, in laboratory tests, ECGs, and vital signs; and antidrug antibodies.

Timepoint(s) of evaluation of this end point: Please refer to section “Study Schedules” of the Protocol

Secondary Outcome(s)

Secondary end point(s): Secondary Efficacy Endpoints – Subjects with Ulcerative Colitis:
The secondary endpoints are as follows:
• Treatment response over time, with response defined as clinical composite score that has decreased by =2 points and =30%, with an accompanying decrease in the subscore for RB =1 point or a subscore for RB =1 , and/or composite score that has decreased by =30% and =3 points compared to the baseline value for induction studies.

Secondary Endpoints – Subjects with Crohn's Disease:
The secondary endpoint is as follows:
• Treatment response over time, with response defined as CDAI score that has decreased by =100 points compared to the baseline value for
induction studies and/or SES-CD that has decreased by =25% compared to the baseline value for induction studies.

Timepoint(s) of evaluation of this end point: Secondary endpoints will be summarized by treatment group using descriptive statistics at each assessment visit.

Secondary ID(s)

SHP647-304

NCT03283085

Source(s) of Monetary Support

Shire Human Genetic Therapies, Inc.

Ethics review

Status: Approved
Approval date: 13/03/2018
Contact: