A study of test product setrusumab in adults with brittle bone syndrome.

Phase 1

• Conditions: Osteogenesis imperfecta; MedDRA version: 20.0 Level: PT Classification code 10031243 Term: Osteogenesis imperfecta System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2016-005096-27-GB
• Lead Sponsor: Mereo Biopharma 3 Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-02-19
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 100

Inclusion Criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
Type of Participant and Disease Characteristics
1. Patients with a clinical diagnosis of OI Type I, III, or IV with a defect in COL1A1/COL1A2, as confirmed by genetic testing
2. Age = 18 years
3. One or more non-traumatic long-bone, rib, hand/feet and/or vertebral fracture(s) in the past 5 years
Sex
4. Male and female
NOTE: The reliability of sexual abstinence for female enrolment eligibility needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the participant. Periodic abstinence (eg, calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception.
Male participants:
5. There are no contraception requirements for male participants with female partners who are woman of childbearing potential (WOCBP).
Female participants:
6. A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
a. Not a WOCBP
OR
b. A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 70 days after the last dose of setrusumab treatment.
Informed Consent
7. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this 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 68
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 32

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:
1. Age > 75 years
2. History of skeletal malignancies or bone metastases at any time
3. History of neural foraminal stenosis (except if due to scoliosis)
4. History of myocardial infarction, angina pectoris, ischaemic stroke or transient ischaemic attack
5. History of or concomitant uncontrolled diseases such as hypo-/hyperparathyroidism, hypo-/hyperthyroidism, Paget’s disease, abnormal thyroid function or thyroid disease or other endocrine disorders or conditions that could affect bone metabolism e.g. Stage IV/V renal disease
6. A history of rickets or osteomalacia or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
7. Documented alcohol and/or drug abuse within 12 months prior to dosing or evidence of such abuse as indicated by the laboratory results during the screening/baseline assessments
8. Documented history of significant psychiatric or medical disorder that would prevent the participant complying with the requirements of the protocol or would make it unsafe for the participant to participate in the study as judged by the investigator
9. Current/previously reported allergy to the study drug or any of its excipients or the class of drug under investigation
Prior/Concomitant Therapy
10. History of external radiation
11. Treatment with any bisphosphonates 3 months prior to baseline and teriparatide, denosumab or other anabolic and anti-reabsorptive medications within 6 months prior to baseline. Note: treatment with zoledronic acid during the follow-up period is explicitly allowed at Months 12 and 18 only at the discretion of the treating physician.
Prior/Concurrent Clinical Study Experience
12. Participation in any clinical investigation within 4 weeks or 5 half-lives of the drug (whichever is longer) prior to initial dosing (or longer if required by local regulations)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified