Last updated: 07/25/2022 08:00:10
An observational, non-interventional study for the follow-up of subjects receiving GSK2398852
Clinicaltrials.gov ID
Not applicable
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
Study complete
Study complete
Trial overview
Official title: An observational, non-interventional study for the follow-up of subjects receiving GSK2398852
Trial description: The objective of the study is to collect comprehensive natural history information on subjects who have received GSK2398852 and GSK2315698, in order to inform the design of future clinical studies. This study will track overall clinical status and key organ function.
Primary purpose:
Not applicable
Trial design:
Not applicable
Masking:
Not applicable
Allocation:
Not applicable
Primary outcomes:
Disease Status
Timeframe: Every 6 months for up to 4 years
Secondary outcomes:
Not applicable
Interventions:
Not applicable
Enrollment:
23
Primary completion date:
2018-01-10
Observational study model:
Cohort
Time perspective:
Prospective
Clinical publications:
Duncan Richards, Helen Millns, Louise Cookson, Mary Ann Lukas. An observational, non-interventional study for the follow-up of patients with amyloidosis who received miridesap followed by dezamizumab in a Phase 1 study. Orphanet J Rare Dis. 2022;17(1):259
DOI: https://doi.org/10.1186/s13023-022-02405-7
PMID: 35810311
Medical condition
Amyloidosis
Product
dezamizumab, miridesap
Collaborators
Not applicable
Study date(s)
May 2015 to October 2018
Type
Observational
Phase
1
Participation criteria
Sex
Female & Male
Age
18 - 70 Years
Accepts healthy volunteers
Not applicable
- 1. Subject has small to moderate amyloid load involving the spleen
- 2. ALT < 3xULN and bilirubin <1.5x ULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
- 1. A positive pre-study Hepatitis B surface antigen or positive Hepatitis C antibody result within 3 months of screening
- 2. The subject has participated in a clinical trial and has received an investigational therapeutic product (unlicensed) within the following time period prior to the first dosing day in the current study: 3 months, 5 half-lives or twice the duration of the biological effect of the investigational product (whichever is longer). This timeframe will not apply to short term administration of GSK2315698 in study CPH114527.
Inclusion and exclusion criteria
Inclusion criteria:
- 1. Subject has small to moderate amyloid load involving the spleen 2. ALT < 3xULN and bilirubin <1.5x ULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%). 3. Male or female between 18 and 70 years of age inclusive, at the time of signing the informed consent. 4. Subject is ambulant and capable of attending for the study visit schedule. 5. Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form. 6. A female subject is eligible to participate if she is of: Non-childbearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea [in questionable cases a blood sample with simultaneous follicle stimulating hormone (FSH) > 40 MlU/ml and estradiol < 40 pg/ml (<147 pmol/L) is confirmatory]. Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use contraception if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of postmenopausal status prior to study enrollment. For most forms of HRT, at least 2-4 weeks will elapse between the cessation of therapy and the blood draw; this interval depends on the type and dosage of HRT. Following confirmation of their post-menopausal status, they can resume use of HRT during the study without use of a contraceptive method.] 7. Male subjects with female partners of child-bearing potential must agree to contraception methods. This criterion must be followed from the time of the first dose of study medication until 85 days post-last dose. 8. Smokers (<10 /day) are permitted but must be willing to abstain for the duration of residential study sessions
Exclusion criteria:
- 1. A positive pre-study Hepatitis B surface antigen or positive Hepatitis C antibody result within 3 months of screening 2. The subject has participated in a clinical trial and has received an investigational therapeutic product (unlicensed) within the following time period prior to the first dosing day in the current study: 3 months, 5 half-lives or twice the duration of the biological effect of the investigational product (whichever is longer). This timeframe will not apply to short term administration of GSK2315698 in study CPH114527. 3. Pregnant females as determined by positive serum or urine hCG test at screening or prior to dosing. 4. Lactating females. 5. Estimated GFR<30 mL/min [<60 mL/min for the first 4 subjects to be enrolled] 6. Evidence of an active urinary sediment on microscopy as evidenced by the presence of red cell casts 7. Decompensated cardiac failure or a recent history of syncope associated with cardiac disease. 8. In a subject in whom there is a clinical suspicion of cardiac amyloid, an echocardiogram is consistent with significant cardiac amyloid, whether symptomatic or not. 9. Clinically significant anaemia- Hb <9 g/dL. 10. Use of prohibited medications 11. Poor or unsuitable venous access. 12. Subjects with a QTcF of >480ms or other ECG abnormalities which, in the opinion of the investigator is clinically significant in that they may increase safety risk. 13. Uncontrolled hypertension with systolic BP > 170 mmHg and /or diastolic > 100 mmHg 14. Presence of any co-morbid condition (e.g. severe or unstable coronary artery disease; moderate-severe chronic obstructive pulmonary disease) which in the opinion of the investigator would increase the potential risk to the subject. 15. Subjects with active vasculitis 16. Subjects with dementia or a diagnosis of cerebral amyloid angiopathy 17. Exclusions from EqMRI scanning Contraindications to MRI scanning including, but not limited to: Intracranial aneurism clips; History of intra-orbital metal fragments that have not been removed by an MD (as confirmed by orbital X-Ray); Pacemakers and non-MR compatible heart valves; Inner ear implants; History of claustrophobia Estimated GFR < 30 mL/min (gadolinium exclusion)
Trial location(s)
Study documents
Protocol
Available language(s): English
Statistical analysis plan
Available language(s): English
Scientific result summary
Available language(s): English
If you wish to request for full study report, please contact - [email protected]
Results overview
Refer to study documents
Recruitment status
Study complete
Actual primary completion date
2018-01-10
Actual study completion date
2018-01-10
Plain language summaries
Not applicable. GSK’s transparency policy provides for Plain Language Summaries for Interventional studies.
Additional information about the trial
Not applicable
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