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K1-70 - A Study in Subjects With Graves' Disease

Phase 1
Completed
Conditions
Graves' Disease
Interventions
Drug: K1-70 intramuscular or K1-70 intravenous
Registration Number
NCT02904330
Lead Sponsor
AV7 Limited
Brief Summary

This study is the first time that K1-70 will be administered to humans. The principal aim of this study is to obtain safety and tolerability data when K1-70 is administered as an IM injection or as an IV infusion to subjects with Graves' disease.

Current therapy for Graves' disease includes treatment with anti-thyroid drugs, destruction of the thyroid using radioiodine, or total surgical thyroidectomy. Beta-blockers and calcium antagonists may be used to control some of the symptoms of hyperthyroidism.

K1-70 is a thyroid stimulating hormone receptor antagonist that may provide new in vivo diagnostic and therapeutic tools for the management of patients with Graves' disease, patients with thyroid cancer and patients who would benefit from controlling receptor activity.

Detailed Description

Graves' disease is one of the most common overt autoimmune disorders. Patients with Graves' disease have thyroid over activity and hyperthyroidism. Symptoms of hyperthyroidism include goitre, fatigue, heat intolerance, sweating, weight loss despite good appetite, shakiness, inappropriate anxiety, palpitations of the heart, shortness of breath, tetchiness and agitation, poor sleep, thirst, nausea and increased frequency of defaecation.

The rationale of this study is to obtain safety and tolerability data when K1-70 is administered as an intramuscular injection or as an IV infusion to subjects with Graves' disease.

This information, together with the pharmacokinetic data, will help establish the doses and dosage regimen suitable for repeat administration to patients.

This is an open-label study. The expected duration of each subject's participation in the study is approximately 18 weeks (including a screening period of up to 4 weeks).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
18
Inclusion Criteria
  • age 18-75 years
  • have Graves' disease and are being treated with anti-thyroid medications OR not treated with anti-thyroid medications (due to side-effects) and who are clinically and biochemically euthyroid or hyperthyroid
  • have a body mass index (weight [kg]/height [m]2) between 18.5 and 35.0 kg/m2

Main

Exclusion Criteria
  • current or chronic history of liver disease
  • history of cancer within the last 5 years except localised skin cancer
  • Graves' orbitopathy with clinical activity score >3/7
  • evidence of optic neuropathy and/or corneal breakdown
  • significant systemic infection
  • history of recurrent or current infection
  • splenectomy
  • recently had major surgery or plan major surgery
  • had thromboembolic event due to a blood clot in the last 12 months
  • have clinically significant laboratory tests
  • a clinically significant allergic condition (excluding hay fever)
  • currently receiving corticosteroids
  • smoke more than 10 cigarettes (or its equivalent in nicotine (including use of e-cigarettes)) per day
  • history of drug abuse

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Single doseK1-70 intramuscular or K1-70 intravenousThe intervention is K1-70 intramuscular or K1-70 intravenous. This is a single, ascending, intramuscular or intravenous dose, sequential group study.
Primary Outcome Measures
NameTimeMethod
Safety and tolerability will be measured using vital signs, ECG, blood samples for haematology, coagulation, clinical biochemistry and hormone panel, urinalysis, eye examinations, physical examinations and examination of injection or infusion site.Over a period of 18 weeks

Safety and tolerability testing consists of vital signs, ECG, blood samples for haematology, coagulation, clinical biochemistry and hormone panel, urine samples for urinalysis, eye examinations, physical examinations and examination of injection or infusion site. All clinically significant results and the number of treatment related adverse events will be reported.

Secondary Outcome Measures
NameTimeMethod
The concentration of K1-70 drug in the blood will be measured over time.Over a period of 18 weeks

Percentage of area under the plasma concentration time curve that is extrapolated will be calculated and reported

The antidrug antibodies will be measured to evaluate the immunogenic potential of K1-70 in Graves' disease patientsOver a period of 18 weeks

The level of antidrug antibodies present in the patient serum will be measured over time and reported.

The effect of a single IM dose or single IV dose of K1-70 on thyroid activity in subjects with Graves' will be measured over timeOver a period of 18 weeks

Free T4 levels will be measured and reported over time.

Trial Locations

Locations (2)

Medicines Evaluation Unit

🇬🇧

Manchester, United Kingdom

Royal Liverpool University Hospital Clinical Research Unit

🇬🇧

Liverpool, United Kingdom

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