Graves Disease
Conditions
Keywords
Graves' Disease, IMVT-1401, Batoclimab, Hyperthyroidism
Brief summary
The purpose of this study is to assess the efficacy and safety of 24 weeks of treatment with batoclimab in adult participants with biochemically documented hyperthyroidism due to GD who have failed to achieve euthyroidism on antithyroid drugs (ATDs).
Interventions
DRUGIMVT-1401 (batoclimab)
Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody.
Sponsors
Immunovant Sciences GmbH
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Have serologically confirmed GD as documented by presence of elevated stimulatory thyrotropin receptor antibody (TSH-R-Ab) level (i.e., \> specimen-to-reference ratio of 140%) at the Screening Visit. * Have active hyperthyroidism due to GD with the following laboratory values at the Screening Visit: * TSH \< LLN * FT3 \> upper limit of normal (ULN) and \<=5 \* ULN * FT4 \> ULN and \<=5 \* ULN Note: Participants who have T3 thyrotoxicosis (i.e TSH \<LLN, FT3 \> ULN and ≤5× ULN, but FT4 within normal range) at the Screening Visit may be enrolled, if they have serologically confirmed GD as per Inclusion Criterion 1. * Are willing and capable of giving written informed consent, which includes being able to comply with all aspects of the study treatment and testing schedule.
Exclusion criteria
* History of hyperthyroidism not caused by GD (e.g., toxic adenoma or toxic multinodular goiter), and/or history or presence of thyroid storm. * History of treatment with radioactive iodine or thyroid surgery. * Total immunoglobulin G (IgG) level \<6 grams per liter (g/L) at the Screening Visit. * Albumin level \<3.5 grams per deciliter (g/dL) (\<35 g/L) at the Screening Visit. * Absolute neutrophil count \<1000 cells per cubic millimeter (cells/mm\^3) at the Screening Visit. Other, more specific
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Who Achieved Normalization of Free Triiodothyronine (FT3) and Free Thyroxine (FT4), or Have FT3 and/or FT4 Below the Lower Limit of Normal (LLN) at Week 24 Without Increase in ATD Dose Compared to Baseline | At Week 24 | Antithyroid drug therapy was indicated as a first-line treatment for GD by effectively controlling hyperthyroidism. FT3 and FT4 were prespecified biomarkers of GD. Fasting blood samples were collected to assess the percentage of participants who achieved normalization of FT3 and FT4 or levels below the LLN without increase in ATD dose compared to baseline. The change in the ATD dose was compared only between baseline and Week 24 to determine if there was an increase. Percentages were estimated using the 2-sided Clopper-Pearson Exact method, with corresponding 95% confidence intervals. Participants who, at Week 24, without an increase in ATD dose compared with baseline, had achieved normalization of FT3 and FT4, or had FT3 and/or FT4 below the lower limit of normal (LLN), were considered responders. Participants with missing Week 24 assessments were considered nonresponders. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Who Achieved Normalization of FT3 and FT4 With ATD Dose ≤50% of the Baseline ATD Dose at Week 24 | At Week 24 | Antithyroid drug therapy was indicated as a first-line treatment for GD by effectively controlling hyperthyroidism. FT3 and FT4 were prespecified biomarkers of GD. Fasting blood samples were collected to assess the percentage of participants who achieved normalization of FT3 and FT4 with ATD dose ≤50% of the baseline ATD. The change in the ATD dose was compared only between baseline and Week 24 to determine if there was at least a 50% reduction. Percentages were estimated using the two-sided Clopper-Pearson exact method, with corresponding 95% confidence intervals. |
| Percentage of Participants Who Are Off ATD Treatment and Achieved Normalization of FT3 and FT4, or Had FT3 and/or FT4 Below the LLN at Week 24 | At Week 24 | Antithyroid drug therapy was indicated as a first-line treatment for GD by effectively controlling hyperthyroidism. FT3 and FT4 were prespecified biomarkers of GD. Fasting blood samples were collected to assess the percentage of participants who achieved normalization of FT3 and FT4 or levels below the LLN. Responders were defined as participants who were off ATD therapy at Week 24. Percentages were estimated using the two-sided Clopper-Pearson exact method, with corresponding 95% confidence intervals. |
Countries
Germany
Participant flow
Recruitment details
A total of 32 participants were enrolled in the study in a proof-of-concept, open-label study that evaluated the safety and efficacy of 24 weeks of treatment with batoclimab in adults with biochemically confirmed hyperthyroidism due to Graves' disease (GD) who had failed to achieve euthyroidism on antithyroid drugs (ATDs).
Pre-assignment details
The total study duration was up to 52 weeks, comprising a 4-week screening period, followed by a 24-week treatment period and a 24-week off-treatment period. Eligible participants from the 24-week treatment period entered the 24-week off-treatment period.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 46.9 years STANDARD_DEVIATION 12.43 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 30 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 30 Participants |
| Sex: Female, Male Female | 25 Participants |
| Sex: Female, Male Male | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 32 |
| other Total, other adverse events | 32 / 32 |
| serious Total, serious adverse events | 3 / 32 |
Outcome results
None listed