Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as IGIV-C, IVIG-C
GAMUNEX-C · 8 trials · 7 indications
AUC (0-7 days) is calculated as AUC (0-21 days)/3 for subjects with a dosing frequency of every 3 weeks and as AUC(0-28 days)/4 for subjects with a dosing frequency of every 4 weeks.
The primary PK endpoint (steady-state AUC values) analysis was performed using analysis of variance (ANOVA) using PK data from a total of 49 subjects from the IV phase and 39 subjects from the SC phase.
Mean Change in Quantitative Myasthenia Gravis (QMG) Scale Score from Baseline (Day 0) to Day 14. The minimum and maximum scores of the QMG Scale are 0 and 39, respectively, and a higher score means a worse outcome.
All-cause mortality rate is percentage of participants in each treatment group who experienced mortality up to Day 29.
To measure improvement in MG symptoms by the mean change in QMG total score from Baseline (Week 0) to Week 24 as compared to placebo. Evaluators score 13 individual items (range from 0=best to 3=worst) and the individual scores are added together for the total score (range 0-39). An average 3-point improvement in QMG score indicates clinically meaningful improvement.
The average daily CS dose was derived for each subject at each scheduled visit based on the prescribed dose and the time interval taking into account any prescribed dose changes between routinely scheduled visits. Subjects who discontinued the study early with adverse outcomes related to MG were considered as not achieving a 50% or greater reduction. The missing dose reduction at Week 39 was imputed using the worst observation carried forward (WOCF) method. For subjects who did not have CS dose prescribed at Week 39 due to other reasons, the last observation carried forward (LOCF) method was used to impute the prescribed CS dose at Week 39. Baseline was defined as the last non-missing measurement taken prior to first dose of study medication. The percent of subjects achieving ≥50% reduction in CS dose from baseline to Week 39 is presented for each treatment group overall and for the baseline daily prednisone equivalent dose level stratification categories.
| Arm | Type | Description |
|---|---|---|
| GAMUNEX®-C administered via IV Q4W | EXPERIMENTAL | - |
| Gamunex-C (IGIV-C 10%) | EXPERIMENTAL | During the IV Phase, participants will receive Gamunex-C 1 g/kg, up to a maximum dose of 90 g, every 3 weeks for a total of seven doses. Gamunex-C may be administered over 1 day or divided into 2 consecutive daily doses of 0.5 g/kg each, except for dose #6, which will be administered as a single 1g/kg dose. |
| Xembify (IGSC 20%) | EXPERIMENTAL | Approximately 7 to 10 days after the last Gamunex-C dose, participants will enter a 16-week SC Phase, where they will receive XEMBIFY 0.456 g/kg once weekly for a total of 16 doses, up to a maximum dose of 41 g. |
| Gamunex-C | ACTIVE_COMPARATOR | Participants received Gamunex-C by means of an infusion pump at a dose of 200 to 800 mg/kg per infusion at an infusion rate of 1 mg/kg/min or up to 8 mg/kg/min depending on participant tolerance. The participant's usual mg/kg dose (given on either a 3 or 4 week repeating schedule) was the same mg/kg dose and schedule that the participant was receiving prior to entering screening. This mg/kg dose and schedule were used throughout the study duration. Gamunex-C was administered every 3 weeks (±4 days) or 4 weeks (±4 days), depending on the participant's prior IVIG dosing schedule. The duration of Gamunex-C treatment included the Gamunex-C PK Phase (up to 4 weeks) plus the additional Gamunex-C Run-in Phase (up to 4.5 months) for participants not receiving Gamunex-C or not on a stable dose of Gamunex-C upon entering the trial. The approximate maximum duration was up to 6 months. |
| IVIG-PEG | EXPERIMENTAL | Participants received IVIG-PEG by means of an infusion pump at a dose of 200 to 800 mg/kg per infusion at an infusion rate of 1 mg/kg/min or up to 8 mg/kg/min depending on participant tolerance. IVIG-PEG was administered every 3 weeks (±4 days) or 4 weeks (±4 days), depending on the participant's prior IVIG dosing schedule. The duration of IVIG-PEG treatment included the IVIG-PEG Treatment Phase (up to 4.5 months) and the IVIG-PEG PK Phase (up to 4 weeks), for an approximate maximum of up to 6 months. |
| IGIV-C 10% | ACTIVE_COMPARATOR | IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols) |
| IGSC 20% | EXPERIMENTAL | Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols) |
| IGIV-C Treatment | EXPERIMENTAL | In this arm, subjects with myasthenia gravis exacerbations were treated with an IV dose of 2 g/kg of IGIV-C, which was administered over 2 consecutive days at a dose of 1 g/kg per day. |
| GAMUNEX-C + Standard Medical Treatment | EXPERIMENTAL | Participants received 2 grams per kilogram (g/kg) of GAMUNEX-C, which was capped to a maximum of 160 g infusion intravenously (IV) for participants weighing more than 80 kg on Day 1. The 2 g/kg net total dose was divided either into infusions of 500 mg/kg body weight over 4 days or 400 mg/kg body weight over 5 days as per investigator's decision. Participants received standard of care interventions as per Principal Investigator's discretion from Day 1 up to Day 29. |
| Standard Medical Treatment | ACTIVE_COMPARATOR | Participants received all standard of care interventions required as per Principal Investigator's discretion throughout the participant's hospitalization, from Day 1 to Day 29. |
| IGIV-C | EXPERIMENTAL | IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified. An initial loading dose of 2 g/kg of body weight will be administered at Baseline (Week 0, Visit 1) followed by maintenance doses of 1 g/kg of body weight administered every third week through Week 21 (Visit 8). |
| Placebo | PLACEBO_COMPARATOR | Placebo: Sterile 0.9% sodium chloride injection or equivalent. Placebo will be infused at the Baseline/Week 0 Visit (Visit 1) using the same volume as would be required for the IGIV-C loading dose. Subsequent placebo maintenance doses will be matched in volume to the IGIV-C maintenance doses and administered every third week until Week 21 (Visit 8). |
| Name | Type | Description |
|---|---|---|
| Gamunex-C, 10% Injectable Solution | DRUG | Sterile solution |
| Gamunex-C | BIOLOGICAL | Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (IGIV-C 10%) |
| Xembify | BIOLOGICAL | Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) |
| IVIG-PEG | BIOLOGICAL | Intravenous infusion. |
| IGIV-C 10% | BIOLOGICAL | IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen |
| IGSC 20% | BIOLOGICAL | IGSC 20% weekly infusions with dose calculated based on previous IgG regimen |
| IGIV-C | BIOLOGICAL | An IV dose of 2 g/kg of IGIV-C was administered over 2 consecutive days at a dose of 1 g/kg per day. |
| Standard Medical Treatment | DRUG | SMT per local policies or guidelines. |
| Placebo | DRUG | - |
Inclusion Criteria: Participants with documented and confirmed diagnosis of any of the diseases below: * B-cell CLL according to iwCLL criteria and Rai staging of intermediate (1 and 2) or high (3 and 4); or * MM according to the International Myeloma Working Group criteria (IMWG), R ISS stage II ...
GAMUNEX-C is an immune globulin (human) 10% product studied across several indications, including chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), primary immunodeficiency, and secondary antibody deficiency associated with chronic lymphocytic leukemia, multiple myeloma, or non-Hodgkin lymphoma. It has also been studied in COVID-19. It is administered as an injectable solution.
GAMUNEX-C is developed by Grifols, S.A., which trades under the ticker GRFS. Grifols is the sponsor of the clinical trials evaluating GAMUNEX-C across its studied indications, including CIDP, primary immunodeficiency, secondary antibody deficiency in blood cancers, and COVID-19.
GAMUNEX-C is in Phase 3 clinical development. Its Phase 3 trials include studies in secondary antibody deficiency associated with chronic lymphocytic leukemia, multiple myeloma, or non-Hodgkin lymphoma, in CIDP, and in primary humoral immunodeficiency. A Phase 2 trial in COVID-19 has also been completed.
GAMUNEX-C trials include NCT07582432, a recruiting Phase 3 study in secondary antibody deficiency associated with chronic lymphocytic leukemia, multiple myeloma, or non-Hodgkin lymphoma, and NCT07540221, a recruiting Phase 3 pharmacokinetics and safety study in CIDP. NCT04561115 in primary humoral immunodeficiency and NCT04480424 in COVID-19 are completed.
Yes, GAMUNEX-C is also known as Gamunex-C, 10% Injectable Solution. These names refer to the same immune globulin (human) 10% product developed by Grifols, S.A. under the ticker GRFS, and they appear interchangeably in the clinical trial listings for its studied indications.
GAMUNEX-C clinical trials have enrolled participants across multiple studies. The Phase 3 trial in secondary antibody deficiency associated with blood cancers plans 49 participants, the CIDP Phase 3 study plans 40, the primary humoral immunodeficiency Phase 3 study enrolled 33, and the completed Phase 2 COVID-19 trial enrolled 100.