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Aqneursa (levacetylleucine)

Ataxia-telangiectasia

Also known as IB1001
Manufacturer
IntraBio
Last updated
206 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care

Corticosteroids

12 evidence topics · 12 sources

Guideline position on steroids for ataxia

“Steroids have been shown to temporarily improve ataxia in ataxia-telangiectasia” (opens the source at this quote in a new tab)

“but should be prescribed with great caution because of their side effects” (opens the source at this quote in a new tab)

“long-term use of steroids may potentially cause severe side effects” (opens the source at this quote in a new tab)

“Some specific treatments to slow the disease progression are being trialled, for example various steroid regimes and amantidine sulphate, but their place in treatment is not yet established.” (opens the source at this quote in a new tab)

Oral betamethasone: randomized crossover trial in 13 children

“In a multicenter, double-blind, randomized, placebo-controlled crossover trial, oral betamethasone (BETA) and placebo were compared in terms of their reduction of ataxia symptoms as assessed with the International Cooperative Ataxia Rating Scale (ICARS).” (opens the source at this quote in a new tab)

“In this study of 13 A-T children, betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (ie, median percent decreases of ataxia symptoms of 28% and 31%, respectively).” (opens the source at this quote in a new tab)

“In conclusion, Oral betamethasone could be a promising therapy to relieve ataxia symptoms in A-T patients; however, long-term effectiveness and safety must be established.” (opens the source at this quote in a new tab)

Oral betamethasone: minimum effective dose in 9 participants

“Nine A-T patients were enrolled to receive betamethasone at increasing dosages of 0.001, 0.005 and 0.01 mg/kg/day.” (opens the source at this quote in a new tab)

“Four of nine patients had a benefit at a dose of 0.005 mg/kg/day of oral betamethasone. Using the higher dosage, only one additional patient had a positive response. Conversely, a daily dose of 0.001 mg/kg was ineffective.” (opens the source at this quote in a new tab)

Oral betamethasone: two years of treatment in 6 participants

“Six patients with ataxia telangiectasia received betamethasone at 0.02 mg/kg/day for two years.” (opens the source at this quote in a new tab)

“Transient improvement of neurological symptom was observed in five of the six patients. However, after two years betamethasone treatment, only one of the six patients showed a slight improvement in the neurological score, one patient showed no change, and the neurological scores of the remaining four patients deteriorated.” (opens the source at this quote in a new tab)

“As an adverse effect of betamethasone, transient adrenal dysfunction was observed in all cases.” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: phase 2 open-label trial

“Twenty two patients (F:M = 1; mean age 11.2 ± 3.5) with a confirmed diagnosis of AT and a preserved or partially supported gait were enrolled for the study. The subjects underwent for six months a monthly infusion of EryDex.” (opens the source at this quote in a new tab)

“An improvement in ICARS (reduction of the score) was detected in the intention-to-treat (ITT) population (n = 22; p = 0.02) as well as in patients completing the study (per protocol PP) (n = 18; p = 0.01), with a mean reduction of 4 points (ITT) or 5.2 points (PP).” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: 24-month extension in 4 participants

“After the end of the first trial, 4 patients continued to be treated with monthly EryDex infusions for an additional 24 months, and their clinical outcome was compared with that of 7 age-matched patients who stopped the treatment after the first 6 infusions.” (opens the source at this quote in a new tab)

“These promising preliminary results call for a large-scale controlled study on protracted treatment of patients with AT with dexamethasone-loaded erythrocytes.” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: ATTeST phase 3 trial design and population

“This multicentre, randomised, double-blind, placebo-controlled, phase 3 trial was done at 22 centres in 12 countries (Australia, Belgium, Germany, India, Israel, Italy, Norway, Poland, Spain, Tunisia, the UK, and the USA).” (opens the source at this quote in a new tab)

“Eligible participants were children aged 6 years or older weighing more than 15 kg who met clinical criteria for ataxia telangiectasia but who had preserved autonomous gait.” (opens the source at this quote in a new tab)

“175 patients received at least one dose of treatment (59 patients received the low dose and 57 received the high dose of intra-erythrocyte dexamethasone sodium phosphate, and 59 received placebo).” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: ATTeST phase 3 trial primary endpoint

“Compared with the placebo group, no differences were identified with regard to change in mICARS score from baseline to 6 months in the low-dose group (least squares mean difference -1·37 [95% CI -2·932 to 0·190]) or the high-dose group (-1·40 [-2·957 to 0·152]; p=0·0765).” (opens the source at this quote in a new tab)

“Although there were no safety concerns, the primary efficacy endpoint was not met, possibly related to delays in treatment reducing the number of participants who received treatment as outlined in the protocol, and potentially different treatment effects according to age.” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: NEAT phase 3 trial design and population

“NEAT was a multicentre, randomised, double-blind, placebo-controlled phase 3 study, conducted at 20 sites across nine countries (Denmark, Germany, Italy, Norway, Poland, Spain, Switzerland, UK, and USA).” (opens the source at this quote in a new tab)

“Between June 24, 2024, and Dec 17, 2025, we screened 125 participants for eligibility, of whom 105 (84%) were randomly assigned to the eDSP group (n=51 [49%]) or the placebo group (n=54 [51%]) and received at least one dose of treatment.” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: NEAT phase 3 trial primary endpoint

“Compared with the placebo group, no significant differences were identified in change in RmICARS score from baseline to 6 months in participants aged 6-9 years: least squares mean difference -1·30 (95% CI -2·77 to 0·18; p=0·085).” (opens the source at this quote in a new tab)

“The primary efficacy endpoint was not achieved, because the effect of eDSP on neurological symptoms did not reach statistical significance.” (opens the source at this quote in a new tab)

“Furthermore, the study did not meet its key secondary endpoint of improvement in Clinical Global Impression of Severity (CGI-S) measured from baseline to month six with a p-value of 0.522.” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: development status

“Company to cease clinical development of eDSP; Intends to preserve cash and explore available options” (opens the source at this quote in a new tab)

Intra-erythrocyte dexamethasone: safety over at least 2 years of treatment

“Sixty-eight patients completed a minimum of 2 years of treatment with EryDex (mean treatment length 39 ± 11 months).” (opens the source at this quote in a new tab)

“Notable adverse events included transient pruritus reported in 23 (34%) patients and findings of low serum iron reported in 27 (40%) patients, while at baseline one fifth of patients had low serum iron.” (opens the source at this quote in a new tab)

“The mean bone mineral density (BMD) z-score showed a decline of 0.4 points over the 24 months of treatment.” (opens the source at this quote in a new tab)