Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

NCT01255358 | Completed Phase 2 Results

• 2 other identifiers: IEDAT-012010-022315-19
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 2

Brief Summary

The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).

Conditions

Nervous System Disorder; Genetic Syndrome

Keywords

Ataxia Teleangiectasia; AT; ERY-DEX; Dexamethasone; Dexamethasone sodium phosphate

Outcome Measures

Primary Outcomes (2)

• Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population

  ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.

  At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)

• Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population

  ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.

  At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)

Secondary Outcomes (7)

• Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population

  At visit 7 (final visit, day 180)

• Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population

  At visit 7 (final visit, day 180)

• Mean Change From V4 to V7 in Ocular Motility - ITT Population

  At visit 7 (final visit, day 180)

• Mean Change From V4 to V7 in Ocular Motility - PP Population

  At visit 7 (final visit, day 180)

• Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population

  At visits 4 (90 days) and 7 (180 days, final visit)

Study Arms (1)

ERY-DEX

EXPERIMENTAL

Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)

Drug: Dexamethasone

Interventions

Dexamethasone DRUG

Dexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). The treatment has to be repeated at intervals of 30 days (±10 days)

Also known as: Dexamethasone sodium phosphate, Dex 21P

ERY-DEX

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• neurological signs of AT
• patients in autonomous gait or helped by a support
• proven molecular diagnosis of AT
• Males and females aged \> 3 years
• Body weight \>15 kg
• Plasma levels of Lymphocytes CD4+/mm3 \> 500 (for patients aged 3-6 years) or \> 200 (older than 6 years)
• written IC to participate.

You may not qualify if:

• Current or previous neoplastic disease
• History of severe impairment of the immunological system
• Chronic conditions representing a contraindication to the use of steroid drugs
• Non compliance with the study request
• Any previous steroid assumption within 30 days before starting ERY-DEX
• Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
• Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods

Sponsors & Collaborators

• Quince Therapeutics S.p.A.: lead

Study Sites (2)

Spedali Civili

Brescia, 25123, Italy

Location

University La Sapienza

Rome, 00185, Italy

Location

MeSH Terms

Conditions

Nervous System Diseases; Genetic Diseases, Inborn

Interventions

Dexamethasone; dexamethasone 21-phosphate

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Results Point of Contact

• Title: Clinical Study Coordinator
• Organization: EryDel spa

guenter.janhofer@erydel.com

+39 02 36504470

Study Officials

• Luciana Chessa, MD

  A.O. Sant'Andrea Rome Italy

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2010
• First Posted: December 7, 2010
• Study Start: February 1, 2011
• Primary Completion: September 1, 2011
• Study Completion: December 1, 2011
• Last Updated: October 10, 2024
• Results First Posted: October 10, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

IT (2)