Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

Trial Information
Who is this study for? Child to adult patients with Ataxia Telangiectasia
What treatments are being studied? N-Acetyl-L-Cysteine
Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
Summary

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of A-T. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.

Am I eligible for this trial?
Participation Requirements
Sex:
All
Minimum Age:
6
Healthy Volunteers:
No

• Individuals who meet all of the following criteria are eligible to participate in the study:

• Written informed consent signed by the patient and/or their legal representative/ parent

• Male or female aged ≥6 years with a confirmed diagnosis of A-T at the time of signing informed consent.

• Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first dose continuing through 28 days after the last dose) or using one of the following highly effective contraceptives (i.e. results in <1% failure rate when used consistently and correctly) 14 days prior to the first dose continuing through 28 days after the last dose:

• intrauterine device (IUD);

• surgical sterilization of the partner (vasectomy for 6 months minimum);

• combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);

• progestogen-only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);

• intrauterine hormone-releasing system (IUS);

• bilateral tubal occlusion.

• Females of non-childbearing potential must have undergone one of the following sterilization procedures at least 6 months prior to the first dose:

• hysteroscopic sterilization;

• bilateral tubal ligation or bilateral salpingectomy;

• hysterectomy;

• bilateral oophorectomy;

• OR

• be postmenopausal with amenorrhea for at least 1 year prior to the first dose and follicle stimulating hormone (FSH) serum levels consistent with postmenopausal status. FSH analysis for postmenopausal women will be done at screening. FSH levels should be in the postmenopausal range as determined by the central laboratory.

• Non-vasectomized male patient agrees to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study medication and the female partner agrees to comply with inclusion criteria 3 or 4. For a vasectomized male who has had his vasectomy 6 months or more prior to study start, it is required that they use a condom during sexual intercourse. A male who has been vasectomized less than 6 months prior to study start must follow the same restrictions as a non-vasectomized male.

• If male, the patient agrees not to donate sperm from the first dose until 90 days after dosing.

• Patients must fall within:

• a) A SARA score of 5 ≤ X ≤ 33 points (out of 40) AND i. Within the 2-7 range (out of 0-8 range) of the Gait subtest of the SARA scale OR ii. Be able to perform the 9 Hole Peg Test with Dominant Hand (9HPT-D) (SCAFI subtest) in 20 ≤ X ≤150 seconds.

• Weight ≥15 kg at screening.

• Patients are willing to disclose their existing medications/therapies for (the symptoms) of A-T, including those on the prohibited medication list. Non-prohibited medications/therapies (e.g. concomitant speech therapy, and physiotherapy) are permitted provided:

• The Investigator does not believe the medication/therapy will interfere with the study protocol/results

• Patients have been on a stable dose/duration and type of therapy for at least 6 weeks before Visit 1 (Baseline 1)

• Patients are willing to maintain a stable dose/do not change their therapy throughout the duration of the study.

• An understanding of the implications of study participation, provided in the written patient information and informed consent by patients or their legal representative/parent, and demonstrates a willingness to comply with instructions and attend required study visits (for children this criterion will also be assessed in parents or appointed guardians).

Where is this trial taking place?
United States
California
University of California - Los Angeles
Recruiting
Los Angeles
Other Locations
Germany
University of Giessen
Recruiting
Gießen
Ludwig Maximilian University of Munich
Recruiting
München
Spain
Hospital Universitario La Paz
Recruiting
Madrid
United Kingdom
Royal Papworth Hospital NHS Foundation Trust
Recruiting
Cambridge
Who do I contact about this trial?
Primary
Michael Strupp, MD
mstrupp@intrabio.com
+44 8081 641283
Backup
Taylor Fields, MSt
tfields@intrabio.com
+44 8081 641283
When is this trial taking place?
Start Date: January 8, 2020
Estimated Completion Date: March 1, 2023
How many participants will be in this trial?
Target number of participants: 39
What treatment is being studied in this trial?
Experimental: Treatment with IB1001
6-weeks treatment with IB1001 administered orally.~Patients ≥13 years old will receive a total daily dose of 4 g/day (administered as 3 doses per day).~Patients 6-12 years old will receive weight-tiered doses:~Patients aged 6-12 years weighing 15 to <25 kg will take 2 g per day: 1 g in the morning and 1 g in the evening.~Patients aged 6-12 years weighing 25 to <35 kg will take 3 g per day: 1 g in the morning, 1 g in the afternoon, and 1 g in the evening.~Patients aged 6-12 years weighing ≥35 kg will take 4 g per day: 2 g in the morning, 1g in the afternoon and 1 g in the evening (as per adults)~After the 6-week treatment period, patients will enter a 6-week post-treatment washout period.
No Intervention: Post-Treatment Washout
After the 6-week treatment period, patients will enter a 6-week post-treatment washout period.
Who are the authors of this trial?
Susan Perlman, Ignacio Pascual Pascual, Nicholas Oscroft, Anke Hensiek, Susanne Schneider, Andreas Hahn

This content was sourced from clinicaltrials.gov