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SchizophreniaPhase 2AL-108NCT00505765

A Multicenter Study of NAP (AL-108) in Schizophrenia

A Phase 2 study testing AL-108 in people with Schizophrenia, run by University of California, Los Angeles at 8 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The TURNS is a NIMH-funded contract for the evaluation of new compounds for the treatment of cognitive impairments in schizophrenia (HHSN 27820044 1003C; P.I.: Steve Marder, M.D.). Read more about this study

Who is it for?
Ages 18 to 60 years
Conditions the study is for
Schizophrenia
What is being tested?
AL-108
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
63 participants
Where
8 sitesUnited States
Study dates
Jul 2007to Apr 2009
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 60 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • DSM IV/DSM IV TR diagnosis of schizophrenia
  • Capable of providing informed consent
  • Males and Females
  • Age: 18 and 60
  • Caucasian or Non Caucasian
  • Subjects will be treated with one of the following second generation antipsychotics: risperidone, olanzapine, quetiapine, ziprasidone, or aripiprazole for the previous two months, with no change in dose in the last month, and/or with injectable depot antipsychotics (fluphenazine or haloperidol decanoate) with no change in last 3 months.
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  • Subjects will meet the following symptom criteria:
  • Average Brief Psychiatric Rating Scale (BPRS) item score >3 (mild)
  • Simpson-Angus Scale total score less than or equal to 6
  • Calgary Depression Scale total score less than or equal to 10
  • Subjects will meet the following cognitive performance criteria:
  • Performance less than the maximum cutoff (in parentheses) for ONE of the following MCCB tests:
  • Letter-number span (20);
  • HVLT total (31); and
  • CPT d-prime (3.47)
  • Able to complete the baseline MCCB validly as assessed by Chief Neuropsychologist or NP tester
  • Raw score of 6 or greater on the WTAR

Who cannot join

  • Current treatment with oral conventional antipsychotics (e.g. fluphenazine, haloperidol) or clozapine.
  • Subjects with a DSM-IV diagnosis of alcohol or substance abuse (other than nicotine) within the last month or a DSM-IV diagnosis of alcohol or substance dependence (other than nicotine) within the last 6 months
  • Subjects with a history of significant head injury/trauma, as defined by one or more of the following:
  • Loss of consciousness (LOC) for more than 1 hour
  • Recurring seizures resulting from the head injury
  • Clear cognitive sequellae of the injury
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  • Cognitive rehabilitation following the injury
  • Subjects with a clinically significant neurological, metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, and/or urological disorder (e.g. unstable angina, decompensated congestive heart failure, CNS infection or history of HIV seropositivity), which would pose a risk to the patient if they were to participate in the study or that might confound the results of the study.
  • Clinically significant abnormalities in physical examination, ECG, or laboratory assessments.
  • Clinically significant renal disease.
  • Women who are pregnant or of child-bearing potential, either not surgically-sterile nor using appropriate methods of birth control
  • Women who are breast-feeding
  • Prior participation in a clinical trial of investigational medication within 60 days.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites
  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Harvard Medical School

    Boston, Massachusetts, 02215

  • Maryland Psychiatric Research Center

    Catonsville, Maryland, 21228

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • UCLA

    Los Angeles, California, 90073

  • Columbia University Medical Center

    New York, New York, 10032

  • Nathan Kline Institute

    Orangeburg, New York, 10962

  • Washington University School of Medicine

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: A Multicenter Ascending Dose, Double Blind, Placebo-controlled Study of NAP (AL-108) in Chronic Schizophrenia

The TURNS is a NIMH-funded contract for the evaluation of new compounds for the treatment of cognitive impairments in schizophrenia (HHSN 27820044 1003C; P.I.: Steve Marder, M.D.). Despite advances in the safety, tolerability, and effectiveness of antipsychotic medications for the treatment of schizophrenia, many patients continue to be plagued by impairments in social and work functioning. Persons with schizophrenia commonly show deficits in a number of areas of cognition that include impairments in attention, memory, and executive functioning (the ability and organize one's behavior). Importantly, a large body of literature now shows a link between cognition and community functioning in schizophrenia. It is believed that treatments that improve cognitive deficits may lead to improvements in work and social functioning.

One approach to improve the community functioning of patients with schizophrenia is to develop new agents that treat the cognitive deficits of the illness. A promising agent is called AL-108. This drug is administered as a nasal spray. Studies in animals suggest that this drug may protect neurons and may improve cognition in schizophrenia. The current study is a twelve-week multicenter, double-blind, randomized clinical trial of two doses of AL-108 (5 and 30 mg/day intranasally) versus placebo in the treatment of persistent cognitive dysfunction in schizophrenia. The study medication will be added to patients' current atypical antipsychotic medication or to their current injectable first-generation antipsychotic medication. The primary outcome measure will consist of the composite score of the MATRICS neuropsychological battery. Secondary outcome measures will include scores on symptoms, functional outcome, and safety measures. Sixty clinically stable patients with schizophrenia, drawn from eight sites, will participate in the study. Twenty-five patients will be enrolled at UCLA.

Read the detailed description

Background

AL-108 is an intranasal drug product containing NAP, an 8 amino-acid peptide (Asn-Ala-Pro-Val-Ser-Ile-Pro-Gln; NAPVSIPQ, MW=824.9) fragment of the much larger (approx. 124KD) Activity-Dependent Neuroprotective Protein (ADNP), which participates in neurodevelopment and neuroprotection. In mice, ADNP knockouts are lethal exhibiting CNS dysgenesis. ADNP mediates its effects in part through interaction with microtubules. Because of its large size, ADNP is assumed to not penetrate the BBB and thus cannot be used pharmacologically. NAP was chosen because it represents the epitope most associated with microtubule interaction and neuroprotection. NAP is absorbed following IV or intranasal administration, and has been shown to cross the BBB.

Rationale for NAP treatment: tubulin function in brain function

The cytoskeleton plays a key role in maintaining the highly asymmetrical shape and structural polarity of neurons that are essential for neuronal physiology. The cytoskeleton is made up of microfilaments, intermediate filaments and microtubules. Microfilaments (4-9 nm diameter) are made up of actin monomers and they function mainly to provide mechanical support and locomotion to the cell. Intermediate filaments are cytoplasmic fibers of ~10nm diameter. They provide supporting framework within the cell. Microtubules (~24nm diameter) consist of tubulin and microtubule associate proteins. They function to transport nutrients and chemical messengers along the cell. Neurofibrillary tangles are twisted bundles of neurofibrils formed when the microtubule-associated protein, tau, dissociates from microtubules and clusters to form an insoluble mass. Under normal conditions tau binds to microtubules, stabilizing neuronal structure and integrity.

Hyperphosphorylation of tau is assumed to be the cause for the formation of neurofibrillary tangles. Although neurofibrillary tangles are most associated with cognitive dysfunction in Alzheimers disease, some increase in neurofibrillary pathology has also been reported in schizophrenia, potentially as consequence of antipsychotic medication (1). Thus, mechanisms underlying microtubular function may be relevant to schizophrenia as well. In association with tubulin polymerization into microtubules, NAP influences tau dynamics by increasing the ratio of non-phosphorylated tau to phosphorylated tau, implying a dynamic process of cellular maintenance of the microtubular network, which is essential for the survival of the cell.

In brain, tubulin frameworks are stabilized by recently described STOP proteins (2) (aka MAP6). Linkages to allelic variation in STOP genes has been reported in schizophrenia, along with altered STOP protein expression in some brain regions (3). STOP knockdown mice show disturbances in dopaminergic neurotransmission (4) along with deficits in PPI and hypermotility that were partially reversed with clozapine (5). Thus, neuropathological features of schizophrenia may be due, in part, to abnormal STOP-related stabilization of microtubular structure, and NAP may stabilize STOP-related abnormal neurophysiological processes in schizophrenia.

Conditions
Schizophrenia
Being tested
AL-108, Placebo
Keywords
Cognition, Schizophrenia
Medical subject headings
Schizophrenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AL-108Drug

    AL-108, 5 mg/day- one spray in each nostril once per day

  • AL-108Drug

    AL-108, 30 mg/day- 3 sprays in each nostril, twice per day

  • PlaceboDrug

    Placebo- 3 sprays in each nostril, twice per day

  • PlaceboDrug

    Placebo- one spray in each nostril, once per day

Study groups in NCT00505765
Study groupRoleWhat they receive
AL-108, 30 mg/dayExperimentalAL-108, 30 mg/day- 3 sprays in each nostril, twice per day
Receives: Drug: AL-108
AL-108, 5 mg/dayExperimentalAL-108, 5 mg/day- one spray in each nostril once per day
Receives: Drug: AL-108
Placebo, 3 sprays BIDPlacebo comparatorPlacebo- 3 sprays in each nostril, twice per day
Receives: Drug: Placebo
Placebo, 1 Spray DailyPlacebo comparatorPlacebo- one spray in each nostril, once per day
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in MATRICS Consensus Cognitive Battery Composite Score Change

    The MATRICS Consensus Cognitive Battery (MCCB) measures functioning across various cognitive domains and is comprised of ten tests that assess seven cognitive domains (speed of processing, attention/vigilance, working memory, verbal learning, visual learning, reasoning and problem solving, and social cognition) Its measurements are based on timed paper-and-pencil, computerized, and orally-administered tests, as well as spatial tests using geometric cubes. MCCB composite T scores are between 40 and 60 (normal range) and < 40 (below normal range).

    Measured: Baseline, week 6

  2. Change in MATRICS Consensus Cognitive Battery (MCCB)

    The MATRICS Consensus Cognitive Battery (MCCB) measures functioning across various cognitive domains and is comprised of ten tests that assess seven cognitive domains (speed of processing, attention/vigilance, working memory, verbal learning, visual learning, reasoning and problem solving, and social cognition) Its measurements are based on timed paper-and-pencil, computerized, and orally-administered tests, as well as spatial tests using geometric cubes. MCCB composite T scores are between 40 and 60 (normal range) and < 40 (below normal range).

    Measured: Baseline, 12 weeks

Secondary outcomes (4)
  1. Change in UCSD Performance-Based Skills Assessment (UPSA) Summary Scores

    UPSA includes 5 skill areas (subscales) with scores that each range from 0-20. The UPSA yields an overall total score which is the sum of the five subscales and ranges from 0-100. Higher scores are associated with more independent living.

    Measured: Baseline, week 6

  2. Change in UCSD Performance-Based Skills Assessment (UPSA) Summary Scores

    UPSA includes 5 skill areas (subscales) with scores that each range from 0-20. The UPSA yields an overall total score which is the sum of the five subscales and ranges from 0-100. Higher scores are associated with more independent living.

    Measured: Baseline, 12 weeks

  3. Change in SCoRS Interviewer Global Rating

    Schizophrenia Cognition Rating Scale (SCoRS) assessed functional capacity by completing a 20-question rating scale via interviews with the subject and an informant, focusing on cognitive impairment and its impact on daily functioning. After the interview, the interviewer rated subject's overall difficulty on a Global Scale of 1-10. Higher scores indicate greater cognitive impairment.

    Measured: Baseline, 6 weeks

  4. Change in SCoRS Interviewer Global Rating

    Schizophrenia Cognition Rating Scale (SCoRS) assessed functional capacity by completing a 20-question rating scale via interviews with the subject and an informant, focusing on cognitive impairment and its impact on daily functioning. After the interview, the interviewer rated subject's overall difficulty on a Global Scale of 1-10. Higher scores indicate greater cognitive impairment.

    Measured: Baseline, 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Jul 2007 (estimated)
  2. Study startJul 2007
  3. Primary completionApr 2009
  4. Study completionApr 2009

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
63 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00505765
Sponsor's protocol ID
TURNS03
Other IDs
HHSN 278200441003C
Acronym
AL-108
Status last verified
Jan 2017
First submitted
20 Jul 2007
Last updated
10 Mar 2017
Data monitoring committee
Yes

Publications

  • McMahon RP, Arndt S, Conley RR. More powerful two-sample tests for differences in repeated measures of adverse effects in psychiatric trials when only some patients may be at risk. Stat Med. 2005 Jan 15;24(1):11-21. doi: 10.1002/sim.1837.

    PubMed 15515151
  • Edwards D, Madsen J. Constructing multiple test procedures for partially ordered hypothesis sets. Stat Med. 2007 Dec 10;26(28):5116-24. doi: 10.1002/sim.2905.

    PubMed 17476650
  • Jackman AH, Doty RL. Utility of a three-item smell identification test in detecting olfactory dysfunction. Laryngoscope. 2005 Dec;115(12):2209-12. doi: 10.1097/01.mlg.0000183194.17484.bb.

    PubMed 16369168
  • Gozes I, Divinski I. The femtomolar-acting NAP interacts with microtubules: Novel aspects of astrocyte protection. J Alzheimers Dis. 2004 Dec;6(6 Suppl):S37-41. doi: 10.3233/jad-2004-6s605.

    PubMed 15665412
  • Rothermundt M, Arolt V, Bayer TA. Review of immunological and immunopathological findings in schizophrenia. Brain Behav Immun. 2001 Dec;15(4):319-39. doi: 10.1006/brbi.2001.0648.

    PubMed 11782102
  • Rapaport MH, Delrahim KK. An abbreviated review of immune abnormalities in schizophrenia. CNS Spectr. 2001 May;6(5):392-7. doi: 10.1017/s1092852900021763.

    PubMed 15999027
  • Kelly DL, Conley RR. A randomized double-blind 12-week study of quetiapine, risperidone or fluphenazine on sexual functioning in people with schizophrenia. Psychoneuroendocrinology. 2006 Apr;31(3):340-6. doi: 10.1016/j.psyneuen.2005.08.010. Epub 2005 Sep 28.

    PubMed 16198059
  • Gozes I, Meltzer E, Rubinrout S, Brenneman DE, Fridkin M. Vasoactive intestinal peptide potentiates sexual behavior: inhibition by novel antagonist. Endocrinology. 1989 Dec;125(6):2945-9. doi: 10.1210/endo-125-6-2945.

    PubMed 2583049
  • Rotstein M, Bassan H, Kariv N, Speiser Z, Harel S, Gozes I. NAP enhances neurodevelopment of newborn apolipoprotein E-deficient mice subjected to hypoxia. J Pharmacol Exp Ther. 2006 Oct;319(1):332-9. doi: 10.1124/jpet.106.106898. Epub 2006 Jul 5.

    PubMed 16822898
  • Oddo S, Caccamo A, Shepherd JD, Murphy MP, Golde TE, Kayed R, Metherate R, Mattson MP, Akbari Y, LaFerla FM. Triple-transgenic model of Alzheimer's disease with plaques and tangles: intracellular Abeta and synaptic dysfunction. Neuron. 2003 Jul 31;39(3):409-21. doi: 10.1016/s0896-6273(03)00434-3.

    PubMed 12895417
  • Braga RJ, Mendlowicz MV, Marrocos RP, Figueira IL. Anxiety disorders in outpatients with schizophrenia: prevalence and impact on the subjective quality of life. J Psychiatr Res. 2005 Jul;39(4):409-14. doi: 10.1016/j.jpsychires.2004.09.003. Epub 2004 Nov 13.

    PubMed 15804391
  • Alcalay RN, Giladi E, Pick CG, Gozes I. Intranasal administration of NAP, a neuroprotective peptide, decreases anxiety-like behavior in aging mice in the elevated plus maze. Neurosci Lett. 2004 May 6;361(1-3):128-31. doi: 10.1016/j.neulet.2003.12.005.

    PubMed 15135910
  • Gozes I, Alcalay R, Giladi E, Pinhasov A, Furman S, Brenneman DE. NAP accelerates the performance of normal rats in the water maze. J Mol Neurosci. 2002 Aug-Oct;19(1-2):167-70. doi: 10.1007/s12031-002-0028-0.

    PubMed 12212775
  • Gozes I, Giladi E, Pinhasov A, Bardea A, Brenneman DE. Activity-dependent neurotrophic factor: intranasal administration of femtomolar-acting peptides improve performance in a water maze. J Pharmacol Exp Ther. 2000 Jun;293(3):1091-8.

    PubMed 10869414
  • Ito M, Depaz I, Wilce P, Suzuki T, Niwa S, Matsumoto I. Expression of human neuronal protein 22, a novel cytoskeleton-associated protein, was decreased in the anterior cingulate cortex of schizophrenia. Neurosci Lett. 2005 Apr 22;378(3):125-30. doi: 10.1016/j.neulet.2004.12.079.

    PubMed 15781144

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AL-108 in people with Schizophrenia, run by University of California, Los Angeles at 8 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Jan 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in MATRICS Consensus Cognitive Battery Composite Score Change (time frame: Baseline, week 6). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00505765, which also lists 26 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2017.

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