Angelman SyndromePhase 2A-MANECENCT02056665
Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
A Phase 2 study testing MINOCYCLINE in people with Angelman Syndrome, run by Puerta de Hierro University Hospital at 1 site in Spain.
Trial summary
RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY) Read more about this study
- Who is it for?
- Ages 6 to 30 years
- Conditions the study is for
- Angelman Syndrome
- What is being tested?
- MINOCYCLINE
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 1 siteSpain
- Study dates
- Jan 2014to Nov 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 6 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female between 6 and 30 years old.
- Clinical diagnosis of Angelman Syndrome and molecular confirmation of diagnosis.
- The participant has an acceptable guardian can give consent on behalf of the participant.
Who cannot join
- Patients with hypersensitivity to tetracyclines.
- Patients with impaired hepatic or renal function and in those with mainly drug allergy history.
- Any other condition that in the opinion of the investigator is considered clinically relevant and that administration of minocycline contraindicated
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
Read the detailed description
STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME
- Conditions
- Angelman Syndrome
- Being tested
- MINOCYCLINE, PLACEBO (for Minocycline)
- Keywords
- Angelman Syndrome, EFFICACY, SAFETY, MINOCYCLINE, Pediatrics population, Rare diseases
- Medical subject headings
- Angelman Syndrome, cyclopia sequence, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MINOCYCLINEDrug
Also called Commercial name: Aknemin 50, Active substance: MINOCYCLINE, Administration routes: Oral use
Pill Minocycline 50 mg capsule
PLACEBO (for Minocycline)Drug
Also called Commercial name: NA, Non Active substance:, Administration routes: Oral use
Pill manufactured to mimic Minocycline 50 mg capsule
| Study group | Role | What they receive |
|---|---|---|
| MINOCYCLINE 8 weeks | Experimental | Duration of treatment: 8 weeks
* Patients <35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner.
* Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner.
* Patients > 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner. Receives: Drug: MINOCYCLINE; Drug: PLACEBO (for Minocycline) |
| PLACEBO 8 weeks | Placebo comparator | Pill manufactured to mimic Minocycline 50 mg capsule Receives: Drug: MINOCYCLINE; Drug: PLACEBO (for Minocycline) |
| MINOCYCLINE 16 weeks | Experimental | Duration of treatment: 16 weeks
Patients <35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner.
Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner.
Patients > 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner. Receives: Drug: MINOCYCLINE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Increased on the equivalent age of development
Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)
Measured: 8, 16 and 24 weeks
Secondary outcomes (4)
Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior
Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R)
Measured: 8, 16 and 24 weeks
Improvement of EEG.
Improvement of EEG. Measure based on changes in the background activity, type, number and duration of crises, widespread tendency to crises, paroxysmal abnormalities recorded types and the overall evaluation of clinical neurophysiologist
Measured: 8, 16 and 24 weeks
Safety and tolerability
- Physical Examination b) Vital signs c) Laboratory Tests d) Adverse effects (AEs) list for treatment, laboratory values, values outside the reference range and descriptive statistics.
Measured: 8, 16 and 24 weeks
Clinical Global impression (CGI)
Improvement of CGI. . Measure based on changes in the Clinical Global Impression through the perception of parents or guardians, you neurologists and therapist
Measured: 8, 16 and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2014 (estimated)
- Study startJan 2014
- Primary completionSep 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Angelman SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Puerta de Hierro University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, BELEN RUIZ-ANTORAN, Puerta de Hierro University Hospital
- Investigators
- Belen Ruiz-Antorán, Principal investigator, Clinical Pharmacology. Puerta de Hierro University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02056665
- Sponsor's protocol ID
- A-MANECE
- Acronym
- A-MANECE
- Status last verified
- Oct 2014
- First submitted
- 5 Feb 2014
- Last updated
- 21 Oct 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Ruiz-Antoran B, Sancho-Lopez A, Cazorla-Calleja R, Lopez-Pajaro LF, Leiva A, Iglesias-Escalera G, Marin-Serrano ME, Rincon-Ortega M, Lara-Herguedas J, Rossignoli-Palomeque T, Valiente-Rodriguez S, Gonzalez-Marques J, Roman-Riechmann E, Avendano-Sola C. A randomized placebo controlled clinical trial to evaluate the efficacy and safety of minocycline in patients with Angelman syndrome (A-MANECE study). Orphanet J Rare Dis. 2018 Aug 20;13(1):144. doi: 10.1186/s13023-018-0891-6.
PubMed 30126448
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
- ClinicalTrials.gov ID
- NCT02056665
- Acronym / sponsor ID
- A-MANECE / A-MANECE
- Conditions
- Angelman Syndrome
- Keywords
- Angelman Syndrome, EFFICACY, SAFETY, MINOCYCLINE, Pediatrics population, Rare diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Puerta de Hierro University HospitalUniversity, hospital or other
- Study start
- Jan 2014
- Primary completion
- Sep 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 6 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
Detailed description
STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| MINOCYCLINE 8 weeks | Experimental | Drug: MINOCYCLINE, Drug: PLACEBO (for Minocycline)Duration of treatment: 8 weeks * Patients <35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner. * Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner. * Patients > 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner. |
| PLACEBO 8 weeks | Placebo comparator | Drug: MINOCYCLINE, Drug: PLACEBO (for Minocycline)Pill manufactured to mimic Minocycline 50 mg capsule |
| MINOCYCLINE 16 weeks | Experimental | Drug: MINOCYCLINEDuration of treatment: 16 weeks Patients <35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner. Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner. Patients > 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner. |
Who may qualify
- Ages
- 6 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Male or female between 6 and 30 years old.
- Clinical diagnosis of Angelman Syndrome and molecular confirmation of diagnosis.
- The participant has an acceptable guardian can give consent on behalf of the participant.
Key exclusion criteria (3 listed)
- Patients with hypersensitivity to tetracyclines.
- Patients with impaired hepatic or renal function and in those with mainly drug allergy history.
- Any other condition that in the opinion of the investigator is considered clinically relevant and that administration of minocycline contraindicated
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MINOCYCLINEAlso: Commercial name: Aknemin 50, Active substance: MINOCYCLINE, Administration routes: Oral usePill Minocycline 50 mg capsule | Drug | MINOCYCLINE 16 weeks, MINOCYCLINE 8 weeks, PLACEBO 8 weeks |
| PLACEBO (for Minocycline)Also: Commercial name: NA, Non Active substance:, Administration routes: Oral usePill manufactured to mimic Minocycline 50 mg capsule | Drug | MINOCYCLINE 8 weeks, PLACEBO 8 weeks |
Primary outcome
Increased on the equivalent age of development
Time frame: 8, 16 and 24 weeks
Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)
4 secondary outcomes
Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior
Time frame: 8, 16 and 24 weeks
Improvement of EEG.
Time frame: 8, 16 and 24 weeks
Safety and tolerability
Time frame: 8, 16 and 24 weeks
Clinical Global impression (CGI)
Time frame: 8, 16 and 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2015.
- Derived from this study: PubMed 30126448Ruiz-Antoran B, Sancho-Lopez A, Cazorla-Calleja R, Lopez-Pajaro LF, Leiva A, Iglesias-Escalera G, Marin-Serrano ME, Rincon-Ortega M, Lara-Herguedas J, Rossignoli-Palomeque T, Valiente-Rodriguez S, Gonzalez-Marques J, Roman-Riechmann E, Avendano-Sola C. A randomized placebo controlled clinical trial to evaluate the efficacy and safety of minocycline in patients with Angelman syndrome (A-MANECE study). Orphanet J Rare Dis. 2018 Aug 20;13(1):144. doi: 10.1186/s13023-018-0891-6.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Belen Ruiz-Antorán principal investigator
Clinical Pharmacology. Puerta de Hierro University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MINOCYCLINE in people with Angelman Syndrome, run by Puerta de Hierro University Hospital at 1 site in Spain.
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 3 inclusion and 3 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 21 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Increased on the equivalent age of development (time frame: 8, 16 and 24 weeks). 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 NCT02056665, which also lists 1 linked publication.
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 21 Oct 2015.